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MT2013-06C : Treatment of graft Failure after HSCT
Troy Lund
All
Not specified
N/A
NCT02161783
1404M49341
Inclusion Criteria:
• Patients with primary or secondary graft failure, as defined below, may receive a second transplant:
• Primary graft failure is defined as not achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42 following the first transplant.
• Secondary graft failure is defined as achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42, but subsequently drops below 0.5x10^9/L without recovery.
• Loss of chimerism is defined as achieving an ANC ≥0.5x10^9/L for three consecutive, but with less than 10% CD15+ donor cells in the marrow or peripheral blood.
• Recipients should have acceptable organ function defined as:
• Renal: creatinine < 2.0 (adults) and creatinine clearance > 30. For creatinine clearance < 70, consultation with a BMT pharmacist is necessary for chemotherapy dose adjustments.
• Hepatic: bilirubin, AST/ALT, ALP < 10 x upper limit of normal
• Cardiac: left ventricular ejection fraction > 40%
Exclusion Criteria:
• Uncontrolled infection at the time of transplant.
• Patients with Fanconi Anemia or other DNA breakage syndromes.
Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Hematopoietic stem cell infusion
Primary Graft Failure, Secondary Graft Failure
Hematopoietic stem cell transplant, Clinics and Surgery Center (CSC)
COG ADVL1823 - Larotrectinib (LOXO-101, NSC# 788607, IND# 141824) for Previously Untreated TRK Fusion Pediatric Solid Tumors and TRK Fusion Relapsed Pediatric Acute Leukemias
This is a non-randomized, open label, phase II trial that will study the side effects and how well larotrectinib works in treating patients (≤ 30 years of age) with previously untreated TRK fusion solid tumors and TRK fusion acute leukemia that has come back. Larotrectinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Emily Greengard
All
up to 30 Years old
Phase 2
NCT03834961
STUDY00007876
Inclusion Criteria:
• COHORT A: Patients must have a histologic diagnosis of infantile fibrosarcoma with an NTRK1, NTRK2, or NTRK3 fusion identified in a Clinical Laboratory Improvement Act/College of American Pathologists (CLIA/CAP) certified laboratory. Fusions may be identified by fluorescence in situ hybridization (FISH) or molecular techniques (reverse transcriptase-polymerase chain reaction [RT-PCR] using primers flanking the fusion junction or next generation sequencing). For fusions identified by FISH, an ETV6 rearrangement is sufficient for eligibility in Cohort A. Identification of the upstream TRK fusion partner is not required.
• COHORT B: Patients must have a histologic diagnosis of any solid tumor other than infantile fibrosarcoma, including central nervous system (CNS) tumors but excluding high grade gliomas. An NTRK1, NTRK2, or NTRK3 fusion must be identified in a CLIA/CAP certified laboratory. Fusions may be identified by FISH or molecular techniques (RT-PCR using primers flanking the fusion junction or next generation sequencing). For fusions identified by FISH, there must be an identified rearrangement in NTRK1, NTRK2, or NTRK3 (e.g., an ETV6 rearrangement is not sufficient for eligibility) unless the patient has a diagnosis of congenital mesoblastic nephroma in which case an ETV6 rearrangement is sufficient for eligibility. Identification of the upstream TRK fusion partner is not required.
• COHORT C: Patients must have a histologic diagnosis of relapsed or refractory acute leukemia with an NTRK1, NTRK2, or NTRK3 fusion identified in a CLIA/CAP certified laboratory. Fusions may be identified by FISH or molecular techniques (RT-PCR using primers flanking the fusion junction or next generation sequencing). For fusions identified by FISH, there must be an identified rearrangement in NTRK1, NTRK2, or NTRK3 (e.g., an ETV6 rearrangement is not sufficient for eligibility). Identification of the upstream TRK fusion partner is not required.
• SOLID TUMORS (COHORTS A AND B): Patients must have measurable disease. Patients must have disease that cannot be completely resected without a predicted functional, neurologic, or significant cosmetic deficit in the opinion of the investigator.
• LEUKEMIA (COHORT C): Patients must have >= 5% blasts in the bone marrow. Extramedullary disease is permitted.
• Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age. NOTE: Neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
• COHORTS A AND B: No prior anti-cancer therapy, including radiotherapy, other than surgical resection is permitted.
• Patients who experience recurrence after surgery alone and no other anti-cancer therapy will be eligible.
• If not eligible due to prior anticancer therapy, patients may be eligible for the larotrectinib arm of Pediatric MATCH (APEC1621A) or treatment with commercial larotrectinib off study.
• COHORT C: Patients with relapsed leukemia (Cohort C) must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately.
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment.
• A waiting period prior to enrollment is not required for patients receiving standard cytotoxic maintenance chemotherapy (i.e., corticosteroid, vincristine, thioguanine [6MP], and/or methotrexate).
• A waiting period is not required for patients receiving a single dose of intrathecal methotrexate, hydrocortisone, and/or cytarabine within 7 days prior to enrollment
• >= 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy. Additionally, patients must have fully recovered from all acute toxic effects of prior therapy.
• Note: Cytoreduction with hydroxyurea must be discontinued >= 24 hours prior to the start of protocol therapy.
• Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil [ANC] counts): >= 7 days after the last dose of agent. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator prior to enrollment.
• Anti-cancer agents that are antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1. There is an exception for blinatumomab infusions, for which patients must have been off for at least 3 days and all drug related toxicity must have resolved to grade 2 or lower as outlined in the inclusion/exclusion criteria.
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid. A waiting period prior to enrollment is not required for patients receiving corticosteroid for leukemia therapy/cytoreduction.
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair and the study-assigned research coordinator.
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors )
• Stem cell infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD).
• Autologous stem cell infusion including boost infusion: >= 42 days.
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial BM radiation.
• Radiopharmaceutical therapy (e.g., radiolabeled antibody): >= 42 days after systemically administered radiopharmaceutical therapy.
• Patients must not have received prior exposure to TRK inhibitors (including larotrectinib, LOXO-195, entrectinib, lorlatinib, crizotinib, or lestaurtinib).
• For patients with solid tumors without known bone marrow involvement: Peripheral absolute neutrophil count (ANC) >= 1000/mm^3 (within 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement: Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement: Hemoglobin >=
• 0 g/dL at baseline (may receive red blood cell [RBC] transfusions).
• Patients with solid tumors with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts above (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity.
• For patients with leukemia: Platelet count >= 20,000/mm^3 (within 7 days prior to enrollment) (may receive platelet transfusions; must not be known to be refractory to red cell or platelet transfusion)
• For patients with leukemia: Hemoglobin >= 8.0 g/dL at baseline (within 7 days prior to enrollment) (may receive RBC transfusions; must not be known to be refractory to red cell or platelet transfusion)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• 1 month to < 6 months (male 0.4 mg/dL, female 0.4 mg/dL)
• 6 months to < 1 year (male 0.5 mg/dL, female 0.5 mg/dL)
• 1 to < 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
• 2 to < 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
• 6 to < 10 years (male 1 mg/dL, female 1 mg/dL)
• 10 to < 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
• 13 to < 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
• >= 16 years (male 1.7 mg/dL, female 1.4 mg/dL)
• For patients < 1 month of age, serum creatinine levels must be < 1.5 x the treating institution's creatinine upper limit of normal (ULN) for patients < 1 month of age or the creatinine clearance or radioisotope GFR must be >= 70 mL/min/1.73 m^2.
• Patients with solid tumors: Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment). After approval of the study chair or designee, infants with a higher total bilirubin due to physiologic or breast milk jaundice are eligible if the conjugated (direct) bilirubin is =< 2 mg/dL
• Patients with solid tumors: Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
• Patients with solid tumors: Serum albumin >= 2 g/dL (within 7 days prior to enrollment).
• Patients with leukemias: Conjugated (direct) bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment).
• Patients with leukemias: SGPT (ALT) =< 225 U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
• Patients with leukemias: Serum albumin >= 2 g/dL (within 7 days prior to enrollment).
• Patients with seizure disorder may be enrolled if on a stable antiepileptic regimen for >= 14 days and well controlled.
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v] 5) except tendon reflex decreased resulting from prior therapy must be =< grade 2.
Exclusion Criteria:
• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, OR because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Female patients of reproductive potential may not participate unless they have agreed to use a highly effective contraceptive method for the duration of study therapy and for at least one month after the final dose of larotrectinib. Males of reproductive potential with a non-pregnant female partner of child-bearing potential must use a highly effective contraception for the duration of the study and for at least one month after the final dose of larotrectinib. Because of the unknown risk of larotrectinib in nursing infants, nursing women should discontinue breastfeeding during treatment with larotrectinib and for 3 days following the final dose.
• Patients with solid tumors, including CNS tumors, requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. Patients with leukemia may receive systemic corticosteroids for cytoreduction up to 24 hours prior to the start of protocol therapy. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid.
• Patients who are currently receiving another investigational drug are not eligible.
• Patients who are currently receiving other anti-cancer agents are not eligible [except leukemia patients receiving corticosteroids or hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy]. Patients with leukemia should receive a single dose of intrathecal cytarabine, hydrocortisone, and/or methotrexate within 7 days prior to Day 1 of Cycle 1 at the time of the baseline lumbar puncture.
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial.
• Patients currently receiving a strong CYP3A4 inducer or inhibitor are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study. Note: CYP3A4 inducing anti-epileptic drugs and dexamethasone for CNS tumors or metastases, on a stable dose, are allowed.
• Patients with malabsorption syndrome or other conditions that significantly limit enteral absorption are not eligible.
• Patients who are unable to swallow capsules or liquid and do not have gastric access via a nasogastric or gastrostomy tube are not eligible.
• Patients who have an uncontrolled infection are not eligible.
• Patients who have received prior solid organ transplantation are not eligible.
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible.
• Patients with high grade gliomas (HGG) are not eligible.
• All patients and/or their parents or legal guardians must sign a written informed consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Drug: Larotrectinib Sulfate
Central Nervous System Neoplasm, Infantile Fibrosarcoma, Recurrent Acute Leukemia, Refractory Acute Leukemia, Solid Neoplasm
Larotrectinib, TRK fusion, NTRK fusion, Infantile fibrosarcoma
TUMOR-AGNOSTIC PRECISION IMMUNOONCOLOGY AND SOMATIC TARGETING RATIONAL FOR YOU (TAPISTRY) PHASE II PLATFORM TRIAL (BO41932)
TAPISTRY is a Phase II, global, multicenter, open-label, multi-cohort study designed to evaluate the safety and efficacy of targeted therapies or immunotherapy as single agents or in rational, specified combinations in patients with unresectable, locally advanced or metastatic solid tumors determined to harbor specific oncogenic genomic alterations or who are TMB-high as identified by a validated NGS assay.
Emil Lou
All
Not specified
Phase 2
NCT04589845
STUDY00010440
Inclusion Criteria:
• Histologically or cytologically confirmed diagnosis of advanced and unresectable or metastatic solid malignancy
• Measurable disease as defined by Response Evaluation Criteria in Solid Tumors, Version
• 1 (RECIST v1.1), Response Assessment in Neuro-Oncology (RANO) criteria, or International Neuroblastoma Response Criteria (INRC)
• Performance status as follows: Participantss aged >= 18 years: Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2; Participantss aged 16 to < 18 years: Karnofsky score >= 50%; Participants aged < 16 years: Lansky score >= 50%
• For participants aged >= 18 and <18 years: adequate hematologic and end-organ function
• Disease progression on prior treatment, or previously untreated disease with no available acceptable treatment
• Adequate recovery from most recent systemic or local treatment for cancer
• Life expectancy >= 8 weeks
• Ability to comply with the study protocol, in the investigator's judgment
• For female participants of childbearing potential: Negative serum pregnancy test <= 14 days prior to initiating study treatment; agreement to remain abstinent or use single or combined contraception methods that result in a failure rate of < 1% per year for the period defined in the cohort-specific inclusion criteria; and agreement to refrain from donating eggs during the same period
• For male participants: Willingness to remain abstinent or use acceptable methods of contraception as defined in the cohort-specific inclusion criteria
• In addition to the general inclusion criteria above, participants must meet all of the cohort-specific inclusion criteria for the respective cohort
Exclusion Criteria:
• Current participation or enrollment in another therapeutic clinical trial
• Any anticancer treatment within 2 weeks or 5 half-lives prior to start of study treatment
• Whole brain radiotherapy within 14 days prior to start of study treatment
• Stereotactic radiosurgery within 7 days prior to start of study treatment
• Pregnant or breastfeeding, or intending to become pregnant during the study
• History of or concurrent serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the participant's safe participation in and completion of the study or confounds the ability to interpret data from the study
• Incomplete recovery from any surgery prior to the start of study treatment that would interfere with the determination of safety or efficacy of study treatment
• Significant cardiovascular disease, such as New York Heart Association cardiac disease (Class II or higher), myocardial infarction, or cerebrovascular accident within 3 months prior to enrollment, unstable arrhythmias, or unstable angina
• History of another active cancer within 5 years prior to screening that may interfere with the determination of safety or efficacy of study treatment with respect to the qualifying solid tumor malignancy
• In addition to the general exclusion criteria above, in order to be enrolled in a treatment cohort of the study, participants must not meet any of the cohort-specific exclusion criteria
Drug: Entrectinib, Drug: Entrectinib, Drug: Alectinib, Drug: Atezolizumab, Drug: Ipatasertib, Drug: Trastuzumab emtansine, Drug: Idasanutlin, Drug: Inavolisib, Drug: Belvarafenib, Drug: Pralsetinib
Solid Tumors
Clinics and Surgery Center (CSC), Phase I Clinic
Phase-dependent Evaluation of Motor Cortex Excitability With TMS in Stroke
Transcranial magnetic stimulation (TMS) is increasingly being explored as a rehabilitation strategy to enhance plasticity in motor regions. Here, we aim to investigate the effects of transcranial magnetic stimulation (TMS) on motor cortex excitability in individuals who have suffered stroke, as well as to study the influence of the phase of the oscillatory rhythm (mu frequency) on motor excitability in stroke individuals.
All
18 Years and over
N/A
NCT04968743
Inclusion Criteria:
• Suffering from chronic stroke, resulting in self-reported motor deficits (stroke occurring more than 6 months before study enrollment)
• Confident level of English language
Exclusion Criteria:
• Metal or electric implant in the head, neck, or chest area
• Upper extremity botulinum toxin treatment in the last 6 months
• Pregnancy or breastfeeding
• Prior occurrence of unprovoked seizure
Device: Transcranial magnetic stimulation (TMS)
Stroke
A RANDOMIZED PHASE II TRIAL OF TRIPLET THERAPY (A PD-L1 INHIBITOR DURVALUMAB (MEDI4736) IN COMBINATION WITH OLAPARIB AND CEDIRANIB) COMPARED TO OLAPARIB AND CEDIRANIB OR DURVALUMAB (MEDI4736) AND CEDIRANIB OR STANDARD OF CARE CHEMOTHERAPY IN WOMEN WITH PLATINUM-RESISTANT RECURRENT EPITHELIAL OVARIAN CANCER, PRIMARY PERITONEAL OR FALLOPIAN CANCER WHO HAVE RECEIVED PRIOR BEVACIZUMAB
Do the combinations of durvalumab (MEDI4736) plus olaparib and cediranib, durvalumab (MEDI4736) and cediranib, or olaparib and cediranib keep patients disease free longer than the usual approach? This study is being done because we want to find out if these drug combinations are better or worse than the usual approach for recurrent, platinum-resistant ovarian, primary peritoneal, or fallopian tube cancer.
Andrea O'Shea
Phase II
NCT04739800
Fallopian Tube Mucinous Adenocarcinoma, Ovarian Seromucinous Carcinoma, Platinum-Refractory Fallopian Tube Carcinoma, Platinum-Refractory Ovarian Carcinoma, Platinum-Refractory Primary Peritoneal Carcinoma, Recurrent Fallopian Tube Clear Cell Adenocarcinoma, Recurrent Fallopian Tube Endometrioid Adenocarcinoma, Recurrent Fallopian Tube Mucinous Adenocarcinoma, Recurrent Fallopian Tube Transitional Cell Carcinoma, Recurrent Fallopian Tube Undifferentiated Carcinoma, Recurrent Low Grade Fallopian Tube Serous Adenocarcinoma, Recurrent Ovarian Clear Cell Adenocarcinoma, Recurrent Ovarian Endometrioid Adenocarcinoma, Recurrent Ovarian Mucinous Adenocarcinoma, Recurrent Ovarian Seromucinous Carcinoma, Recurrent Ovarian Transitional Cell Carcinoma, Recurrent Ovarian Undifferentiated Carcinoma, Recurrent Platinum-Resistant Fallopian Tube Carcinoma, Recurrent Platinum-Resistant Ovarian Carcinoma, Recurrent Platinum-Resistant Primary Peritoneal Carcinoma, Recurrent Primary Peritoneal Clear Cell Adenocarcinoma, Recurrent Primary Peritoneal Endometrioid Adenocarcinoma, Recurrent Primary Peritoneal High Grade Serous Adenocarcinoma, Recurrent Primary Peritoneal Low Grade Serous Adenocarcinoma, Recurrent Primary Peritoneal Transitional Cell Carcinoma, Recurrent Primary Peritoneal Undifferentiated Carcinoma, Refractory Fallopian Tube Clear Cell Adenocarcinoma, Refractory Fallopian Tube Endometrioid Adenocarcinoma, Refractory Fallopian Tube Mucinous Adenocarcinoma, Refractory Fallopian Tube Transitional Cell Carcinoma, Refractory Fallopian Tube Undifferentiated Carcinoma, Refractory Low Grade Fallopian Tube Serous Adenocarcinoma, Refractory Ovarian Clear Cell Adenocarcinoma, Refractory Ovarian Endometrioid Adenocarcinoma, Refractory Ovarian Mucinous Adenocarcinoma, Refractory Ovarian Seromucinous Carcinoma, Refractory Ovarian Transitional Cell Carcinoma, Refractory Ovarian Undifferentiated Carcinoma, Refractory Primary Peritoneal Clear Cell Adenocarcinoma, Refractory Primary Peritoneal Endometrioid Adenocarcinoma, Refractory Primary Peritoneal High Grade Serous Adenocarcinoma, Refractory Primary Peritoneal Low Grade Serous Adenocarcinoma, Refractory Primary Peritoneal Transitional Cell Carcinoma, Refractory Primary Peritoneal Undifferentiated Carcinoma, Recurrent Fallopian Tube High Grade Serous Adenocarcinoma, Recurrent Ovarian High Grade Serous Adenocarcinoma, Recurrent Ovarian Low Grade Serous Adenocarcinoma, Refractory Fallopian Tube High Grade Serous Adenocarcinoma, Refractory Ovarian High Grade Serous Adenocarcinoma, Refractory Ovarian Low Grade Serous Adenocarcinoma
COG ALTE2031 - StepByStep: A Randomized Trial of a Mobile Health and Social Media Physical Activity Intervention among Adolescent and Young Adult Childhood Cancer Survivors
The study objectives are to compare the effect of 2 different physical activity programs on physical activity levels in adolescents and young adults who received and completed treatment for cancer and to measure lab tests associated with heart health and information collected from surveys to learn how changes in physical activity levels affect health and quality of life in participants.
Lucie Turcotte
All
15 Years to 20 Years old
Phase 3
NCT04089358
STUDY00013015
Inclusion Criteria:
• First diagnosis of malignant neoplasm (International Classification of Diseases for Oncology [ICD-O] behavior code of "3") in first and continuous remission at the time of enrollment
• Curative cancer treatment must have included chemotherapy (including cellular therapy) and/or radiation (including radioactive iodine)
• Note: Childrens Oncology Group (COG) therapeutic trial participation is not required
• All cancer treatment must have been completed within 3-36 calendar months prior to enrollment
• Patients must have a life expectancy of > 1 year
• Self-report of < 420 minutes of moderate-to-vigorous physical activity per week as assessed via the study-specific Physical Activity Worksheet
• Note: See COG Study Web Page for the Godin-Shephard Leisure Time Physical Activity Questionnaire or link to online calculator
• Ambulatory and no known medical contraindications to increasing physical activity
• Note: Patients with amputation, rotationplasty, or other prothesis are not automatically excluded as long as they are ambulatory and have no known medical contraindications to increasing physical activity and all other eligibility criteria are satisfied
• No known significant physical or cognitive impairment that would prevent use of the electronic devices used for the protocol intervention (e.g. Fitbit, smartphone, tablet, or computer)
• Able to read and write English
• Note: For patients < 18 years, consenting parent/legal guardian does not have to be able to read and write English
• All patients and/or their parents or legal guardians must sign a written informed consent
• Note: Informed consent may be obtained electronically/online if allowed by local site policy and Institutional Review Board (IRB)/Research Ethics Board (REB) of record
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
• Post-menarchal female patients who are pregnant or planning to become pregnant in the next year are excluded
• Note: Pregnancy status can be established by clinical history with patient. Post-menarchal female patients are eligible as long as they agree to use an effective contraceptive method (including abstinence) during study participation
• Patients with previous hematopoietic stem cell transplant (HSCT) are excluded
• Note: Patients with previous autologous HSCT, chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate as long as all other eligibility criteria are satisfied
Other: Educational Intervention, Device: FitBit, Other: Goal Setting, Other: Health Promotion and Education, Other: Media Intervention, Behavioral: Telephone-Based Intervention
Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm
A Comparison of ACL Repair With BEAR Device vs. Autograft Patellar Tendon ACL Reconstruction (BEAR-MOON)
This study is designed to determine whether anterior cruciate ligament (ACL) repair with the bridge-enhanced ACL repair (BEAR) device is inferior to autograft patellar tendon ACL reconstruction. We hypothesize that the ACL repair with BEAR technology (which does not require harvest of autograft tissue like patellar tendon) will not be inferior to patellar tendon ACL reconstruction in either of the co-primary outcomes. Our co-primary endpoints will be assessed by blinded assessors and include instrumented anterior laxity (KT-1000) and a patient reported outcome measure (IKDC Subjective Score). All patients 18-40 years of age presenting to surgeons in the study who are candidates for ACL surgery (scheduled within 50 days of injury) will be offered participation in the randomized control trial (RCT). Patients will be randomized and will undergo specified rehabilitation protocols post-operatively with primary assessments of KT1000 and IKDC Subjective Score at 6 months, 1 year, and 2 years.
All
18 Years to 40 Years old
N/A
NCT03776162
INCLUSION CRITERIA
In order to be eligible to participate in this study, an individual must meet all of the
following criteria:
• 18-40 years of age
• Complete ACL tear as confirmed by MRI
• Selected surgical treatment of ACL injury
• Believed to be a surgical candidate for ACL reconstruction by treating physician
• Time from injury to surgery is ≤50 days
• Stated willingness to comply with all study procedures for the duration of the study, including lifestyle, activity, and sports restrictions
• Provision of signed and dated informed consent form EXCLUSION CRITERIA AT PRE-OPERATIVE EXAM An individual who meets any of the following criteria, either during a pre-operative exam or during intraoperative arthroscopic evaluation, will be excluded from participation in this study:
• Any prior surgery on affected or unaffected knee
• Confirmed or suspected contralateral ACL tear
• ACL tear found to be only partial and the treating physician feels it does not require surgery because it is "stable"
• Diagnosis of posterolateral corner injury (complete lateral collateral ligament tear, biceps femoris tendon avulsion, arcuate ligament tear, popliteus ligament tear) that requires concurrent or staged surgical treatment
• Diagnosis of Grade 3 medial collateral injury that requires concurrent or staged surgery
• Insufficient ACL tissue on MRI
• Diagnosis of complete patellar dislocation
• Diagnosis of complete patellar tendon or quadriceps tear
• Obesity with a BMI ≥35
• Does not speak or understand English
• History of regular tobacco or nicotine use in any form
• History of drug or alcohol abuse
• Inability to take oral medications
• Use of intra-articular corticosteroids in the affected knee within last 6 months
• Chronic use of oral corticosteroids (e.g., to treat lupus, rheumatoid arthritis, asthma, etc.)
• History of prior infection in knee
• History of chemotherapy treatment
• History of sickle cell disease
• History of anaphylaxis
• Any condition that, in the opinion of the investigator, could affect healing (e.g., diabetes, inflammatory arthritis, etc.)
• Pregnancy or lactation
• Known allergic reactions to meat products or collagen
• Known allergy to bovine collagen, bovine gelatin, or other bovine products
• Known adverse reaction to any bovine product
• Febrile illness within 7 days
• Treatment with another investigational drug or other intervention, either concurrently or previously, that would interfere with surgical healing EXCLUSION CRITERIA AT INTRAOPERATIVE ARTHROSCOPIC EVALUATION
• Time from injury to surgery has exceeded 50 days
• No ACL tear found upon arthroscopic inspection
• ACL tear found to be only partial, with normal pivot shift and endpoint, and does not require surgery
• Displaced bucket handle meniscal injury requiring repair
• Diagnosis of full-thickness chondral injury of Grade 3 or 4, or that requires more than microfracture (i.e., osteochondral autograft transplant), on either condyle
• Unrecognized lateral or medial sided Grade 3 ligamentous injury that requires concurrent or staged surgery
• Tibial stump length is < 1cm
• Tibial footprint attachment is < 50% intact
• Any other reason the ACL stump should be deemed irreparable (e.g., tissue quality too poor to hold suture)
• 18-40 years of age
• Complete ACL tear as confirmed by MRI
• Selected surgical treatment of ACL injury
• Believed to be a surgical candidate for ACL reconstruction by treating physician
• Time from injury to surgery is ≤50 days
• Stated willingness to comply with all study procedures for the duration of the study, including lifestyle, activity, and sports restrictions
• Provision of signed and dated informed consent form EXCLUSION CRITERIA AT PRE-OPERATIVE EXAM An individual who meets any of the following criteria, either during a pre-operative exam or during intraoperative arthroscopic evaluation, will be excluded from participation in this study:
• Any prior surgery on affected or unaffected knee
• Confirmed or suspected contralateral ACL tear
• ACL tear found to be only partial and the treating physician feels it does not require surgery because it is "stable"
• Diagnosis of posterolateral corner injury (complete lateral collateral ligament tear, biceps femoris tendon avulsion, arcuate ligament tear, popliteus ligament tear) that requires concurrent or staged surgical treatment
• Diagnosis of Grade 3 medial collateral injury that requires concurrent or staged surgery
• Insufficient ACL tissue on MRI
• Diagnosis of complete patellar dislocation
• Diagnosis of complete patellar tendon or quadriceps tear
• Obesity with a BMI ≥35
• Does not speak or understand English
• History of regular tobacco or nicotine use in any form
• History of drug or alcohol abuse
• Inability to take oral medications
• Use of intra-articular corticosteroids in the affected knee within last 6 months
• Chronic use of oral corticosteroids (e.g., to treat lupus, rheumatoid arthritis, asthma, etc.)
• History of prior infection in knee
• History of chemotherapy treatment
• History of sickle cell disease
• History of anaphylaxis
• Any condition that, in the opinion of the investigator, could affect healing (e.g., diabetes, inflammatory arthritis, etc.)
• Pregnancy or lactation
• Known allergic reactions to meat products or collagen
• Known allergy to bovine collagen, bovine gelatin, or other bovine products
• Known adverse reaction to any bovine product
• Febrile illness within 7 days
• Treatment with another investigational drug or other intervention, either concurrently or previously, that would interfere with surgical healing EXCLUSION CRITERIA AT INTRAOPERATIVE ARTHROSCOPIC EVALUATION
• Time from injury to surgery has exceeded 50 days
• No ACL tear found upon arthroscopic inspection
• ACL tear found to be only partial, with normal pivot shift and endpoint, and does not require surgery
• Displaced bucket handle meniscal injury requiring repair
• Diagnosis of full-thickness chondral injury of Grade 3 or 4, or that requires more than microfracture (i.e., osteochondral autograft transplant), on either condyle
• Unrecognized lateral or medial sided Grade 3 ligamentous injury that requires concurrent or staged surgery
• Tibial stump length is < 1cm
• Tibial footprint attachment is < 50% intact
• Any other reason the ACL stump should be deemed irreparable (e.g., tissue quality too poor to hold suture)
Procedure: ACL Reconstruction (Bone Patellar Tendon Bone Graft), Device: Bridge Enhanced ACL Repair (BEAR)
Anterior Cruciate Ligament Tear
Anterior Cruciate Ligament Tear, Anterior Cruciate Ligament Reconstruction, Anterior Cruciate Ligament Repair, Knee Surgery
Early identification of right ventricular dysfunction and failure in cardiothoracic and liver surgical patients
This is prospective, non-interventional, observational study. The main purpose of this study is to develop an algorithm for the early detection of RV dysfunction/failure. The algorithm will be based on the RV pressure waveform gradients (the difference between early right ventricular diastolic pressure and end right ventricular diastolic pressure), RV end-diastolic pressure, and RV contractility (dpdt) and validated through associated clinical measures including perioperative mean arterial blood pressure, advanced hemodynamic measures like cardiac output, stroke volume and systemic vascular resistance, perioperative cerebral oximetry (a measure of cerebral perfusion), intraoperative transesophageal echocardiography and clinical outcomes. These associations will be used to train multiple mathematical models to discriminate between patients with and without RV dysfunction/failure as a primary endpoint. Examining the association between the slope of the diastolic right ventricular waveform (RV end-diastolic pressure, and RV contractility) and perioperative hemodynamics, TEE, cerebral saturation and clinical outcomes will allow us to estimate the specificity and sensitivity of our model.
Tjorvi Perry
18 Years and over
NA
ANES-2020-29144
STUDY00010185
Inclusion Criteria:
All patients 18 years or older presenting to the University of Minnesota Medical Center for any cardiac surgery with cardiopulmonary bypass (CPB), single or double lung transplantation or liver transplantation.
Exclusion Criteria:
The following patients will be excluded;
? Patients with a history of internal jugular vein thrombosis or known reasons for not being able to thread a central venous catheter through either internal jugular vein
? Patients with a history of known esophageal strictures, esophageal or stomach cancer, esophageal varices, or any patient in whom a TEE is contraindicated
? Patients with permanent pacemakers whose right ventricle is being paced and not in normal sinus rhythm
? Patients unable to consent to participating in the study
? Patients who are pregnant will be excluded, as part of standard care, all female patients are screened for pregnancy prior to surgery.
? Patients under the age of 18 years old. Heart & Vascular
MT2013-34C: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia: Standard of Care Considerations
Christen Ebens
All
up to 70 Years old
N/A
NCT02162420
1404M50183
Inclusion Criteria:
• Aged 0 - 70 years
• Acceptable hematopoeitic stem cell donor
• Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
• requirement for red blood cell and/or platelet transfusions or
• requirement for G-CSF or GM-CSF or erythropoietin or
• refractory cytopenias having one of the following three
• platelets <50,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• hemoglobin <9g/uL or transfusion dependent
• Diagnosis of DC with a triad of mucocutaneous features:
• oral leukoplakia
• nail dystrophy
• abnormal reticular skin hyperpigmentation, or
• Diagnosis of DC with one of the following:
• short telomeres (under a research study)
• mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)
• mutation in shelterin complex (TINF2)
• mutation in telomere-capping complex (CTC1)
• Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:
• Refractory cytopenia defined by bone marrow cellularity <50% (with < 30% residual hematopoietic cells)
• Diagnosis of SAA with refractory cytopenias having one of the following three:
• platelets <20,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• absolute reticulocyte count <20,000/uL
• Severe Aplastic Anemia (SAA) requiring a 2nd transplant
• Graft failure as defined by blood/marrow chimerism of < 5%
• Early myelodysplastic features
• With or without clonal cytogenetic abnormalities
• Adequate organ function defined as:
• cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure
• pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement
• renal: Glomerular filtration rate (GFR) ≥30% predicted
• Voluntary written consent
Exclusion Criteria:
• Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
• Pregnant or lactating
• Uncontrolled infection
• Prior radiation therapy (applies to SAA patients only)
• Diagnosis of Fanconi anemia based on DEB
• Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with >30% blasts
Drug: Alemtuzumab, Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Stem Cell Transplant, Drug: Anti-thymocyte globulin
Dyskeratosis Congenita, Aplastic Anemia
Clinics and Surgery Center (CSC), severe aplastic anemia, Hematopoietic Stem Cell Transplant
SGLT2 Inhibitors as a Novel Treatment for Pediatric Non-Alcoholic Fatty Liver Disease
This is a pilot drug study to evaluate the feasibility and obtain a preliminary estimate of efficacy and safety of the SGLT2 inhibitor, empagliflozin, in adolescents with obesity (BMI-percentile ≥95th) who have MRI-confirmed NAFLD (hepatic fat fraction ≥ 5.5%) and have normal fasting glucose. We will examine changes in body composition, arterial stiffness, biomarkers of fatty liver disease, and insulin sensitivity over 26-weeks.
Justin Ryder
All
12 Years to 17 Years old
Phase 2
NCT03867487
STUDY00003825
Inclusion Criteria:
For clinical referral to screening visit:
• Age: 12 to <20 years old
• Diagnosis of Obesity: BMI-percentile ≥95th (using age- and sex- based Center for Disease Control definitions) or BMI ≥30 kg/m2
• Elevated alanine aminotransferase (ALT) more than twice the upper limit of normal by gender (≥44 U/L for girls, ≥50 U/L for boys) within 3 months prior to screening (used for historic ALT value) OR diagnosis of NAFLD from ultrasound, MRI, or participants with biopsy-proven NASH within 12 moths of screening
• History of lifestyle modification to treat obesity or NAFLD To be obtained at screening visit:
• Confirmation of Obesity
• Tanner stage 2
• Normal fasting glucose tolerance (fasting blood glucose <100 mg/dL)
• If Screening ALT is used as inclusion criteria [if > 2x historic ALT value (historical value obtained clinically within 12 months of screening visit), repeated after 4 weeks [unable to randomize until completed]]. If the repeat ALT is more than 50% increased or decreased over the screening ALT, a third ALT should be obtained. If a third ALT is not within 50% of the previous value, then the subject is ineligible but may be screened at a later date. If ALT is not used:
• An ultrasound will be done to diagnose NAFLD if the diagnosis has not previously been made by ultrasound, MRI or biopsy
• A MRI-derived HFF ≥ 5.5%
• Willingness to adhere to lifestyle considerations throughout the study
Exclusion Criteria:
• ALT > 250U/L at screening
• History of significant alcohol intake or current use
• Impaired fasting glucose (>100 mg/dL)
• Diabetes (type 1 or 2)
• Current or recent (<6 months prior to enrollment) use of weight loss medication(s)
• Vitamin E supplementation
• Previous bariatric surgery
• Use of metformin
• Prior use of empagliflozin
• Lower limb infection/ulceration within 3 months of screening
• Metal or magnetic implants, devices or objects inside of or on the body, which are not MRI compatible
• Structural and functional urogenital abnormalities, that predispose for urogenital infections
• Recent initiation (<3 months prior to enrollment) of anti-hypertensive or lipid medication(s)
• Major psychiatric disorder
• Current pregnancy or plans to become pregnant.Females unwilling to be tested for pregnancy. Females will be tested for pregnancy. Females who are sexually active and not protected by an effective method of birth control (e.g. UID or medication or patch)
• Tobacco use
• Significant liver dysfunction (levels >5 times the upper limit of normal (ULN)): ALT (ULN = 50 U/L) AST (ULN = 48 U/L) GGT (ULN = 48 U/L) ALP (ULN = 115 U/L)
• Platelets < 150,000 cells/mm3
• Total bilirubin 1.3 mg/dL
• INR 1.3
• Albumin <3.2 g/dL
• Gilbert's Syndrome
• Any known causes of liver disease (except NAFLD and NASH)
• Significant renal dysfunction (estimated glomerular filtration rate [eGFR] < 90 mL/min/1.73 m2),
• Diagnosed monogenic obesity
• History of cancer
• Untreated thyroid disorder
• History of decompensation events (ascites, variceal bleeding, hepatic encephalopathy, or hepatocellular carcinoma)
• Current or recent (<6 months prior to enrollment) use of medication(s) associated with weight gain (e.g. atypical anti-psychotics)
Drug: Empagliflozin 10 MG, Drug: Placebo Oral Tablet
Non-Alcoholic Fatty Liver Disease, NAFLD, Pediatric NAFLD
Artificial Urinary Sphincter Clinical Outcomes (AUSCO)
The study objective is to evaluate the AMS 800 Artificial Urinary Sphincter™ (AUS) in men with primary stress urinary incontinence as measured by pad weight tests. The AMS 800 Artificial Urinary Sphincter is used to treat urinary incontinence due to reduced outlet resistance (intrinsic sphincter deficiency) following prostate surgery. Treatment success defined as ≥ 50% reduction in 24-hour pad weight test from baseline at 12 months post device activation.
Sean Elliott
Male
18 Years and over
NA
NCT04088331
STUDY00008574
Inclusion Criteria:
• Male
• ≥ 18 years of age
• Has undergone either a radical prostatectomy, transurethral resection of the prostate or other invasive prostate surgery
• Demonstrates primary stress urinary incontinence
• Positive screening 24-hour pad weight test (≥100 grams)
• Experiences at least 3 incontinence episodes per day during baseline diary or presents with continuous incontinence
• Negative urine culture
• Willing and able to undergo surgical implantation of the AUS device
• Willing and able to comply with the follow-up requirements
• Willing and able to forego any other surgical urinary incontinence treatments while participating in the study
• Willing and able to sign the informed consent
Exclusion Criteria:
• Previously had or currently has a device implanted (AUS/Sling, or otherwise) for treatment of SUI or urge incontinence
• Primary urgency incontinence
• Postvoid residual volume greater that 150 ml or a history of difficulty emptying the bladder
• Recurrent vesicourethral anastomotic stricture or urethral stricture disease within the past 6 months
• Known urogenital malignancy other than previously treated prostate cancer
• Recurrent prostate cancer that is expected to require intervention during the study follow-up period
• History of recurrent bladder stones within the past 12 months prior to signing the informed consent
• Neurogenic bladder
• Need for intermittent catheterization
• Known history of bleeding diathesis or coagulopathy
• Immunosuppressed or on medical therapy which would impact the immune system
• Uncontrolled diabetes, defined as (HbA1c>10)
• Has a genitourinary mechanical prosthesis that was implanted within 3 months from the date of consent
• Had a post-implantation infection associated with the device after genitourinary mechanical prosthesis was implanted
• Undergone bulking procedure within 6 months of the baseline assessment
• Poor candidate for surgical procedures and/or anesthesia due to physical or mental conditions
• Urinary incontinence due to or complicated by an irreversibly obstructed lower urinary tract
• Irresolvable detrusor hyperreflexia or bladder instability
• Currently enrolled or plans to enroll in another device or drug clinical trial
• Currently using an indwelling catheter or condom catheter for treatment of incontinence and is not willing to discontinue use at least 4 weeks prior to baseline assessment
• Known allergy or sensitivity to rifampin or to minocycline HCl or other tetracyclines (only applicable when implanting with InhibiZone version of this device)
• Systemic lupus erythematosus because minocycline HCl has been reported to aggravate this condition (only applicable when implanting with InhibiZone version of this device)
Device: AMS 800 Artificial Urinary Sphincter
Stress Urinary Incontinence
Clinics and Surgery Center (CSC)
The role of cytomegalovirus and inflammation on patient symptoms and outcomes in ovarian cancer
The central research idea for this award is that CMV reactivation is an unrecognized complicating factor in the treatment of ovarian cancer. Based on preliminary data, our hypotheses are: a) ovarian cancer patients with elevated biomarkers of CMV report greater fatigue and poorer quality of life; b) CMV and inflammation (C-reactive protein; CRP) levels in ovarian cancer patients are higher post-chemotherapy than baseline; c) ovarian cancer patients who experience increases in CMV and/or CRP report greater fatigue and poorer quality of life; d) ovarian cancer patients who experience increases in CMV and CRP will have shorter recurrence-free and overall survival. We propose the following specific aims and studies to address these research questions: Aim 1: Determine the relationship between CMV and ovarian cancer patient-reported symptoms post-treatment. A cross-sectional study of 200 women with ovarian cancer within two years of completing first line chemotherapy will be recruited for a one-time blood draw and survey. We will assess CMV status by measuring IgG serology and CMV DNA levels in serum (indicative of active infection/reactivation) and determine their relationship with patient-reported symptoms including fatigue and quality of life. Aim 2: Characterize the changes in CMV and CRP levels following chemotherapy and their association with patient symptoms and outcomes. A prospective study of 150 women with newly diagnosed ovarian cancer will evaluate the association between serum CMV IgG, CRP IgG, and CMV DNA levels and patient-reported symptoms, recurrence-free and overall survival. Planned blood samples include before, after and 6 months post-chemotherapy completion with planned surveys and clinical follow-up at least every six months for five years.
Rachel Vogel
Female
18 Years and over
NA
NCT03921658
STUDY00005451
Inclusion Criteria:
• Age ≥18
• Ability to read and write in English
• women with newly diagnosed with ovarian, primary peritoneal, or fallopian tube cancer
• Treatment plan includes chemotherapy
• Able to provide written voluntary consent before performance of any study related procedure.
• Cohort 1 only: within 2 years of completing initial chemotherapy treatment
• Cohort 2 only: prior to starting chemotherapy
Exclusion Criteria:
• Inability to provide informed written consent
• Previous exposure to chemotherapy
• Life expectancy < 3 months or in hospice care or nursing home
Ovarian Cancer, Fallopian Tube Cancer, Primary Peritoneal Carcinoma
Clinics and Surgery Center (CSC)
Concurrent Aerobic Exercise and Cognitive Training to Prevent Alzheimer's in At-risk Older Adults (Exergames)
The purpose of this study is to examine the efficacy of a new low-cost Virtual Reality Cognitive Training (VRCT) combined with concurrent cycling intervention called exergame on improving cognition in at-risk community-dwelling older adults at risk for Alzheimer's disease and dementia. We will conduct this study in two phases: Phase I (Feasiblity Testing)aims: Aim1. Develop a prototype exergame that supports integrated, concurrent cycling and VRCT. Aim 2. Examine the feasibility of the exergame in older adults at risk for AD. Phase II (Effect Testing) aims: Aim 1. Develop a fully-featured version of the VRCT aspect of the exergame. Aim 2. Determine the efficacy of the exergame in older adults at risk for AD using an RCT. We hypothesize that cognitive improvement will be greatest for exergame subjects followed by cycling subjects, and least in control subjects. Aim 3. Assess the distraction effect of the concurrent VRCT in exergame on gains in aerobic fitness. We hpothesize that exergame subjects will achieve similar gains in aerobic fitness to cycling only subjects (difference is < 1 standard deviation).
All
65 Years and over
N/A
NCT04311736
Inclusion Criteria:
• Cognitive complaint (defines as answering yes to the question "Do you feel that your memory or thinking skills have gotten worse recently within the last 2 years?");
• Not engaging in aerobic exercise or cognitive training >2 days/week, 30 minutes a session in the past 3 months;
• Age 65 years and older;
• Written consent.
• Medical clearance to participate in a supervised exercise program
Exclusion Criteria:
• Resting heart rate > 100 or <50 beats/min with symptoms;
• Dementia or mild cognitive impairment (self-report, diagnosis, or scoring <26 on the Telephone Interview for Cognitive Status;
• Evidence that cognitive decline or memory complaints were caused by underlying neurological or psychiatric disorder or chemical dependency as determined by primary health care provider;
• Current enrollment in another intervention study;
• ACSM contraindications to exercise or other factors that make exercise impossible or unsafe.
Behavioral: Exergame, Behavioral: Cycling, Behavioral: Stretching
Mild Cognitive Impairment, Exercise Training
mild cognitive impairment, cognitive decline, aerobic exercise, physical exercise, exercise training, cognitive exercise, cognitive games, virtual reality
CHEC-OB-17: CHaractErizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes
Cystic fibrosis (CF) is caused by gene mutations leading to absence or dysfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein which functions as a chloride channel in epithelial cells lining the respiratory tract, gastrointestinal system and sweat glands. Over the past several years, CFTR modulators (small molecule therapies that improve activity of the CFTR protein) have been shown in large clinical trials to improve clinical outcomes in people with CF resulting in the FDA approval of the modulators ivacaftor and lumacaftor/ivacaftor. In clinical trials, SC measurements emerged as an important marker of CFTR activity. The CHEC-SC study is looking at SC levels in people with CF who are currently being treated with CFTR modulator therapies. This study is being done to answer the following questions: • Why do different CF patients have larger or smaller reductions in sweat chloride after treatment with CFTR modulator therapy? • Does the sweat chloride value achieved after treatment with CFTR modulator therapy impact long-term health outcomes in people with CF?
Joanne Billings
NA
NCT03350828
STUDY00001595
Cystic Fibrosis
CF, CFTR Modulator, Clinics and Surgery Center (CSC), Cystic Fibrosis, Sweat, Sweat chloride
HPS-4/TIMI 65/ORION-4: A Double-blind Randomized Placebo-controlled Trial Assessing the Effects of Inclisiran on Clinical Outcomes Among People With Atherosclerotic Cardiovascular Disease
The HPS-4/TIMI 65/ORION-4 study aims to provide evidence about both the efficacy and safety of inclisiran. It is intended to be conducted at approximately 150 clinical sites in Europe (primarily in the UK) and North America. Approximately 15,000 participants aged 55 years or older with pre-existing atherosclerotic cardiovascular disease will be randomized between inclisiran sodium 300 mg and matching placebo (given by subcutaneous injection on the day of randomization, at 3 months and then every 6-months) in a 1:1 ratio for a planned median duration of about 5 years. Consistent with relevant guideline recommendations for people with vascular disease, it is intended that participants be on intensive background LDL-lowering therapy (for example, atorvastatin 40 or 80 mg daily, simvastatin 40 or 80 mg daily, or rosuvastatin 20 or 40 mg daily) at screening. In order to achieve a target LDL cholesterol reduction of at least 1.2 mmol/l [45 mg/dL], it is intended to recruit a study population with a mean LDL cholesterol of at least 2.6 mmol/l [100mg/dL] at baseline.
Les Forgosh
18 Years and over
Phase III
NCT03705234
STUDY00009102
Inclusion Criteria:
History or evidence of at least one of the following:
Prior MI; or
Prior ischemic stroke; or
Peripheral artery disease as evident by prior lower extremity artery revascularization or aortic aneurysm repair.
Exclusion Criteria:
None of the following must be satisfied (based on self-reported medical history):
Acute coronary syndrome or stroke less than 4 weeks before the Screening visit or during the Run-in period;
Coronary revascularization procedure planned within the next 6 months;
Known chronic liver disease;
Current or planned renal dialysis or transplantation;
Previous exposure to inclisiran or participation in a randomized trial of inclisiran;
Previous (within about 3 months), current or planned treatment with a monoclonal antibody targeting PCSK9, or with a drug known to be contra-indicated with inclisiran (none currently known);
Known to be poorly compliant with clinic visits or prescribed medication;
Medical history that might limit the individual's ability to take trial treatments for the duration of the study (e.g. severe respiratory disease; cancer or evidence of spread within approximately the last 5 years, other than non-melanoma skin cancer; or history of alcohol or substance misuse) or may put the individual at significant risk in the opinion of the investigator (or their authorised deputy) if he/she were to participate in the trial;
Women of child-bearing potential, current pregnancy, or lactation;
Current participation in a clinical trial with an unlicensed drug or device; or
Staff personnel directly involved with the study and any family member of the investigational study staff. Atherosclerotic Cardiovascular Disease, Heart & Vascular
Cardiovascular, Cholesterol, Heart Disease, Inclisiran, LDL Cholesterol, Lipid, PCSK9, RNA interference
HYDROXYCHLOROQUINE (HCQ) FOR PREVENTION OF ABNORMAL GLUCOSE TOLERANCE AND DIABETES IN RELATIVES AT-RISK FOR TYPE 1 DIABETES MELLITUS (Protocol TN-22) (TN-22)
The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo-controlled clinical trial. All participants will receive close monitoring for progression of T1D. Participants will receive hydroxychloroquine or placebo and close monitoring for progression to Stage 2 (abnormal glucose tolerance) or Stage 3 (clinically overt) T1D. To assess the efficacy, safety and mode of action of hydroxychloroquine to prevent progression from Stage 1 to Stage 2 or Stage 3 of T1D.
Antoinette Moran
All
3 Years and over
Phase 2
NCT03428945
STUDY00004135
Inclusion Criteria:
• Participant in TrialNet Pathway to Prevention Study (TN01)
• Age 3 years or greater at the time of randomization
• Willing to provide informed consent
• Normal glucose tolerance by OGTT within 7 weeks (no more than 52 days) of baseline
• Two or more diabetes-related autoantibodies present on two separate samples
• Weight of 12 kg or greater at screening
• If a female participant with reproductive potential, willing to avoid pregnancy and undergo pregnancy testing prior to randomization and at each study visit
• Anticipated ability to swallow study medication.
Exclusion Criteria:
• Abnormal Glucose Tolerance or Diabetes
• History of treatment with insulin or other diabetes therapies
• Ongoing use of medications known to influence glucose tolerance
• Ongoing or anticipated future use of medications known to have untoward interactions with hydroxychloroquine
• Known hypersensitivity to 4-aminoquinoline compounds
• G6PD deficiency
• History of retinopathy
• Have an active infection at time of randomization
• Have serologic evidence of current or past HIV, Hepatitis B (positive for Hepatitis B core antibody or surface antigen), or Hepatitis C infection
• Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
• Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
• Be pregnant or breastfeeding.
Drug: Hydroxychloroquine, Drug: Placebo
Type1 Diabetes Mellitus
TrialNet
MT2021-03: A Multicenter Randomized Controlled Trial of Best Available Therapy versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis (BEAT-MS)
Adam Carpenter
All
18 Years to 55 Years old
Phase 3
NCT04047628
STUDY00007288
Inclusion Criteria:
Participant(s) must meet all of the following criteria to be eligible for this study:
• Diagnosis of Multiple Sclerosis (MS) according to the 2017 McDonald Criteria
• (Kurtzke) Expanded Disability Status Scale (EDSS) ≥ 2.0 and ≤ 5.5 at the time of randomization (Day 0)
• T2 abnormalities on brain Magnetic Resonance Imaging (MRI) that fulfill the 2017 McDonald MRI criteria for dissemination in space --A detailed MRI report or MRI images must be available for review by the site neurology investigator.
• Highly active treatment-resistant relapsing MS, defined as ≥ 2 episodes of treatment failure in the 36 months prior to the screening visit (Visit -2). The two treatment failure events need not occur during treatment with different Disease- modifying Therapy (DMT), but must meet all the criteria as described below:
• Each episode of treatment failure must occur following ≥ 3 months of treatment with an FDA-approved DMT for relapsing forms of MS, or with rituximab or ofatumumab, and
• At least one episode of treatment failure must occur with an oral agent or a monoclonal antibody, specifically: dimethyl fumarate (Tecfidera®), diroximel fumarate, teriflunomide (Aubagio®), cladribine (Mavenclad®), daclizumab (Zinbryta®), siponimod (Mayzent®), ozanimod, fingolimod (Gilenya®), rituximab (Rituxan®), ocrelizumab (Ocrevus®), natalizumab (Tysabri®), alemtuzumab (Campath®, Lemtrada®), or ofatumumab (Arzerra®), and
• At least one episode of treatment failure must have occurred within the 12 months prior to the screening visit (Visit -2), and
• At least one episode of treatment failure must be a clinical MS relapse (see item d.i. below). The other episode(s) must occur at least one month before or after the onset of the clinical MS relapse, and must be either another clinical MS relapse or MRI evidence of disease activity (see item d.ii. below): i. Clinical MS relapse must be confirmed by a neurologist's assessment and documented contemporaneously in the medical record. If the clinical MS relapse is not documented in the medical record, it must be approved by the study adjudication committee, and ii. MRI evidence of disease activity must include ≥ 2 unique active lesions on a brain or spinal cord MRI. A detailed MRI report or MRI images must be available for review by the site neurology investigator. A unique active lesion is defined as either of the following:
• A gadolinium-enhancing lesion, or
• A new non-enhancing T2 lesion compared to a reference scan obtained not more than 24 months prior to the screening visit (Visit -2).
• Candidacy for treatment with at least one of the following high efficacy DMTs: Cladribine, natalizumab, alemtuzumab, ocrelizumab, rituximab, and ofatumumab (after approval by the FDA for relapsing MS). --Note: Rituximab, ofatumumab, and ocrelizumab are considered equivalent for candidacy. Candidacy for treatment for each DMT is defined as meeting all of the following:
• No prior treatment failure with the candidate DMT, and
• No contraindication to the candidate DMT, and
• No treatment with the candidate DMT in the 12 months prior to screening.
• Insurance or public funding approval for MS treatment with at least one candidate DMT, and
• Ability to comply with study procedures and provide informed consent, in the opinion of the investigator.
Exclusion Criteria:
Subject(s) who meet any of the following criteria will not be eligible for this study:
• Diagnosis of primary progressive Multiple Sclerosis (MS) according to the 2017 McDonald criteria
• History of neuromyelitis optica or anti-myelin oligodendrocyte glycoprotein (anti-MOG) antibodies associated encephalomyelitis
• Prior treatment with an investigational agent within 3 months or 5 half-lives, whichever is longer
• Either of the following within one month prior to randomization (Day 0):
• Onset of acute MS relapse, or
• Treatment with intravenous methylprednisolone 1000 mg/day for 3 days or equivalent.
• Initiation of natalizumab, alemtuzumab, ocrelizumab, or rituximab between screening visit (Visit -2) and randomization (Day 0)
• Brain MRI or Cerebrospinal fluid (CSF) examination indicating a diagnosis of progressive multifocal leukoencephalopathy (PML)
• History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS)
• Presence of unexplained cytopenia, polycythemia, thrombocythemia or leukocytosis
• History of sickle cell anemia or other hemoglobinopathy
• Evidence of past or current hepatitis B or hepatitis C infection, including treated hepatitis B or hepatitis C -Note: Hepatitis B surface antibody following hepatitis B immunization is not considered to be evidence of past infection.
• Presence or history of mild to severe cirrhosis
• Hepatic disease with the presence of either of the following:
• Total bilirubin ≥ 1.5 times the upper limit of normal (ULN) or total bilirubin
• 3.0 times the ULN in the presence of Gilbert's syndrome, or
• Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥ 2.0 times the ULN.
• Evidence of HIV infection
• Positive QuantiFERON - TB Gold or TB Gold Plus test results (e.g., blood test results that detect infection with Mycobacterium tuberculosis) Note: A Purified Protein Derivative (PPD) tuberculin test may be substituted for QuantiFERON - TB Gold or TB Gold Plus test.
• Active viral, bacterial, endoparasitic, or opportunistic infections
• Active invasive fungal infection
• Hospitalization for treatment of infections or parenteral (IV or IM) antibacterials, antivirals, antifungals, or antiparasitic agents within the 30 days prior to randomization (Day 0) unless clearance is obtained from an Infectious Disease specialist
• Receipt of live or live-attenuated vaccines within 6 weeks of randomization (Day 0)
• Presence or history of clinically significant cardiac disease including:
• Arrhythmia requiring treatment with any antiarrhythmia therapy, with the exception of low dose beta blocker for intermittent premature ventricular contractions
• Coronary artery disease with a documented diagnosis of either:
• Chronic exertional angina, or
• Signs or symptoms of congestive heart failure.
• Evidence of heart valve disease, including any of the following:
• Moderate to severe valve stenosis or insufficiency,
• Symptomatic mitral valve prolapse, or
• Presence of prosthetic mitral or aortic valve.
• Left ventricular ejection fraction (LVEF) < 50%
• Impaired renal function defined as Estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m^2 according to the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula
• Forced expiratory volume in one second (FEV1) <70% predicted (no bronchodilator)
• Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected for Hgb) < 70% predicted
• Poorly controlled diabetes mellitus, defined as HbA1c >8%
• History of malignancy, with the exception of adequately treated localized basal cell or squamous skin cancer, or carcinoma in situ of the cervix. -Note:Malignancies for which the participant is judged to be cured by therapy completed at least 5 years prior to randomization (Day 0) will be considered on an individual basis by the study adjudication committee.
• Presence or history of any moderate to severe rheumatologic autoimmune disease requiring treatment, including but not limited to the following:
• systemic lupus erythematous
• systemic sclerosis
• rheumatoid arthritis
• Sjögren's syndrome
• polymyositis
• dermatomyositis
• mixed connective tissue disease
• polymyalgia rheumatica
• polychondritis
• sarcoidosis
• vasculitis syndromes, or
• unspecified collagen vascular disease.
• Presence of active peptic ulcer disease, defined as endoscopic or radiologic diagnosis of gastric or duodenal ulcer
• Prior history of AHSCT
• Prior history of solid organ transplantation
• Positive pregnancy test or breast-feeding
• Inability or unwillingness to use effective means of birth control
• Failure to willingly accept or comprehend irreversible sterility as a side effect of therapy
• Psychiatric illness, mental deficiency, or cognitive dysfunction severe enough to interfere with compliance or informed consent
• History of hypersensitivity to mouse, rabbit, or Escherichia coli-derived proteins
• Any metallic material or electronic device in the body, or condition that precludes the participant from undergoing MRI with gadolinium administration
• Presence or history of ischemic cerebrovascular disorders, including but not limited to transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral embolism, or cerebral hemorrhage
• Presence or history of other neurological disorders, including but not limited to:
• central nervous system (CNS) or spinal cord tumor
• metabolic or infectious cause of myelopathy
• genetically-inherited progressive CNS disorder
• CNS sarcoidosis, or
• systemic autoimmune disorders potentially causing progressive neurologic disease or affecting ability to perform the study assessments.
• Presence of any medical comorbidity that the investigator determines will significantly increase the risk of treatment mortality, or
• Presence of any other concomitant medical condition that the investigator deems incompatible with trial participation.
Procedure: Autologous Hematopoietic Stem Cell Transplantation, Biological: Best Available Therapy (BAT)
Relapsing Multiple Sclerosis, Relapsing Remitting Multiple Sclerosis, Secondary Progressive Multiple Sclerosis, Rare Diseases
Treatment-Resistant Relapsing Multiple Sclerosis (MS), Autologous Hematopoietic Stem Cell Transplantation (AHSCT), Autologous Peripheral Blood Stem Cells (PBMCs) Graft, Best Available Therapy (BAT), Disease-Modifying Therapy (DMT), BAT DMT, Clinics and Surgery Center (CSC)
Using Smartphone Sensor Technology to Characterize Ambulatory Patterns of Participants With Peripheral Artery Disease
Supervised exercise training (SET) improves functioning and quality of life for patients with peripheral artery disease (PAD). However, these programs have primarily been conducted in research settings, thus the physical activity that patients complete in real-world settings (urban and rural hospitals) is unknown. The proposed project will use a novel smartphone app called Daynamica, to summarize patients activity patterns and also to enhance patient-study staff communication and subsequently improve PAD patient health outcomes.
All
40 Years and over
NCT04124315
Inclusion Criteria:
• Diagnosis of atherosclerotic PAD and referred to hospital-based SET
• Ability to complete an evaluation of physical function and walk on a treadmill
• Resting ankle-brachial index (ABI) of ≤0.90 or stenosis ≥50% in a peripheral vessel or those with lifestyle limiting vascular-related claudication
• Those with a resting ABI of 0.91-0.99 (borderline) who have completed an exercise-ABI assessment with a >20% drop compared to resting values
• Those with ABI >1.40 who have had an abnormal toe-brachial index of ≤0.70
Exclusion Criteria:
• Lower extremity amputation(s) which interfere(s) with walking on the treadmill.
• Individuals with critical limb ischemia defined by ischemic rest pain or ischemic ulcers/gangrene on the lower extremities
• PAD of non-atherosclerotic nature (e.g., fibromuscular dysplasia, irradiation, endofibrosis)
• Females who are pregnant
• Coronary artery bypass grafts or major surgical procedures within 6 months prior to screening
• Individuals whose walking exercise is primarily limited by symptoms of chronic obstructive pulmonary disease, angina, or heart failure
• Individuals who have had a myocardial infarction within 3 months prior to screening
• Individuals who have had a transient ischemic attack or stroke 3 months prior to screening
• Individuals with uncontrolled hypertension (≥180 systolic or ≥100 diastolic resting blood pressure) during screening
• Poorly controlled diabetes defined as glycated hemoglobin >12%
• Abnormal results of blood work not conducive to safely participating in an exercise trial (e.g., anemic, electrolyte abnormalities)
• Inability to speak English
• Other clinically significant cardiovascular, pulmonary, renal, endocrine, hepatic, neurological, psychiatric, immunological, gastrointestinal, hematological, or metabolic disease that is, in the opinion of the study team, not stabilized or may otherwise confound the results of the study
Other: Accelerometry, Other: Daynamica app
Peripheral Artery Disease
peripheral artery disease, accelerometer, smartphone, supervised exercise therapy
MT2020-35 - COG AAML1831 - A Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations
The overall goal of this study is to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, and to find out what effects, good and/or bad, the drug gilteritinib has when given with chemotherapy to children and young adults with newly diagnosed AML and the FLT3/ITD mutation or non-ITD FLT3 activating mutations.
Peter Gordon
All
up to 22 Years old
Phase 3
NCT04293562
STUDY00010751
Inclusion Criteria:
• All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831. Submission of diagnostic specimens must be done according to the Manual of Procedures
• Patients must be less than 22 years of age at the time of study enrollment
• Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
• Patient must have 1 of the following:
• >= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
• In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
• < 20% bone marrow blasts with one or more of the genetic abnormalities associated with childhood/young adult AML as provided in the protocol (sample obtained within 14 days prior to enrollment)
• A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell [WBC] count >= 10,000/uL with >= 10% blasts or a WBC count of >= 5,000/uL with >= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment)
• ARM C: Patient must be >= 2 years of age at the time of Late Callback
• ARM C: Patient must have FLT3/ITD allelic ratio > 0.1 as reported by Molecular Oncology
• ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
• ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• ARM D: Patient must be >= 2 years of age at the time of Late Callback
• ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
• ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
• NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
• NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
• NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
• NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:
• Fanconi anemia
• Shwachman Diamond syndrome
• Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
• Telomere disorders
• Germline predispositions known, or suspected by the treating physician to increase risk of toxicity with AML therapy
• Any concurrent malignancy
• Juvenile myelomonocytic leukemia (JMML)
• Philadelphia chromosome positive AML
• Mixed phenotype acute leukemia
• Acute promyelocytic leukemia
• Acute myeloid leukemia arising from myelodysplasia
• Therapy-related myeloid neoplasms
• Patients with persistent cardiac dysfunction prior to enrollment, defined as ejection fraction (EF) < 50% (preferred method Biplane Simpson's EF) or if EF unavailable, shortening fraction (SF) < 24%. *Note: if clinically safe and feasible, repeat echocardiogram is strongly advised in order to confirm cardiac dysfunction following clinical stabilization, particularly if occurring in the setting of sepsis or other transient physiologic stressor. If the repeat echocardiogram demonstrates an EF >= 50%, the patient is eligible to enroll and may receive an anthracycline-containing Induction regimen
• Administration of prior anti-cancer therapy except as outlined below:
• Hydroxyurea
• All-trans retinoic acid (ATRA)
• Corticosteroids (any route)
• Intrathecal therapy given at diagnosis
• In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
• Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
• Lactating females who plan to breastfeed their infants
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
• ARM D: Patient does not have any congenital long QT syndrome or congenital heart block
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Asparaginase, Drug: Asparaginase Erwinia chrysanthemi, Behavioral: Cogstate Assessment Battery, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Drug: Dexrazoxane Hydrochloride, Drug: Etoposide, Drug: Gemtuzumab Ozogamicin, Drug: Gilteritinib Fumarate, Drug: Liposome-encapsulated Daunorubicin-Cytarabine, Drug: Methotrexate, Drug: Mitoxantrone Hydrochloride, Drug: Therapeutic Hydrocortisone
Acute Myeloid Leukemia
New biomonitoring methodologies to measure DNA adducts in urinary cells
Tobacco smoking and the consumption of well-done cooked meats are considered risk factors for bladder cancer. However, exactly how these exposures contribute to bladder cancer is still mostly unknown. The purpose of this pilot study is to evaluate the potential of urine to serve as a biospecimen for the analysis of cancer biomarkers. Participants will be asked to collect their urine from when they first wake up until 6 hours afterward.
Robert Turesky
NA
2016NTLS094
1605M87561
Inclusion Criteria:
• Daily Smoker
• 21 years of age or older
• In general good health
• Willing to collect urine for 6 hours
Exclusion Criteria:
• Unstable health condition
• Pregnant or nursing
Prevention & Wellness
cigarette, nicotine, smoking, tobacco
Magnesium sulfate as adjuvant analgesia and its effect on opi-ate use of post-operative transplant patients in the pediatric ICU
This study will be a prospective analysis of a post-operative transplant cohort in the PICU to determine whether using magnesium sulfate as an analgesic adjuvant can decrease overall opiate requirement in this patient population. It will indirectly also look at opiate-induced side effects, effects on overall PICU course, and applicability/safety of a magnesium infusion in pediatric patients. It is well known that post-operative analgesia in children is one of many challenges faced by surgeons and intensivists, both due to the invasiveness of procedures as well as the biopsychosocial variance in these populations. TPIAT (total pancreatectomy and islet autotransplantation) and liver transplant patients at our institute have protocols designed for their management, part of which includes continuous opiate dosing, other adjuvants (such as tylenol, ketorolac, ketamine), and sometimes paravertebral nerve blocks. All of these medications, despite their benefits, come with their own unique side effect profile. Opiates remain no stranger to this, in addition to a distinct growing shortage nationwide. Magnesium sulfate has been cited as a potential source of adjuvant analgesia by its action on the NMDA receptor. Pediatric populations where magnesium has shown potential analgesic benefit include post-tonsillectomy, post-osteotomy (cerebral palsy), post-operative scoliosis repair, sickle cell, and hsevere headache management. Literature also supports use in adult populations, which includes more expansive operative cohorts. Added benefit of magnesium is its overall safety profile (symptoms not present until levels significantly above normal indices), cost-effectiveness, and incidental overall prevalence of hypomagnesemia within PICU populations. We plan to implement a magnesium therapy protocol to all of our liver and TPIAT transplant children in the pediatric ICU with dosing that has been used both efficaciously (in comparison to available adult data) and safely (in comparison to other pediatric studies). This will be done via a bolus dose in the operating room followed by infusion dosing for the next 48 hours. Magnesium levels will be checked serially to ensure they remain below toxic levels. We will track opiate dosage metrics throughout their post-operative ICU admission, as well as other secondary outcomes listed elsewhere. The control will be a retrospective chart review of the same primary and secondary outcome measures from previous post-transplant patients in this PICU. The study protocol has been approved by the U.S Food & Drug Administration, which will also be involved in monitoring of the study.
Gwenyth Fischer
All
3 Years to 18 Years old
Phase 2
NCT04812028
STUDY00005974
Inclusion Criteria:
Experimental Group:
• Be scheduled for and receive a liver transplant or total pancreatectomy and islet cell autotransplantation Control Group:
• Received a liver transplant or total pancreatectomy and islet cell autotransplantation.
Exclusion Criteria:
Experimental Group:
• Pregnant or unwilling to abstain from sex if not practicing birth control during participation in the study.
• Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
• Known allergic reactions to components of the MgSO4
• History of heart block or myasthenia graves in past medical history.
• Presence of cardiac pacemaker
• Any patient with preoperative creatinine level > 1.5x upper limit of normal. Control Group:
• Any patient who had filed as research-exempt (opt-out of research previously).
• Any patient with preoperative creatinine level > 1.5x upper limit of normal.
Drug: Magnesium sulfate
Postoperative Pain
MT2019-37: A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Trial of Alpha 1 - Antitrypsin (AAT) Combined with Corticosteroids vs Corticosteroids Alone for the Treatment of High Risk Acute Graft-versus-Host Disease (GVHD) Following Allogeneic Hematopoietic Stem Cell Transplant (CTN 1705)
This study is a phase III, multicenter, double-blinded, randomized, placebo-controlled trial designed to compare AAT and corticosteroids (CS) to placebo and CS as first line therapy for patients with high-risk acute GVHD. The primary objective of this trial is to compare the rate of complete response (CR) and partial response (PR) on Day 28 post-randomization between AAT and CS versus placebo to match (PTM) and CS in patients with high-risk acute GVHD.
Najla El Jurdi
All
18 Years and over
Phase 3
NCT04167514
STUDY00007934
Inclusion Criteria:
• Patients 18 years of age or older
• Initial presentation of acute GVHD after allogeneic hematopoietic cell transplantation for any indication
• Any graft or donor source or conditioning intensity
• Clinical diagnosis of acute GVHD requiring systemic therapy with corticosteroids
Exclusion Criteria:
• Prior exogenous AAT exposure for GVHD prophylaxis
• Relapsed, progressing, or persistent malignancy
• de novo chronic GVHD or overlap syndrome developing before or present at the time of enrollment
• Receiving other drugs for the treatment of GVHD
• Receiving systemic CS for any indication within 7 days before the onset of acute GVHD
Biological: Alpha-1 antitrypsin (AAT), Drug: Placebo
Graft Versus Host Disease (GVHD)
Clinics and Surgery Center (CSC)
Tdcs And cogNitive traininG cOmbined for AUD (TANGO)
The purpose of this study is to test a new treatment for Alcohol Use Disorder that supports long-term abstinence. This new treatment uses transcranial direct current stimulation (tDCS) combined with cognitive training to decrease the chance of relapse. Additionally, we are interested in examining whether specific genetic differences are related to treatment outcomes. For this, we would collect a saliva sample from you. Providing a genetic sample (saliva) is optional and takes about 5 minutes.
All
18 Years to 65 Years old
N/A
NCT05062369
Inclusion Criteria:
• Meet the Diagnostic and Statistical Manual of Mental Disorders diagnostic criteria for AUD
• Abstinent from alcohol use
• Must have the intention to remain in the Lodging Plus program until the end of the intervention portion of the study.
Exclusion Criteria:
• Any medical condition or treatment with neurological sequelae (i.e. stroke, tumor, loss of consciousness>30 min, HIV)
• A head injury resulting in a skull fracture or a loss of consciousness exceeding 30 minutes (i.e., moderate or severe TBI)
• Any contraindications for tDCS or MRI scanning (tDCS contraindication: history of seizures; MRI contraindications; metal implants, pacemakers or any other implanted electrical device, injury with metal, braces, dental implants, non-removable body piercings, pregnancy, breathing or moving disorder)
• Any primary psychotic disorder (e.g. schizophrenia, schizoaffective disorder); Participants with other treated and stable psychiatric disorders will be included
• Presence of a condition that would render study measures difficult or impossible to administer or interpret
• Primary current substance use disorder diagnosis on a substance other than alcohol except for caffeine or nicotine
• Clinical evidence for Wernicke-Korsakoff syndrome
• Left-handedness
• Entrance to the treatment program under a court mandate
Device: TaskFlow Transcranial Electrical Stimulation device, Behavioral: Executive Function Focused Cognitive Training
Alcohol Use Disorder, Addiction
Lifestyle Counseling and Medication for Adolescent Weight Management (QUEST)
This study will compare the effectiveness and durability of intensive behavioral counseling vs. medical management plus low-intensity behavioral counseling on BMI, body fat, cardiometabolic risk factors, and quality of life in adolescents with severe obesity. We hypothesize that liraglutide plus low-intensity behavioral counseling will elicit superior reductions in BMI (primary efficacy endpoint) and body fat and greater improvements in cardiometabolic risk factors and quality of life compared to intensive behavioral counseling at 56 weeks.
Aaron Kelly
All
12 Years to 17 Years old
Phase 2
NCT04873245
STUDY00012932
Inclusion Criteria:
• Severe obesity (Body Mass Index (BMI) >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
• Age 12 to < 18 years old and Tanner stage >1
Exclusion Criteria:
• Diabetes (type 1 or 2)
• Current or recent (< 6 months prior to enrollment) use of anti-obesity medication(s) defined as orlistat, phentermine, topiramate, combination phentermine/topiramate, liraglutide (or other GLP-1RA) and/or combination naltrexone/bupropion (monotherapy use of naltrexone or bupropion is not an exclusion)
• Previous bariatric surgery
• Any history of treatment with growth hormone
• Medically-documented history of bulimia nervosa
• Major psychiatric disorder as determined by the local medical monitor
• Unstable depression requiring hospitalization within the previous 6 month
• Any history of suicide attempt
• History of suicidal ideation or self-harm within the previous 30 days
• Current pregnancy or plans to become pregnant
• ALT or AST >/= 5 times the upper limit of normal
• Creatinine > 1.2 mg/dL
• Uncontrolled hypertension as determined by the local medical monitor
• Diagnosed and medically-documented monogenic obesity
• Medically-documented history of cholelithiasis
• Untreated thyroid disorder
• Medically documented history of pancreatitis
• Personal or family history of medullary thyroid carcinoma or multiple endocrine neoplasia syndrome type 2
• Clinically significant heart disease as determined by the local medical monitor
• Personal history of malignant neoplasms within the past five years
• Hypersensitivity to any component of semaglutide
Behavioral: Intensive Behavioral Program, Drug: Semaglutide and Behavioral Program
Obesity, Childhood
Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications
The purpose of this study is to measure levels of blood and brain chemicals related to oxidative stress and inflammation in healthy volunteers and individuals with Type 1 Gaucher disease (GD1) to see if these levels are altered by GD1. We will also examine if there is a change in these blood and brain chemicals after participants begin taking oral N-acetylcysteine (NAC), which is available both as a prescription medication and a natural product that has antioxidant and anti-inflammatory effects.
James Cloyd
All
18 Years and over
Phase 2
NCT02583672
1506M74581
Inclusion Criteria:
• All participants must be 18 years or older.
• All participants must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
• Individuals with GD1 who are medically stable for participation in the study in the opinion of the investigator.
• GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose (e.g. on a units/kg basis) for at least 2 years.
• GD1 patients who have had a change in therapy, i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
• Healthy subjects who will be frequency-matched for age.
• All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study and during the course of the study.
Exclusion Criteria:
• Medically unstable conditions in any group as determined by the investigators.
• Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
• Women who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception.
• History of asthma that is presently being treated.
• Patients enrolled in another interventional study.
• Allergy to N-acetylcysteine.
• Patients who cannot or are unwilling to have blood drawn.
• Inability to undergo MRI scanning, including but not limited to: unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs.
• Unable to adhere to study protocol for whatever reason.
Drug: N-acetylcysteine
Gaucher Disease Type 1
Gaucher Disease, GD1, N-acetylcysteine, glutathione, GSH
Phase II trial of androgen deprivation therapy (ADT) and pembrolizumab for advanced stage androgen receptor-positive salivary gland carcinoma: Big Ten Cancer Research Consortium BTCRC-HN17-111
This is a Phase II multi-center, single-arm, non-blinded study combining androgen deprivation therapy (ADT) and pembrolizumab for patients with metastatic or locally recurrent androgen receptor-positive salivary gland carcinoma, not amenable to surgery or radiation. The primary objective is to determine the objective response rate (ORR) of pembrolizumab when given with goserelin in patients with locally recurrent or metastatic androgen receptor-positive salivary gland carcinoma not amenable to curative-intent treatment with surgery or radiation per RECIST 1.1.
Manish Patel
All
18 Years and over
Phase 2
NCT03942653
STUDY00004710
Inclusion Criteria:
• Written informed consent and HIPAA authorization for release of personal health information. NOTE: HIPAA authorization may be included in the informed consent or obtained separately.
• Age ≥ 18 years at the time of consent.
• Locally advanced, recurrent, or metastatic salivary gland carcinoma that is not amenable to curative surgery or radiation
• ECOG Performance Status of 0 or 1 within 28 days prior to registration.
• Local, pathologic testing of androgen receptor-positive salivary gland carcinoma will be performed as standard of care. Archival tissue must be available for central confirmation of androgen receptor-positive disease and for correlative studies. AR positivity will be defined according to IHC staining of tumor tissue with at least 20% of tumor staining positive with moderate intensity (1+ or greater).
• Measurable disease according to RECIST v1.1 for solid tumors within 28 days prior to registration.
• For patients who have been treated with prior therapy, patients must have documented progression of disease on their prior therapy for entry into the study.
• Patients with prior chemotherapy, radiation, or surgery as part of curative intent therapy are allowed. Any number of prior lines of systemic therapy is permitted for entry into this study so long as prior therapy did not include anti-androgen therapy or immune checkpoint blockade.
• If prior cancer treatment, the subject must have recovered from toxic effects of prior cancer treatment (other than alopecia) to ≤ Grade 1.
• Adequate organ function as defined below; all screening labs to be obtained within 28 days prior to registration.
• Absolute neutrophil count (ANC) ≥1500/µL
• Platelets ≥75,000/µL
• Hemoglobin ≥8.0 g/dL or ≥5 mmol/L
• Creatinine (Cr) OR Measured or calculated creatinine clearance (GFR can also be used in place of Cr or creatinine clearance) ≤1.5 × ULN OR ≥30 mL/min for participant with creatinine levels >1.5 × institutional ULN
• Total bilirubin ≤1.5 ×ULN OR direct bilirubin ≤ULN for participants with total bilirubin levels >1.5 × ULN
• AST (SGOT) and ALT (SGPT) ≤2.5 × ULN (≤5 × ULN for participants with liver metastases) o International normalized ratio (INR) OR prothrombin time (PT) & aPTT ≤1.5 × ULN unless participant is receiving anticoagulant therapy as long as PT or aPTT is within therapeutic range of intended use of anticoagulants
• A male participant must agree to use contraception during the treatment period and for at least 8 months after the last dose of study treatment and refrain from donating sperm during this period.
• Females of childbearing potential must have a negative serum pregnancy test within 72 hours prior to registration. NOTE: Females are considered of child bearing potential unless they are surgically sterile (have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are naturally postmenopausal for at least 12 consecutive months
• Females of childbearing potential and males with partners of childbearing potential must be willing to abstain from heterosexual activity or to use a highly effect form of contraception from the time of informed consent until 8 months after treatment discontinuation.
• As determined by the enrolling physician or protocol designee, ability of the subject to understand and comply with study procedures for the entire length of the study
Exclusion Criteria:
• Women of childbearing age with a positive serum pregnancy test within 72 hours prior to study registration.
• Has received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (eg, CTLA-4, OX40, CD137).
• Has received prior androgen deprivation therapy including orchiectomy, gonadotropin-releasing hormone (GnRH) agonists/antagonists, androgen receptor blocker, abiraterone, or enzalutamide.
• Has received prior systemic anti-cancer therapy including investigational agents within 14 days prior to registration.
• Has had an allogenic tissue or solid organ transplant.
• Has received prior palliative radiotherapy within 7 days of start of study treatment. Participants must have recovered from all radiation-related toxicities and require less than 10mg of prednisone (or equivalent corticosteroid) daily.
• Has received a live vaccine or live-attenuated vaccine within 28 days prior to the first dose of study drug. Administration of killed vaccines is allowed.
• Has a diagnosis of immunodeficiency or is receiving chronic systemic steroid therapy (in dosing exceeding 10 mg daily of prednisone equivalent) or any other form of immunosuppressive therapy within 7 days prior to the first dose of study drug.
• Has a known additional malignancy that is progressing or has required active treatment within the past 2 years. Note: Participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (e.g. breast ductal carcinoma in situ, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
• Has known active CNS metastases and/or carcinomatous meningitis. Participants with previously treated brain metastases may participate provided they are radiologically stable, i.e. without evidence of progression for at least 14 days by repeat imaging (note that the repeat imaging should be performed during study screening), clinically stable, and without requirement of steroid treatment for at least 14 days prior to first dose of study treatment.
• Has ≥Grade 3 hypersensitivity to pembrolizumab and/or any of its excipients.
• Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg, levothyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
• Has a history of (non-infectious) pneumonitis/interstitial lung disease that required steroids or has current pneumonitis/interstitial lung disease.
• Has an active infection requiring systemic therapy.
• Has a known history of Human Immunodeficiency Virus (HIV).
• Has a known history of active TB (Bacillus Tuberculosis).
• Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
• Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the study, starting with the screening visit through 120 days after the last dose of trial treatment.
Drug: Goserelin Acetate, Drug: Pembrolizumab
Salivary Gland Carcinoma
Clinics and Surgery Center (CSC)
COG APAL2020SC - Pediatric Acute Leukemia (PedAL) Screening Trial Developing New Therapies for Relapsed Leukemias
This study aims to use clinical and biological characteristics of acute leukemias to screen for patient eligibility for available pediatric leukemia sub-trials. Testing bone marrow and blood from patients with leukemia that has come back after treatment or is difficult to treat may provide information about the patient's leukemia that is important when deciding how to best treat it, and may help doctors find better ways to diagnose and treat leukemia in children, adolescents, and young adults (0-<22 years old).
Emily Greengard
All
up to 22 Years old
NA
NCT04726241
STUDY00015549
Inclusion Criteria:
• Patients must be less than 22 years of age at the time of study enrollment
• Patient must have one of the following:
• Patient has known or suspected relapsed/refractory (including primary refractory) AML
• This includes isolated myeloid sarcoma
• Patient has known or suspected relapsed/refractory (including primary refractory) myeloid leukemia of Down syndrome
• Patient has known or suspected relapsed ALL that meets one of the following criteria:
• Second or greater B-ALL medullary relapse, excluding KMT2Ar.
• Any first or greater B-ALL medullary relapse involving KMT2Ar.
• Any first or greater T-ALL medullary relapse with or without KMT2Ar.
• Patient has known or suspected relapsed/refractory (including primary refractory) mixed phenotype acute leukemia
• Patient has known or suspected de novo or relapsed/refractory (including primary refractory) treatment related AML
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Procedure: Biospecimen Collection
Recurrent Acute Lymphoblastic Leukemia, Recurrent Acute Myeloid Leukemia, Recurrent B Acute Lymphoblastic Leukemia, Recurrent Mixed Phenotype Acute Leukemia, Recurrent T Acute Lymphoblastic Leukemia, Refractory Acute Myeloid Leukemia, Refractory Mixed Phenotype Acute Leukemia, Therapy-Related Acute Myeloid Leukemia
Circuit-Based Deep Brain Stimulation for Parkinsons disease; Udall Project 1 Aim 2 and 3
Study objectives: -To characterize spontaneous and movement-related LFP changes in STN and GP in externalized patients under conditions that modulates the severity of tremor, bradykinesia and rigidity (off meds/off stim; on meds/off stim; off meds/on stim, on meds/on stim). -To characterize and compare the relative effect of different forms of closed loop stimulation (e.g., triggered at specific thresholds of low beta/HFO PAC or beta band activity) to standard isochronal high frequency DBS on motor signs and performance during movement.
Michael Park
All
22 Years to 85 Years old
N/A
NCT03079037
1701M04144
Inclusion Criteria:
• Diagnosis of idiopathic PD
• A history of a good response to levodopa (carbidopa/levodopa) defined as at least a 30% improvement in motor UPDRS score
• DBS surgery or IPG battery replacement at UMN is planned as part of routine clinical care.
Exclusion Criteria:
• Other significant neurological disorder
• History of dementia
• Prior history of stereotactic neurosurgery
• Patients with post-operative complications or adverse effects (e.g. ON stimulation dystonias) that affect patient safety or confound the experiment will be excluded from further study
• Pregnant women
Device: Stimulation
Parkinson Disease
Clinics and Surgery Center (CSC)
COG AHEP1531 - Pediatric Hepatic Malignancy International Therapeutic Trial (PHITT)
This partially randomized phase II/III trial studies how well cisplatin and combination chemotherapy works in treating children and young adults (≤ 30 years of age) with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy after surgery may kill more tumor cells.
Emily Greengard
All
up to 30 Years old
Phase 2/Phase 3
NCT03533582
STUDY00003718
Inclusion Criteria:
• Patients in Group F must have a body surface area (BSA) >= 0.6 m^2
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must be newly diagnosed with histologically-proven primary pediatric hepatic malignancies including hepatoblastoma or hepatocellular carcinoma, except as noted below; patients with a diagnosis of hepatocellular neoplasm, not otherwise specified, should be classified and treated per hepatoblastoma treatment arms; note that rapid central pathology review is required in some cases; please note: all patients with histology as assessed by the institutional pathologist consistent with pure small cell undifferentiated (SCU) HB will be required to have testing for INI1/SMARCB1 by immunohistochemistry (IHC) according to the practices at the institution
• Patients with histology consistent with pure SCU must have positive INI1/SMARCB1 staining
• For all Group A patients, WDF status as determined by rapid review will be used to further stratify patients to Group A1 or A2
• For Groups B, C and D, rapid review is required if patients are either >= 8 years of age or have an alphafetoprotein (AFP) =< 100 at diagnosis
• For all Groups E and F patients, rapid central pathology review is required
• In emergency situations when a patient meets all other eligibility criteria and has had baseline required observations, but is too ill to undergo a biopsy safely, the patient may be enrolled without a biopsy
• Clinical situations in which emergent treatment may be indicated include, but are not limited to, the following circumstances:
• Anatomic or mechanical compromise of critical organ function by tumor (e.g., respiratory distress/failure, abdominal compartment syndrome, urinary obstruction, etc.)
• Uncorrectable coagulopathy
• For a patient to maintain eligibility for AHEP1531 when emergent treatment is given, the following must occur:
• The patient must have a clinical diagnosis of hepatoblastoma, including an elevated alphafetoprotein (AFP), and must meet all AHEP1531 eligibility criteria at the time of emergent treatment
• Patient must be enrolled on AHEP1531 prior to initiating protocol therapy; a patient will be ineligible if any chemotherapy is administered prior to AHEP1531 enrollment
• Note: If the patient receives AHEP1531 chemotherapy emergently PRIOR to undergoing a diagnostic biopsy, pathologic review of material obtained in the future during either biopsy or surgical resection must either confirm the diagnosis of hepatoblastoma or not reveal another pathological diagnosis to be included in the analysis of the study aims
• Patients may have had surgical resection of the hepatic malignancy prior to enrollment; all other anti-cancer therapy for the current liver lesion is prohibited
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2 or
• A serum creatinine based on age/gender as follows:
• Age: maximum serum creatinine (mg/dL)
• 1 month to < 6 months: 0.4 (male and female)
• 6 months to < 1 year: 0.5 (male and female)
• 1 to < 2 years: 06 (male and female)
• 2 to < 6 years: 0.8 (male and female)
• 6 to < 10 years: 1 (male and female)
• 10 to < 13 years: 1.2 (male and female)
• 13 to < 16 years: 1.5 (male), 1.4 (female)
• >= 16 years: 1.7 (male), 1.4 (female)
• Total bilirubin =< 5 x upper limit of normal (ULN) for age
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) < 10 x upper limit of normal (ULN) for age
• Shortening fraction of >= 28% by echocardiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment) or
• Ejection fraction of >= 47% by echocardiogram or radionuclide angiogram (for patients on doxorubicin-containing regimens [Groups C, D, E2, and F] assessed within 8 weeks prior to study enrollment)
• Group F patients only: QT/corrected QT (QTc) interval =< 450 milliseconds for males and =< 470 milliseconds for females (assessed within 8 weeks prior to study enrollment)
• Normal pulmonary function tests (including diffusion capacity of the lung for carbon monoxide [DLCO]) if there is clinical indication for determination (e.g. dyspnea at rest, known requirement for supplemental oxygen) (for patients receiving chemotherapy [Groups A, B, C, D, E2, F]); for patients who do not have respiratory symptoms or requirement for supplemental oxygen, pulmonary function tests (PFTs) are NOT required
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
• Prior chemotherapy or tumor directed therapy (i.e. radiation therapy, biologic agents, local therapy (embolization, radiofrequency ablation, and laser); therefore, patients with a pre-disposition syndrome who have a prior malignancy are not eligible
• Patients who are currently receiving another investigational drug
• Patients who are currently receiving other anticancer agents
• Patients with uncontrolled infection
• Patients who previously received a solid organ transplant, other than those who previously received an orthotopic liver transplantation (OLT) as primary treatment of their hepatocellular carcinoma
• Patients with hypersensitivity to any drugs on their expected treatment arm
• Group C: Patients who have known deficiency of dihydropyrimidine dehydrogenase (DPD)
• Group D:
• Patients with chronic inflammatory bowel disease and/or bowel obstruction
• Patients with concomitant use of St. John's wort, which cannot be stopped prior to the start of trial treatment
• Group F:
• Patients with peripheral sensitive neuropathy with functional impairment
• Patients with a personal or family history of congenital long QT syndrome
• These criteria apply ONLY to patients who may receive chemotherapy (all groups other than Group E1):
• Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential
• Lactating females who plan to breastfeed their infants
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
• Note for Group F: patients of childbearing potential should use effective birth control during treatment with sorafenib and for at least 2 weeks after stopping treatment
Drug: Carboplatin, Drug: Cisplatin, Drug: Doxorubicin, Drug: Etoposide, Drug: Fluorouracil, Drug: Gemcitabine, Drug: Irinotecan, Other: Laboratory Biomarker Analysis, Drug: Oxaliplatin, Other: Patient Observation, Drug: Sorafenib, Drug: Vincristine Sulfate
Childhood Hepatocellular Carcinoma, Childhood Malignant Liver Neoplasm, Fibrolamellar Carcinoma, Hepatoblastoma, Hepatocellular Malignant Neoplasm, Not Otherwise Specified
GLNE 007 Evaluation of Stool Based Markers for the Early Detection of Colorectal Cancers and Adenomas
The purpose of this study if to determine if stool or blood can be used to detect colon cancers as early or earlier than colonoscopy. The researchers plan to use these samples to learn about specific proteins (also known as biomarkers) that may indicate colon polyps, colon cancer or an increased risk of developing colon cancer. In order to learn more about preventing and detecting colon and rectal cancer, we are collecting samples from subjects with cancer, adenomas, and colonoscopies who may be at risk for polyps.
Aasma Shaukat
All
18 Years and over
NA
NCT00843375
STUDY0006174
Inclusion Criteria:
• Willing to sign informed consent
• Able to physically tolerate removal of up to 60 ml of blood
• Adults at least 18 years old
• Willing to collect 1-2 stool samples and prepare a Fecal Immunochemical Test (FIT)
• Pregnant or nursing women who otherwise meet the eligibility criteria may participate
• Subjects with one of the following:
• Colorectal adenocarcinoma-not treated and in colon at time of stool collection (CRC bin)
• Adenoma-pathologically confirmed adenoma present in colon at time of stool collection (Adenoma Bin)
• Higher Risk Non-neoplastic Bin
• Subjects with a personal history of adenomas (confirmed by pathology) with none present on qualifying colonoscopy
• Subjects with a personal history of CRC (longer than 3 years ago because of exclusion criteria of cancer within last 3 years) with none present at time of qualifying colonoscopy
• Any family history of CRC (1st degree relative)
• Current positive screening stool test for blood, for DNA or for both within 12 months with no follow-up intervention.
• Average Risk, Non-neoplastic Bin
• No history or current finding of any colorectal neoplasia including CRC, adenomas, sessile serrated adenomas and no family history of CRC.
• Subjects who had CRC that was successfully treated at least three years ago may be considered eligible for the adenoma bin if their polyps are adenomas and there is no evidence of CRC, or for the higher risk non-neoplastic bin as noted above.
• Subjects whose screening colonoscopy shows any of these types of polyps may be included in the non-neoplastic or the higher risk non-neoplastic bin if they meet the other criteria noted above.
• Hyperplastic polyps
• Benign mucosal polyps
• Polypoid granulation tissue
• Prolapsed mucosal polyps
• Inflammatory polyp
• Transitional mucosal polyp
• Lipoma
• Gangleoneuroma
• Neuroma
• Hamartomatous polyp
Exclusion Criteria:
• Cancer patients who have had any surgery, radiation, or chemotherapy for their current colorectal cancer prior to collecting the baseline samples
• History of or clinically active Inflammatory Bowel Disease
• Known HNPCC or FAP
• Inability to provide informed consent.
• Other active malignancy within 3 years of enrollment except any of the following:
• Squamous cell carcinoma of the skin
• Basal cell carcinoma of the skin
• Carcinoma in situ of the cervix, Stages Ia or Ib invasive squamous cell carcinoma of the cervix treated by surgery only. (Excluded if had pelvic radiation)
• Stage Ia Grade 1 adenocarcinoma of the endometrium treated with surgery
• Patients on active chemotherapy or radiation treatment for any purpose
• Known HIV or chronic active viral hepatitis
• Women who are pregnant
• CT colonography (virtual colonoscopy) patients
Colonic Neoplasms