Search Results
MT2021-08: Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults with Hematological Malignancies
The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.
• hematological cancer needing stem cell transplant
• 60 years old or younger
• pregnant or breast feeding
• active infection
• positive for HIV, Hepatitis B or C
• brain metastasis
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease
PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.
• 0 to 24 months of age
• clinical diagnosis on Gaucher disease, Type 2 (GD2)
• Bi-allelic GBA1 mutation
• child has a reliable caregiver (i.e., parent/legal guardian) who is willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities
• diagnosis of a significant CNS disease other than GD2
• able to walk independently
• any other significant medical diagnosis (study staff will review)
• significant laboratory test result abnormalities
• unable to tolerate diagnostic imaging (MRI, CT scan) or unable to tolerate contrast agent
• unable to have sedation or anesthesia
2020IS043; MT2020-06; A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF JSP191 FOR HEMATOPOIETIC CELL TRANSPLANTATION CONDITIONING TO ACHIEVE ENGRAFTMENT AND IMMUNE RECONSTITUTION IN SUBJECTS WITH SCID
This study is looking at whether giving a new type of experimental medicine, called JSP191, can prepare the body to help the stem cell transplant work better, so the immune system can grow and fight infections. The study doctor and Sponsor also want to see how safe and well tolerated this experimental medicine is. They will study whether it is safe to give to patients and look at how much medication to give and what side effects may occur. During this study, the optimal dose of JSP191 will be determined and additional patients will be enrolled in this study using that dose level.
• at least 3 months old
• diagnosis of typical Severe Combined Immunodeficiency (SCID)
• patient with human leukocyte antigen (HLA) matched related or unrelated donors
• acute or uncontrolled infections
• patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• patients with active malignancies
• active Graft-versus-host disease (GVHD) within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD
MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders
COG AALL1731 - A Phase 3 Trial Investigating Blinatumomab (IND# 117467, NSC# 765986) in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients with Localized B-Lymphoblastic Lymphoma (B-LLy)
This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients (365 Days to 31 Years) with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better then combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.
• Age: patients must be > 365 days and < 10 years of age (B-ALL patients without Down Syndrome-DS) OR no more than 31 years of age (B-ALL patients with DS) OR no more than 31 years of age (B-LLy patients with or without DS)
• Diagnosis: Patient has newly diagnosed B-cell ALL, with or without Down syndrome: > 25% blasts on a BM aspirate; OR if a BM aspirate is not obtained or is not diagnostic of B-ALL, the diagnosis can be established by a pathologic diagnosis of B-ALL on a BM biopsy OR a complete blood count (CBC) documenting the presence of at least 1,000 circulating leukemic cells;
• OR Patient has newly diagnosed B-cell LLy Murphy Stages I or II, with or without Down syndrome
• White Blood Cell Count (WBC) Criteria: B-ALL patients without DS must have an initial white blood cell count < 50,000
• B-ALL patients with DS are eligible regardless of the presenting WBC
• Patient must not have secondary ALL that developed after treatment of a prior malignancy with cytotoxic chemotherapy
• Patients must not have received any prior cytotoxic chemotherapy for either the current diagnosis of B-ALL or B-LLy or for any cancer diagnosed prior to initiation of protocol therapy on AALL1731
• Patients requiring radiation at diagnosis
• Female patients who are pregnant or breastfeeding their infants
ONC201 for the Treatment of Newly Diagnosed H3 K27M-mutant Diffuse Glioma Following Completion of Radiotherapy: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Study (ACTION)
The study will test dordaviprone (also known as ONC201), a new drug that is being developed for treating people with glioma. ONC201 may help to remove tumor cells without affecting normal cells of the body. ONC201 is a first-in-class drug, which means it is a new drug having unique action.
• body weight at least 10 kg (22 pounds)
• new diagnosis of H3 K27M-mutant diffuse glioma
• started radiotherapy within 12 weeks from the initial diagnosis
• completed radiotherapy within 2 to 6 weeks prior to starting the study treatment
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• primary spinal tumor
• diffuse intrinsic pontine glioma (DIPG)
• evidence of leptomeningeal spread of disease or cerebrospinal fluid dissemination
• received whole-brain radiotherapy or proton therapy
• study staff will review additional criteria
MT2017-17:T Cell receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in patients with Inherited Bone Marrow Failure (BMF) Disorders
The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.
• up to 65 years of age
• have a diagnosis of Fanconi anemia
• have a suitable donor for peripheral blood cells
• women of childbearing potential and men with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
• see link to clinicaltrials.gov for additional criteria
• women who are pregnant or breastfeeding
• cancer within previous 2 years
A Phase 1/2 Study of the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Relatlimab Plus Nivolumab in Pediatric and Young Adult Participants with Recurrent or Refractory Classical Hodgkin Lymphoma and Non-Hodgkin Lymphoma Protocol Number: CA224069 (RELATIVITY-069)
CA224069 is an open-label, Phase 1/2 clinical trial of relatlimab + nivolumab in children, adolescents and young adults with Recurrent or Refractory Classical Hodgkin Lymphoma (R/R cHL) and Non-Hodgkin Lymphoma (NHL). Part A will encompass safety and dose determination of relatlimab + nivolumab. Part B will be composed of an expansion cohort of cHL (Cohort 1) and an exploratory assessment in NHL (Cohort 2).
• up to 30 years old
• pathologically confirmed high-risk recurrent/relapsed or refractory (R/R) classical Hodgkin lymphoma (cHL), after non-response to or failure of first-line standard therapy prior to a definitive therapy e.g.high-dose chemotherapy/autologous stem cell transplant (HDCT/ASCT)
• participants with pathologically confirmed R/R NHL after failure or non-response to second line therapy, including but not limited to primary mediastinal B-cell lymphoma, diffuse large B-cell lymphoma (DLBCL), mediastinal gray zone lymphoma (MGZL), anaplastic large cell lymphoma (ALCL), or peripheral T-cell lymphoma (PTCL)
• aggressive B-cell lymphomas subtypes including Burkitt lymphoma (BL), lymphoblastic lymphoma, and NK/T-cell lymphoma/leukemia
• prior autologous stem cell transplantation (HDCT/ASCT)
• see link to clinicaltrials.gov for additional exclusion criteria
COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, 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.
• newly diagnosed with a Stage I germ cell tumor or metastatic germ cell tumor
• see link to clinicaltrials.gov for detailed inclusion criteria
• patients must have had no prior systemic therapy for the current cancer diagnosis
• patients must have had no prior radiation therapy (exception of CNS irradiation of brain metastases for standard risk 1 patients)
• female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs
• lactating females who plan to breastfeed their infants
• there are additional exclusion criteria (study staff will review)
JAK Inhibitors to Preserve C-Peptide Production in New Onset T1D: A Phase 2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subtype-Selective JAK Inhibitors for Preservation of Pancreatic Cell Function in Newly Diagnosed Type 1 Diabetes Mellitus
This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.
• age 12-35 years (inclusive)
• diagnosis of T1D within 100 days of first study visit
• positive for at least one islet cell autoantibody
• HbA1c no more than 10 %
• body weight at least 35kg (77 pounds)
• willing to comply with intensive diabetes management and wear a Continuous Glucose Monitoring Device (CGM)
• up to date on recommended immunizations (including flu and COVID-19)
• willing to use highly effective contraception for 3 months after the last dose of study medication
• current use of a medication that affects glucose control
• treatment with other immunosuppressive agents (including biologics or steroids), other than inhaled or topical glucocorticoids
• current history of infection (HIV, Hepatitis B, TB, herpes etc.)
• current or past use of tobacco or nicotine containing products more than the equivalent of 5 cigarettes per day
• current or past medical or mental health conditions (study staff will review)
• women who are pregnant, breast feeding, or planning to become pregnant
A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN). (APPARENT)
To evaluate the efficacy and safety of iptacopan compared to placebo in patients with idiopathic immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN)
• 12 to 60 years old
• diagnosis of idiopathic IC-MPGN as confirmed by kidney biopsy within 12 months prior to starting the study for adults and within 3 years for adolescents
• must have a vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection prior to the start of study treatment
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• have had cell or a solid organ transplantation, including kidney transplantation
• Body mass index (BMI) >38 kg/m2
• Body weight <35 kg (77 pounds)
• additional criteria that study staff will review
MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.
The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease
• must be 2 to 50 years old
• diagnosis of Sickle Cell Disease
• weigh a minimum of 6 kg (13.2 pounds)
• treated and followed for at least the past 24 months
• experienced at least 4 protocol-defined VOEs in the past 24 months
• experienced HU failure at any point in the past or must have intolerance to HU
• female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from starting the study to at least 6 months after drug product infusion.
• if allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available
• unable to receive a transfusion
• prior allogeneic transplant or gene therapy
• prior or current malignancy or immunodeficiency disorder, except cured tumors such as squamous cell carcinoma of the skin
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
SPR001-205 A Phase 2 Study to Evaluate the Safety, Pharmacokinetics,;and Exploratory Pharmacodynamics of SPR001 (Tildacerfont) in Children: Aged 6 to 17 Years with Congenital Adrenal Hyperplasia
The goal of this study is to test the safety and effectiveness of tildacerfont in children with congenital adrenal hyperplasia (CAH). When a child is enrolled in the study, in addition to taking the study drug (tildacerfont), he or she will continue to take his or her standard glucocorticoid doses. A part of the study will be to test different doses of the study drug and to measure adrenal hormones at each visit. Children will be in the study for 18 weeks and will have to visit the study clinic 5 times.
• age 2 to 17 years
• childhood diagnosis of classic congenital adrenal hyperplasia (CAH) a genetic mutation in CYP21A2
• currently taking steroids to treat CAH and on a stable dose for 1 month or more
• clinically significant unstable medical or mental health condition (study staff will review)
• females who are pregnant or nursing
• unable to swallow medications
COG APEC14B1 The Project: Every Child Protocol: A Registry, Eligibility Screening, Biology and Outcome Study Additional Title: EVERYCHILD (APEC14B1) PCR - COG Foundation
This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.
• must be =< 25 years of age at time of original diagnosis, except for patients who are being screened specifically for eligibility onto a COG (or COG participating National Clinical Trials Network [NCTN]) therapeutic study, for which there is a higher upper age limit
• patients with a known or suspected neoplasm that occurs in the pediatric, adolescent or young adult populations
• enrollment must occur within 6 months of initial disease presentation OR within 6 months of refractory disease, disease progression, disease recurrence, second or secondary malignancy
• see link to clinicaltrials.gov for additional inclusion criteria
A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated with Lonapegsomatropin (SkyPASS) (SkyPASS)
The purpose of this study is to evaluate the long-term safety and effectiveness of Skytrofa treatment in children. Patient care will follow the normal treatment practices at the clinic. No additional visits will be performed beyond the usual clinical practice.
• 1 to 18 years old
• on treatment with SKYTROFA (lonapegsomatropin)
• participating in any interventional clinical study for short stature
Projecting Outcomes in Respiratory Technology Dependent Children After Serious Illness (Project ORCAS)
We are hoping to understand more about how being in the ICU impacts children who use breathing support at home and their families. As part of this research study, families will be asked to complete the following during the four study periods: - Complete questionnaires - Airway oscillometry will be used to test your child’s breathing - Complete short daily surveys
• Pediatric ICU admission for at least 24 hours
• Presence of CRTN (any use of supplemental respiratory support, from nasal cannula to around-the-clock mechanical ventilation via a tracheostomy, when the child is at his or her clinical baseline)
• age at least 4 weeks (and at least 40 weeks corrected gestational age) and <17 years at the time of enrollment;
• Presence of at least one parent or legal guardian in the home who would be able to provide information about the child?s daily activities outside the hospital via web- and texting-based interfaces
• Child is enrolled in an interventional study related to provision of pediatric critical care services
• Family does not speak English to an extent to be able to adequately participate in consent discussions or study protocols
A Phase 1/2a Open-Label Dose-Ranging and Observer-Blind Placebo-Controlled, Safety and Immunogenicity Study of mRNA-1647 Cytomegalovirus Vaccine in Female and Male Participants 9 to 15 Years of Age; mRNA-1647-P104
This study it to test an investigational vaccine called mRNA-1647 that is being developed for preventing cytomegalovirus (CMV) infection in people. CMV is a common virus that can spread easily through an infected person’s saliva or other body fluids such as blood, urine, and breast milk. We want see if the trial vaccine can prevent CMV infection in participants who have not been previously infected, to understand the safety (how many side effects you may have) of the trial vaccine, and to see if the trial vaccine results in participants making antibodies to CMV.
• female or male 9 to 15 years of age
• in good general health
• BMI requirements ( study staff will review)
• female participants of childbearing potential: negative pregnancy test and adequate contraception for at least 28 days prior to receiving vaccine through 3 months following vaccine administration
• received, or plans to receive, any nonstudy vaccine less than 28 days prior to or after any study medication
• any diagnosis or condition requiring significant changes in management or medication within the 2 months before starting the study
• contact study staff for review of additional exclusion criteria
Causal Modeling of Ecological Momentary Assessment and Wearable Data in Youth
Researchers want to find out more about how physical, cognitive, and emotional factors affect eating.
• 13-17 years old
• BMI at least at the 95th percentile
• own a smartphone and are willing to wear a Garmin (we provide), download the Garmin Connect App on their smartphone and authorize Garmin to transfer study data study staff
• medication changes in the last 28 days for medications that are likely to affect appetite, mood, and attention
Transcranial Magnetic Stimulation to Augment Exposure and Response Prevention for Pediatric OCD (NExT)
We are doing this study to see if we can improve the standard treatment for OCD, Exposure with Response Prevention, by pairing it with Transcranial Magnetic Stimulation to the parts of the brain that cause OCD symptoms.
• 12 to 21 years old
• right-handed
• currently have OCD symptoms
• inability to have a MRI
• left-handed
• study staff will review additional exclusion criteria
ALX-HPP-501: An Observational,Longitudinal Prospective, Long-term Registry of Patients with Hypophosphatasia
This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.
• confirmed diagnosis of HPP.
• documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
• able to read and speak English
• currently participating in an Alexion-sponsored clinical trial
MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
MT2022-30: A double-blind, randomized, placebo-controlled, interventional, multicenter, phase llI clinical trial to investigate the safety and efficacy of ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs) on epidermolysis bullosa (EB)", allo-APZ2-EB-III
This is a study of an investigational drug (also known as the “study drug”) called allo-APZ2-OTS as a possible treatment for RDEB. The study drug contains allogeneic dermal ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs). These cells suppress inflammation and produce proteins that are important for wound healing. The study drug is expected to counteract local inflammation, improve wound healing and skin strength, and speed up wound closure.
• confirmed diagnosis of RDEB or JEB
• wound meeting the following criteria: 5-50 cm, present for at least 21 days and less than 9 months without signs of acute infection
• women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial
• current cancer diagnosis including squamous cell carcinoma and basal cell carcinoma
• women who are pregnant or breast feeding
• clinically significant or unstable medical or mental health diagnosis (study staff will review)
Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry
The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.
• diagnosed with rheumatic disease prior to age 16 years for juvenile idiopathic arthritis (JIA)
• onset prior to age 19 years for all other rheumatic diseases
• younger than 21 years
Self-Weighing for Weight Management in Adolescents Seeking Obesity Treatment: A Randomized Pilot
This study will test whether adding in daily weighing (5-7 days per week) is helpful in weight loss. In adults, it has been found to be helpful to have individuals who are obese to be more successful with their weight loss efforts if they weigh themselves at home every day. We want to see if this could be helpful in children as well. This research will last for about 12 weeks (3 months).
• aged 12 to <18 years
• BMI at the 95th percentile or greater
• weight loss or maintenance determined as the treatment goal by PWMC provider
• any unhealthy weight control behaviors
• participation in other PWMC study
• developmental delay
• diagnosis that might cause weight fluctuations in weight
• severe anxiety or depression
MAYFLOWERS-0B-20: A Prospective Study Evaluating Maternal and FetaL Outcomes in the ERa of ModulatorS (MAYFLOWERS) (MAYFLOWERS)
This study is being done to look at how pregnancy affects the health of women with cystic fibrosis (CF) and to look at the health of babies born to women with CF. It is expected that most but not all women will be taking the newest CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator medication called TRIKAFTA®. It is not known how this medication will affect the health of mother and infant.
• at least 16 years old
• pregnant, intending to continue pregnancy
• enrolled in the Cystic Fibrosis Foundation Patient Registry (CFFPR)
• none
PEPN2121 : A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that that has either come back (relapsed) or does not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means that tumor cells are missing the SMARCB1 and SMARCA4 genes, which is related to having more aggressive cancers that are harder to treat. Immunotherapy with monoclonal antibodies, such as tiragolumab and atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
• patients must be >= 12 months of age at the time of study enrollment. For part A, patients must be <18 years old at enrollment. For part B, there is no upper age limit
• patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors verified through institutional immunohistochemistry (IHC) or molecular confirmation of a pathologic tumor bi-allelic SMARCB1 (INI1) or SMARCA4 loss or mutation from a Clinical Laboratory Improvement Act (CLIA) certified lab
• see link to clinicaltrials.gov for complete eligibility criteria
• patients who have undergone allogeneic bone marrow or stem cell transplant are not eligible
• patients with known, untreated CNS metastases will be excluded
Uplifting Equitable Park Use and Promoting Physical Activity among African American Families in Minnesota: A Culturally-Responsive, Community-Engaged Approach
This is a study about a special park program built for kids and families who identify as African American or Black, live in the Twin Cities region, and participate in less physical activity than is recommended. This research study takes approximately 4 months, from June to September, with 1 additional visit in May. All activities will happen at French Regional Park, in Plymouth, MN.
• African American parent and child aged 8-12 years old
• live in the Twin Cities metropolitan area
• participating in less than 60 minutes/day and 150 minutes/week of physical activity
• willing to attend weekly activities at French Regional Park in Plymouth, MN, for 12 to 16 weeks
• live with participating parent or caregiver at least half of the time
• able to read and speak English
• currently doing 150 minutes or more physical activity per week for adults or more than 60 minutes per day for children
• unable to be physically active
• currently pregnant or planning to become pregnant or breastfeeding
PEPN2111 - A Phase 1/2 Trial of CBL0137 (NSC# 825802, IND# 155843) in Patients with Relapsed or Refractory Solid Tumors including CNS Tumors and Lymphoma
A Phase I/II trial of single agent intravenous CBL0137 in pediatric patients (≥ 12 months and ≤ 30 years) with relapsed/refractory solid tumors, including CNS tumors and lymphoma.
• 12 months to 30 years old
• patients with relapsed or refractory solid tumors or lymphoma, including patients with CNS tumors or known CNS metastases, or patients with progressive or recurrent DIPG (diagnosed by biopsy or imaging characteristics) and other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy, or patients with relapsed or refractory osteosarcoma
• patients 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
• patients have consented to receive a central venous catheter prior to the administration of CBL0137
• see link to clnicaltrials.gov for complete inclusion and exclusion criteria
• pregnant or breast-feeding women
• patients who have an uncontrolled infection
• patients who have received a prior solid organ transplantation
MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
• patient's parent/ legal guardians are unable to provide informed consent.