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413 Study Matches
MT2017-17: Alpha/Beta T Cell Depleted (alpha/beta TCD) Hematopoietic Cell Transplantation in Patients with Fanconi Anemia
The primary objective is to determine the incidence of grade II-IV acute graft versus host disease (GVHD) by Day 100 using an alpha/beta T cell depleted peripheral blood stem cells (PBSC) and without routine GVHD prophylaxis.
Margaret MacMillan, MD
STUDY00003182
Clinics and Surgery Center (CSC)
Magnetic Resonance Imaging of Osteonecrosis of the Femoral Head
The purpose of this research study is to investigate new magnetic resonance imaging (MRI) methods to better detect and monitor osteonecrosis of the femoral head (ONFH) before and after treatment. ONFH causes injury to the hip joint that can lead to osteoarthritis (the breaking/wearing down of cartilage & tissues within the joint) and the eventual need for a hip replacement. It can be difficult to detect ONFH early on using current medical imaging techniques, which is when treatments may be the most effective. Furthermore, available treatments are not always effective at preventing the progression (spread or growth) of ONFH. This research may benefit others with ONFH by providing more effective medical imaging tools to detect ONFH earlier and inform treatment decisions to increase the chance of stopping or delaying the progression of ONFH and preventing hip osteoarthritis.
Casey Johnson
STUDY00016964
Inclusion Criteria:
• diagnosed with Stage 1 or Stage 2 osteonecrosis of the femoral head (ONFH)
• intend to have core decompression surgery to treat the ONFH
Exclusion Criteria:
• excluded from having an MRI based on Center for Magnetic Resonance Research (CMRR) safety criteria
• existing implantation of metal device in affected hip
• any health conditions that would pose a challenge for you to participate
• unavailable to undergo follow up MRI 6 months after core decompression treatment
Bone, Joint & Muscle
core decompression treatment, MRI, Osteonecrosis, early diagnosis, hip
A Phase 1b, Randomized, Vehicle-Controlled, Double-Blind, Pharmacokinetics, Pharmacodynamics, and Safety Study of ARQ-255 Topical Suspension in Healthy Volunteers and Subjects with Alopecia Areata
This is a phase 1b, vehicle-controlled, double-blind, multicenter study evaluating safety, tolerability, pharmacokinetic (PK), efficacy, and biomarker changes associated with treatment with ARQ-255 topical suspension 3% or vehicle in healthy volunteers and subjects with patchy AA affecting 40-75% of the total scalp.
Maria Hordinsky
STUDY00017979
Inclusion Criteria:
• 18 to 70 years of age
• have alopecia areata
• able to apply topical study medication
Exclusion Criteria:
• alopecia totalis
• alopecia universalis
Dermatology (Skin, Hair & Nails)
alopecia areata
Frailty Assessment by Edmonton Frail Scale to Predict Outcome in Patients Undergoing Cardiovascular Surgery "Frail Heart" Study
We are looking at measures of frailty (including an assessment questionnaire and other data from the medical record) and the relationship to outcomes from cardiac or vascular surgery. The questionnaire will take about 10 minutes to complete and we will contact you by phone once every three months for one year after your surgery.
Tjorvi Perry
STUDY00009831
Inclusion Criteria:
People who are having any of the following heart surgery procedures:
• thoracic aortic surgery
• coronary artery bypass graft surgery
• Aortic valve replacement
• Mitral valve replacement or repair
• Tricuspid valve replacement or repair
• Pulmonary valve replacement
• Infective endocarditis surgery
• Open and interventional abdominal aortic revascularization
Exclusion Criteria:
• People who have liver cirrhosis
• People who aren't able to make independent health care decisions
Heart & Vascular
Cardiac Surgery, Coronary Artery Bypass, Heart Valve Replacement, Abdominal Aortic Surgery
Phase 3 Multicenter, Double-Blind, Placebo-Controlled Trial of Viralym-M;(ALVR105) for the Treatment of Patients With Virus-Associated Hemorrhagic Cystitis After Allogeneic Hematopoietic Cell Transplant
Jo-Anne Young, MD
STUDY00011838
Inclusion Criteria:
• 1 year of age or older
• had an allogeneic hematopoietic cell transplant (HCT) performed at least 21 days and less than one year before entry into the study
• there are specific requirements for absolute neutrophil & platelet count over three days before starting the study
• diagnosed with Hemorrhagic Cystitis (HC)
• study staff will review specific laboratory results that are required
Exclusion Criteria:
• ongoing therapy with high-dose corticosteroids
• treatment with antithymocyte globulin, alemtuzumab (Campath-1H), or other immunosuppressive T cell-targeted monoclonal antibodies ?28 days before starting the study
• evidence of active Grade >2 acute graft versus host disease (GVHD)
• uncontrolled or progressive bacterial or fungal infections
• uncontrolled or progressive viral infections not targeted
• other medical conditions or complications of HCT (study staff will review)
• pregnant, breast feeding or planning to become pregnant.
Infectious Diseases, Kidney, Prostate & Urinary
Clinics and Surgery Center (CSC), HC, Hemorrhagic Cystitis
MT2021-11: An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases
This study is intended to determine the clinical benefit of tabelecleucel (EBV-specific cytotoxic T-lymphocytes) in subjects with EBV-associated diseases.
Joseph Maakaron
STUDY00013494
Inclusion Criteria:
Diagnosis of EBV+ disorder
Eastern Cooperative Oncology Group performance status <= 3 for participants aged >= 16 years; Lansky score >= 20 for participants from >=1 year to < 16 years
Adequate organ function test results, unless organ dysfunction is considered to be due to the underlying EBV-associated disease by the investigator
Cohort-specific Inclusion Criteria:
For participants with PID LPD:
R/R or newly diagnosed PID LPD for whom the standard first-line therapy is inappropriate, as determined by investigator. The LPD is confirmed by at least biopsy-proven EBV+ LPD or positive cerebrospinal fluid (CSF) cytology with or without radiographically measurable intracranial disease with EBV detected in CSF.
Participants with R/R disease must have had at least one prior line of systemic therapy and one of the following: radiographic disease progression per Lugano Classification (Cheson BD, et al. J Clin Oncol. 2014;27:3059) during or after treatment or failure to achieve a CR or partial response (PR) (defined by Lugano radiographic criteria) after standard first-line therapy
Participant may have systemic disease only, systemic and CNS disease, or CNS disease only
For participants with AID LPD:
R/R or newly diagnosed AID LPD for whom the standard first line therapy is inappropriate, as determined by the investigator. The LPD is confirmed by at least biopsy-proven EBV+ LPD or positive CSF cytology, with or without radiographically measurable intracranial disease, with EBV detected in CSF.
Participants with R/R disease must have had at least one prior line of systemic therapy and one of the following: radiographic disease progression per Lugano Classification during or after treatment or failure to achieve a CR or PR (defined by Lugano radiographic criteria) after standard first-line therapy
Participant may have systemic disease only, systemic and CNS disease, or CNS disease only
For participants with AID etiology or AID attributable to immunosenescence, objective laboratory evidence of immunodeficiency
For participants with CNS PTLD:
R/R or newly diagnosed EBV+ CNS PTLD for whom the standard first-line therapy is inappropriate, as determined by the investigator. The CNS PTLD is histologically confirmed by at least biopsy-proven EBV+ CNS PTLD or positive CSF cytology with or without radiographically measurable intracranial disease with EBV detected in CSF.
Participants with R/R disease must have had at least one prior line of systemic therapy and one of the following: radiographic disease progression per Lugano Classification during or after treatment or failure to achieve a CR or PR (defined by Lugano radiographic criteria) after standard first-line therapy
Participant may have systemic and CNS disease or CNS disease only
For participants with EBV+ PTLD, including CD20-negative disease:
Biopsy-proven EBV+ PTLD for whom standard first-line therapy (rituximab and/or chemotherapy) is inappropriate, as determined by the investigator
Participants must have systemic disease measurable per Lugano Classification criteria, except when contraindicated or mandated by local practice, then MRI may be used
For participants with sarcoma, including LMS, or smooth muscle tumors:
EBV+ sarcoma or smooth muscle tumor with rapidly progressive disease defined as progressive disease per RECIST 1.1 criteria as documented radiographically within a 6-month interval prior to enrollment
Participants with newly diagnosed EBV+ sarcoma for whom the standard first-line therapy is inappropriate, as determined by the investigator
Biopsy-proven EBV+ sarcoma meeting one of the criteria's of pathologically confirmed EBV+ Leiomyosarcoma or EBV+ sarcoma or smooth muscle tumor
Measurable disease using diagnostic CT and/or MRI following RECIST 1.1 criteria (Eisenhauer et al. 2009. Eur J Cancer 45[2]:228-247)
Exclusion Criteria:
Currently active Burkitt, T-cell, natural killer/T-cell lymphoma/LPD, Hodgkin, plasmablastic, transformed lymphoma, active hemophagocytic lymphohistiocytosis, or other malignancies requiring systemic therapy
Serious known active infections, defined as ongoing uncontrolled adenovirus infection or infections requiring systemic therapy at the time of enrollment, or known history of human immunodeficiency virus (HIV) infection
Suspected or confirmed Grade >= 2 acute graft-versus-host disease (GvHD) per the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus grading system or extensive chronic GvHD per National Institutes of Health (NIH) consensus criteria at the time of the enrollment
Need for vasopressor or ventilatory support at the time of enrollment
Prior therapy (in order of increasing washout period) prior to enrollment as follows:
Within 4 weeks or 5 half-lives (whichever is shorter) for any investigational product and/ or any chemotherapy (systemic or intrathecal), targeted small molecule therapy, or antibody/biologic therapy. Note: prior anti-CD20 antibody use is permitted within the washout period if a subsequent disease response assessment indicates disease progression
Within 8 weeks: prior tabelecleucel (>8 weeks prior to enrollment) is permitted if response was obtained or if usual protocol-directed therapeutic options were not exhausted, for cellular therapies (chimeric antigen receptor therapies directed at T-cells or T-cell subsets, donor lymphocyte infusion, other CTLs or virus-specific T-cells); and/or therapies which could impact tabelecleucel function (anti-thymocyte globulin, alemtuzumab)
Any prior treatment with EBV-CTLs with the exception of tabelecleucel as above
Women who are breastfeeding or pregnant
Unwilling to comply with protocol specified contraceptive/reproductive restrictions from enrollment through 90 days after the last treatment
Ongoing need for daily steroids of > 0.5 mg/kg prednisone or glucocorticoid equivalent, ongoing methotrexate, or extracorporeal photopheresis (for participants with CNS disease, protocol-specified dexamethasone is permitted and concludes by the time of enrollment)
Any conditions that may put the study outcomes at undue risk (life expectancy < 60 days or any life-threatening illness, medical condition, or organ system dysfunction)
For participants with PID LPD or AID LPD: history of prior allogeneic HCT or solid organ transplant
For participants with EBV+ PTLD: prior systemic therapy for PTLD Clinics and Surgery Center (CSC)
Modifying Progesterone and Estradiol Levels to Prevent Postpartum Cigarette Smoking Relapse and Reduce Secondhand Smoke Exposure in Infants and Children
Sharon Allen, PhD
STUDY00010569
Inclusion Criteria:
• age 18 to 45 years old
• self-reported stable physical and mental health
• self-report uncomplicated pregnancy at gestational week 30 or beyond, or self-report the birth of a child within the past 6 months
• history of 4 or more cigarettes per month during the six months prior to pregnancy
• motivated to remain abstinent after delivery
• willing to protect against pregnancy following day 0 to week 12 of the study
• participants must live in the continental US and have a device to fully participate
Exclusion Criteria:
• current daily use of nicotine replacement therapy or smoking cessation medications, with the exception of e-cigarettes
• current major depressive disorder
• contraindication to progesterone treatment
• current or history of deep vein thrombosis, pulmonary embolus, clotting or bleeding disorder, hypertension, stroke, heart disease, or liver dysfunction or disease; or peanut allergy)
• current or within the past 3 months treatment for illicit drug use or alcohol use
• any condition or issue that, in the opinion of the clinical team, precludes participation in study
Mental Health & Addiction, Women's Health
Smoking, Smoking Cessation
Cardiac Performance System Data Collection Study
This is a non-significant risk device study to evaluate the performance of a new non-invasive device for monitoring of cardiac function (called the Cardiac Performance System, based on a previous iteration called the “Integrated CardioRespiratory System” FDA 510(k) K173156). Measurements of cardiac output, pulmonary artery pressure, and pulmonary capillary wedge pressure from the Cardiac Performance System (CPS) will be compared to those from cardiac catheterization. CPS measurements will take place adjacent to the catheterization lab before entering the lab while the patient is being prepared. This is a relatively low-impact study requiring only a minimal amount of additional time.
Tamas Alexy
STUDY00018102
Inclusion Criteria:
• at least 18 years of age
Exclusion Criteria:
• wounds or surgical incisions where the sensor would be placed
Heart & Vascular
Clinics and Surgery Center (CSC), Cardiac Disease, Heart Disease
Efficacy and safety of GLP-1 agonist therapy in overweight and obese subjects with cystic fibrosis-related diabetes: a pilot study
In this study we will be looking at the safety and effectiveness of the medication GLP-1 (Semaglutide) in patients who are overweight and have been diagnosed with Cystic Fibrosis Related Diabetes (CFRD).
Amir Moheet
STUDY00018575
Inclusion Criteria:
• diagnosis of cystic fibrosis
• diabetic using insulin
• BMI 26 kg/m2 or greater
• able to read & speak English
Exclusion Criteria:
• personal or family history of medullary thyroid cancer
• chronic GI problems requiring hospitalization in the past year
• history of suicide attempts or active ideas of suicide
Rare Diseases, Breathing, Lung & Sleep Health
Cystic Fibrosis, Clinics and Surgery Center (CSC)
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
Margaret MacMillan, MD
STUDY00016450
Inclusion Criteria:
• hematological cancer needing stem cell transplant
• 60 years old or younger
Exclusion Criteria:
• pregnant or breast feeding
• active infection
• positive for HIV, Hepatitis B or C
• brain metastasis
Cancer
Blood Cancers, Hematologic Malignancy, Leukemia, Stem Cell Transplant, Clinics and Surgery Center (CSC)
KIN-1902-2001: A Randomized, Double-blind, Placebo-controlled Phase 2 Study with Open-label Extension to Assess the Efficacy and Safety of Namilumab in Subjects with Chronic Pulmonary Sarcoidosis (RESOLVE-Lung)
We are studying a new medication, namilumab, given to treat Chronic Pulmonary Sarcoidosis. We are looking at the effectiveness, how well the drug is tolerated, and the side effects that occur. For the first part of the study, some people will receive the namilumab; the others will receive an inactive (placebo) drug. In the second part of the study, everyone may receive the namilumab.
Maneesh Bhargava
STUDY00014721
Inclusion Criteria:
• Diagnosis of pulmonary sarcoidosis with some respiratory symptoms
• If receiving prednisone, must be on a stable dose of 25 mg or less for 4 weeks
• If receiving immunosuppressive therapy, must agree to stop if eligible to participate in the study
• Completion of primary series of COVID-19 vaccination
Exclusion Criteria:
• Pregnancy or breast-feeding
• Smoking or using any form of inhaled tobacco or cannabis within 6 months
Rare Diseases, Respiratory System
Clinics and Surgery Center (CSC), Sarcoidosis
Chilled Platelet Study (CHIPS)
The purpose of this research study is to compare the transfusion of cold-stored (refrigerated) platelets to standard room temperature stored platelets. The goal of the trial is to determine whether platelets stored cold are similar or better at stopping bleeding compared to platelets stored at room temperature and, if so, to determine the maximum duration of cold storage that maintains a similar effect on bleeding
Claudia Cohn
STUDY00013172
Inclusion Criteria:
Age greater than 28 days and less than 85 years
Planned complex cardiac surgery with planned use of cardiopulmonary bypass
Exclusion Criteria:
Expected order for washed or volume reduced platelets
Patient with known anti-platelet antibodies
Platelet transfusion refractoriness due to anti-HLA antibodies
Known or suspected pregnancy
Previously randomized in this study
Conscious objection or unwillingness to receive blood products
Known IgA deficiency
Known congenital platelet disorder
Known congenital bleeding disorder
Planned post-operative extracorporeal membrane oxygenation (ECMO), ventricular assist device (VAD), and/or continuous renal replacement therapy (CRRT)/ hemodialysis
Patients intended to receive whole blood either intra-operative or post-operative for bleeding
Platelet transfusion (of any type) within 24 hours prior to the date of surgery
Pre-operative thrombocytopenia, defined as platelet count <75x10(9)/L, based on the most recent labs completed within 72 hours prior to the date of surgery.A feasibility study of topical cannabinoids for treatment of aromatase inhibitor-associated musculoskeletal syndrome (AIMSS) in women with hormone receptor-positive breast cancer (CanAroma)
Aromatase inhibitors (AIs) are commonly used in treating hormone-positive breast cancer. Unfortunately, many patients receiving this treatment experience Aromatase Inhibitor-Associated Musculoskeletal Syndrome (AIMSS), with symptoms like joint and bone pain and joint stiffness. The current therapies used to improve AIMSS symptoms have limited effectiveness and can cause their own side effects. In this research study, we are examining the feasibility of topical medical cannabis cream as a treatment option for AIMSS.
Anne Blaes, MD
STUDY00015727
Inclusion Criteria:
• Stage I-III Breast Cancer.
• Currently taking Aromatase Inhibitor for at least 60 days.
• Aromatase Inhibitor use for no more than 48 months.
• Experiencing aromatase inhibitor-associated musculoskeletal syndrome (AIMSS) symptoms.
• Minnesota resident
Exclusion Criteria:
• Currently using or has used cannabinoid products in the past 4 weeks.
• Active skin lesions on hands/wrists.
• Current or planned acupuncture treatments to hands and wrists.
• Plan to increase doses of other pain medications for improving AIMSS symptoms.
Cancer
aromatase inhibitors, Breast Cancer, hand, pain, stiffness, wrist, Cancer Survivors
Role of Pharmacotherapy in Counteracting Weight Regain in Adolescents with Severe Obesity
In this study we want to find out more about weight loss and how diet and medications can affect weight loss. This study will last for up to 58 weeks. There are two phases to the study: - A weight loss phase with prescribe meals that lasts 6 weeks. - A study medication/placebo phase that lasts up 52 weeks. You will not know if you are receiving the medication or the placebo.
Aaron Kelly
STUDY00008743
Inclusion Criteria:
• severe obesity (BMI >/= 120% of the 95th percentile or BMI >/= 35 kg/m2)
• 12 to less than 18 years of age at enrollment
• female participants who are sexually active with males and who are able to get pregnant must agree to use two forms of contraception throughout the trial
Exclusion Criteria:
• diabetes (type 1 or 2)
• current or recent (< six months prior to enrollment) use of anti-obesity medication(s) (use of naltrexone or bupropion alone is not an exclusion)
• previous metabolic/bariatric surgery
• current use of a stimulant medication
• history of glaucoma
• current or recent (<14 days) use of monoamine oxidase inhibitor
• history of treatment with growth hormone
• history of bulimia nervosa
• major psychiatric disorder
• any history of active suicide attempt
• history of suicidal ideation or self-harm within the previous 30 days
• current pregnancy or plans to become pregnant during study participation
• current tobacco use
• history of cardiac, endocrine, kidney disease (study staff will review)
Children's Health, Diabetes & Endocrine
Clinics and Surgery Center (CSC), Obesity, overweight, weight loss
A Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Efficacy, Safety and Pharmacokinetics of Voclosporin in Adolescents with Lupus Nephritis (VOCAL)
The aim of this study is to investigate whether voclosporin, added to standard treatment, is able to reduce activity of lupus nephritis over a study treatment period of 24 weeks, and to determine its safety as well as the best dose for treatment of lupus nephritis in children or adolescents.
Michelle Rheault
STUDY00018537
Inclusion Criteria:
• 12 to 17 years old
• diagnosis of systemic lupus erythematosus (SLE)
• active lupus nephritis confirmed by a kidney biopsy
Exclusion Criteria:
• currently need dialysis
• clinically significant active medical or mental health conditions (study staff will review)
• certain medications, including: immunosuppression biologic agents, cyclophosphamide, calcineurin inhibitors (CNIs), start or change dose of ACE inhibitors/ARBs within 4 weeks prior to starting study, IV corticosteroids and IV immunoglobulin within 2 weeks of starting study
Immune Diseases, Kidney, Prostate & Urinary
Adolescent Lupus Nephritis, Lupus, Nephritis
Prospective, Multicenter, Single-Arm Study of VanquishTM Water Vapor Ablation for PrOstate CanceR (VAPOR 2)
The purpose of this study is to evaluate the safety and effectiveness of the Vanquish Water Vapor Ablation Device (“Vanquish”) in subjects with Gleason Grade Group 2 (GGG2) localized intermediate-risk prostate cancer.
Christopher Warlick, MD
STUDY00019145
Inclusion Criteria:
• 50 years or older
• PSA no more than 15 ng/ml
• cancer stage less than or equal to T2c
• had a multiparametric MRI within the last 12 months and MRI software guided fusion biopsy of the prostate within the last 6 months
Exclusion Criteria:
• prior surgery, intervention, or minimally invasive therapy, for the prostate cancer or bladder neck
• taking medications that have hormonal effects on the prostate or PSA or or testosterone supplement
• significant medical or mental health diagnosis (study staff will review)
Cancer, Kidney, Prostate & Urinary
Clinics and Surgery Center (CSC), Prostate Cancer, Prostate Cancer
20-0001: A Phase 1b/2, Multi-Centered, Randomized, Double-Blind, Placebo-Controlled Trial of the Safety and Microbiological Activity of a Single Dose of Bacteriophage Therapy in Cystic Fibrosis Subjects Colonized with Pseudomonas aeruginosa
We are looking at a new intravenous drug, Bacteriophage, to treat Pseudomonas Aeruginosa in people at least 18 years of age who have cystic fibrosis. The drug is given one time and different doses will be evaluated to see if they work and to look at side effects.
Joanne Billings
STUDY00013957
Inclusion Criteria:
• Cystic Fibrosis (CF) diagnosis based on clinical symptoms and confirmed by either an abnormal sweat chloride test or CFTR gene variations
• P. aeruginosa isolated from a sputum, throat culture, or other respiratory specimen in the past 12 months.
Exclusion Criteria:
• body weight less than 30 kg
• Forced Expiratory Volume 1 second less than 20% of predicted value
• women who are pregnant, planning to become pregnant during the study, or breastfeeding
• anticipate change chronic antibiotic regimens during the study
Respiratory System, Rare Diseases
CF, Cystic Fibrosis, P. aeruginosa, Pseudomonas Aeruginosa
A Phase 3 Randomized Controlled Trial of Post-Surgical Stereotactic Radiotherapy (SRT) versus Surgically Targeted Radiation Therapy (STaRT) with Gamma Tile for Treatment of Newly Diagnosed Metastatic Brain Tumors.
Clark Chen
STUDY00012456
Inclusion Criteria:
Patients aged 18 years old and above. Eligibility is restricted to this age group given that the battery of neurocognitive tests utilized in this protocol are not developed or validated for use in a younger population.
One to four newly diagnosed brain metastases, identified on the screening MRI, from an extracranial primary tumor.
One lesion, designated the index lesion, is planned for surgical resection and is to be between 2.5 cm and 5.0 cm on the screening MRI. Index lesions > 2.0 cm but <2.5 cm are also eligible if surgery is deemed clinically necessary and appropriate for an attempted gross total resection by the neurosurgeon.
Non-index lesions must measure < 4.0 cm in maximal extent on the screening MRI brain scan. The unresected lesions will be treated with SRT as outlined in the treatment section of the concept.
All metastases must be located > 5 mm from the optic chiasm and outside the brainstem. Dural based metastasis are eligible.
Previous and/or concurrent treatment with systemic therapies (e.g., chemotherapy, targeted therapeutics, immunotherapy) is permitted and must follow protocol guidelines as follows: Systemic therapy is allowed a minimum of one week from last systemic therapy cycle to surgical resection, and one week after surgical resection to allow a minimum of one week before starting/resuming systemic therapy, depending on the specific systemic agent(s), as recommended by medical/neuro-oncology. Systemic therapy is not allowed 1 day before SRT, the same day as the SRT, or 1 day after the completion of the SRT or longer, depending on the specific systemic agent(s), as recommended by medical/neuro-oncology. Agents that are delivered by implant or depot injections (such as hormonal therapies) are excluded from these restrictions.
KPS score of ≥70.
Stable systemic disease or reasonable systemic treatment options predicting a life expectancy of ≥6 months.
Ability to complete an MRI of the head with contrast
Adequate renal and hepatic function to undergo surgery, in investigators opinion.
For women of childbearing potential only, a negative urine or serum pregnancy test done < 7 days prior to randomization is required. Women must be willing to notify investigator immediately if they become pregnant at any time during the trial period.
Men and women of childbearing potential must be willing to employ adequate contraception throughout the study and for men for up to 3 months after completing treatment.
Subjects must be fluent in English or Spanish language. English speaking subjects will complete Neurocognitive assessments. Non-English speaking subjects will not complete the Neurocognitive assessments as the psychometric properties for translated tests are either not known or not as robust.
Willingness and ability to provide written informed consent and HIPAA authorization prior to performance of any study-related procedures. A legally authorized representative may provide consent if the potential subject lacks the capacity to provide consent themselves.
Exclusion Criteria
Age <18 years.
KPS<70
Past radiation or surgical therapy to the index lesion or the newly diagnosed non-index lesion(s) is exclusionary. However, up to a total of 2 prior courses of SRT treatment to previously diagnosed lesions are allowed as long as any treated lesions are were >15mm from the index lesion.
Patients with >4 newly diagnosed metastases on screening MRI
Pregnant patients.
Primary germ cell tumor, small cell carcinoma, or lymphoma.
Leptomeningeal metastasis (LMD). Note: For the purposes of exclusion, LMD is a clinical diagnosis, defined as radiologic or clinical evidence of leptomeningeal involvement with or without positive cerebrospinal fluid (CSF) cytology.
Prior WBRT for brain metastases.
Concomitant therapy that, in the investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study device.
Comorbid psychiatric or neurologic disease or injury impacting cognition, in the opinion of the treating physician, that might impair patient's ability to understand or comply with the requirements of the study or to provide consent
Subjects who, in the investigator's opinion, are unable to understand the protocol or to give informed consent, have a history of poor cooperation, noncompliance with medical treatment, or difficulty in returning for follow up care. Clinics and Surgery Center (CSC)
A Phase III, adjudicator-blinded, randomised study to evaluate the efficacy and safety of treatment with olorofim versus treatment with AmBisome? followed by standard of care (SOC) in patients with invasive fungal disease (IFD) caused by Aspergillus species (OASIS)
This is a Phase III, randomised, adjudicator-blinded, multicentre, active comparator, parallel, two-arm study to evaluate the efficacy and safety of treatment with olorofim versus treatment with AmBisome® followed by SOC in ITT patients with proven IA at any site or probable lower respiratory tract disease (LRTD) IA where: (i) Therapy with a mould-active azole is inappropriate OR a non-azole antifungal is required, AND (ii) For whom the site Investigator agrees that therapy with AmBisome® at 3 mg/kg/day followed by SOC would be an appropriate treatment. Approximately 225 patients (150 to receive olorofim and 75 to receive AmBisome®) will be enrolled at approximately 100 centres globally over a period of approximately 30 months. The study is comprised of a screening period, a treatment period of up to 84 days (± 7 days), and a safety follow-up (FU) period of 4 weeks ± 7 days. The maximum duration of the study for any individual patient is approximately 18 weeks
Jo-Anne Young, MD
STUDY00019092
Inclusion Criteria:
• over 18 years old
• weigh more than 40 kg (88 pounds)
• Invasive Aspergillosis (IA) at any site
• require therapy with an antifungal agent other than a mold-active azole
Exclusion Criteria:
• women who are pregnant or breastfeeding
• known history of allergy, hypersensitivity, or any serious reaction to any component of the study drug
• people with chronic aspergillosis, aspergilloma, or allergic bronchopulmonary aspergillosis
• human immunodeficiency virus (HIV) infection but not currently receiving antiretroviral therapy
• certain heart and liver conditions (study staff will review)
Infectious Diseases, Respiratory System
Aspergillosis, IA, Invasive Aspergillosis (IA), Lung Infection
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.
Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).
Ashish Gupta
STUDY00006923
Inclusion Criteria:
Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype.
Be ≥2 and ≤50 years of age at time of consent.
Weigh a minimum of 6 kg.
Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age).
Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent.
Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
Exclusion Criteria:
Subjects for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available.
Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for subjects ≤ 16 years of age (e.g. TCD velocity >200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis.
Clinically significant, active bacterial, viral, fungal, or parasitic infection
Advanced liver disease, such as
clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
Inadequate bone marrow function, as defined by an absolute neutrophil count of <1×10^9/L (<0.5×10^9/L for subjects on hydroxyurea treatment) or a platelet count <100×10^9/L.
Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
Unable to receive pRBC transfusion.
Prior receipt of an allogeneic transplant.
Prior receipt of gene therapy.
Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
Immediate family member with a known or suspected Familial Cancer Syndrome.
Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion.
Any other condition that would render the subject ineligible for HSCT.
Participation in another clinical study with an investigational drug within 30 days of screening.
Presence of a chromosomal abnormality or genetic mutation that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
Presence of genetic mutations that result in the inactivation of 2 or more α-globin genesA Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors; Protocol Number: LOXO-RET-18036 (J2G-OX-JZJJ) (LIBRETTO-121)
This is an open-label, multi-center, Phase 1/2 study of oral LOXO-292 in pediatric patients with an activating RET alteration and an advanced solid or primary CNS tumor.
Emily Greengard
STUDY00008874
Inclusion Criteria:
Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies
Evidence of an activating RET gene alteration in the tumor and/or blood
Measurable or non-measurable disease
Karnofsky (participants 16 years and older) or Lansky (participants younger than 16) performance score of at least 50
Participant with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior and must not have required increasing doses of steroids within the last 7 days
Adequate hematologic, hepatic and renal function.
Ability to receive study drug therapy orally or via gastric access
Willingness of men and women of reproductive potential to observe conventional and effective birth control
Exclusion Criteria:
Major surgery within two weeks prior to planned start of LOXO-292
Clinically significant, uncontrolled cardiac, cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292
Active uncontrolled systemic bacterial, viral, fungal or parasitic infection
Clinically significant active malabsorption syndrome
Pregnancy or lactation
Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant required a modification to current thyroid medication in the 7 days before start of LOXO-292)
Uncontrolled symptomatic hypercalcemia or hypocalcemia
Known hypersensitivity to any of the components of the investigational agent, LOXO-292 or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension
Prior treatment with a selective RET inhibitor(s) (including investigational selective RET inhibitor[s])A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 21 (LGMD21) (Fortify)
This study will use BBP-418 study drug in patients with LGMD to assess the clinical biomarkers, efficacy and safety of BBP-418 during the 36 months treatment phase.
Peter Kang
STUDY00018570
Inclusion Criteria:
• 12 to 60 years of age
• genetically confirmed diagnosis of limb girdle muscular dystrophy
• have clinical symptoms of weakness
• weight at least 30 kg (66 lbs.)
• willing to use a highly effective method of birth control until 12 weeks after last dose of study medication
Exclusion Criteria:
• any significant medical or mental health diagnosis including abnormal lab values (study staff will review)
• surgery for scoliosis or other indication planned during the time of the study
• use of ribose or other sugar alcohol-containing supplement within 90 days of staring the study
• use of a systemic corticosteroid for the treatment of muscular dystrophy within 90 days of starting the study
Rare Diseases
Clinics and Surgery Center (CSC), Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), Muscular Dystrophy
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults with Dermatomyositis (VALOR)
In this study, brepocitinib will be compared to a placebo. Brepocitinib is an investigational medicine because it has not yet been approved by any regulatory agency for use. Researchers will compare the results of taking the placebo to the results of taking the study medicine to see if there are any differences. This medicine may be helpful for your disease, but we do not have any information about this yet. 67% of participants will receive brepocitinib and 33% will receive the placebo which will be decided randomly by chance. Participation will last for up to 64 weeks (15 months). Visits will be scheduled about every 4 to 6 weeks.
David Pearson
STUDY00016860
Inclusion Criteria:
• diagnosis of dermatomyositis
• active muscle and skin disease or being treated with medications
• age 18-75
• weight at least 40 kg, less than 130 kg and a BMI less than 40 kg/m2
Exclusion Criteria:
• history of cancer in past 5 years
• dermatomyositis with irreversible muscle involvement
• active or recent infections
Arthritis & Rheumatic Diseases, Dermatology (Skin, Hair & Nails)
Clinics and Surgery Center (CSC), dermatomyositis
MT2023-06: A CLINICAL STUDY TO ASSESS THE EFFICACY AND SAFETY OF LERIGLITAZONE IN ADULT MALE SUBJECTS WITH CEREBRAL ADRENOLEUKODYSTROPHY (CALYX)
- To evaluate the efficacy of leriglitazone compared to placebo at increasing survival. - To evaluate the efficacy of leriglitazone compared to placebo at slowing radiological progression. - To evaluate the safety and tolerability of leriglitazone compared to placebo.
Troy Lund
STUDY00018982
Key
Inclusion Criteria:
Subject is male and aged ≥18 years.
Subject has progressive cALD, defined as GdE+ brain lesions.
Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.
Subject has a Loes score ≥0.5 and ≤12 at Screening.
Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease
Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening.
Key Exclusion Criteria:
Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).
Subject has known type 1 or type 2 diabetes.
Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione.
Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.
Subject with current participation in another interventional clinical study or within 1 month prior to Screening.
Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results. Rare Diseases
Clinics and Surgery Center (CSC), CEREBRAL ADRENOLEUKODYSTROPHY
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.
Colleen Correll
1506M74443
Inclusion Criteria:
• 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
Arthritis & Rheumatic Diseases, Cancer
juvenile idiopathic arthritis (JIA), pediatric rheumatic disease
A Phase 1b/2 Study of Abemaciclib in Combination with Irinotecan and Temozolomide (Part A) and Abemaciclib in Combination with Temozolomide (Part B) in Pediatric and Young Adult Patients with Relapsed/Refractory Solid Tumors and Abemaciclib in Combination with Dinutuximab, GM-CSF, Irinotecan, and Temozolomide in Pediatric and Young Adult Patients with Relapsed/Refractory Neuroblastoma (Part C). Protocol Number: I3Y-MC-JPCS
The study's purpose is to see if the drug abemaciclib is safe and effective in combination with temozolomide and irinotecan (Part A) and abemaciclib in combination with temozolomide (Part B) in pediatric and young adult participants with relapsed/refractory solid tumors.
Emily Greengard
STUDY00013998
Inclusion Criteria:
Parts A and B only:
Participants must be less than or equal to (≤)18 years of age.
Body weight greater than or equal to (≥)10 kilograms and body surface area (BSA) ≥0.5 -- Participants with any relapsed/refractory malignant solid tumor (excluding lymphoma), including central nervous system tumors, that have progressed on standard therapies.
For sites that are actively enrolling Parts B and C, participants with neuroblastoma who are eligible for Part C will be excluded from Part B unless approved by Lilly CRP/CRS.
Part C only:
Participants must be less than (<) 21 years of age.
Participants have a BSA ≥0.3 m².
Participants with first relapse/refractory neuroblastoma.
All Parts
Participants must have measurable or evaluable disease by RECIST v1.1 or RANO.
A Lansky score ≥50 for participants <16 years of age or Karnofsky score ≥50 for participants ≥16 years of age.
Participants must have discontinued all previous treatments for cancer or investigational agents and must have recovered from the acute effects to Grade ≤1 at the time of enrollment.
Able to swallow.
Adequate hematologic and organ function ≤2 weeks (14 days) prior to first dose of study drug.
Females of reproductive potential must have negative urine or serum pregnancy test at baseline (within 7 days prior to starting treatment).
Female participants of reproductive potential must agree to use highly effective contraceptive precautions during the trial. For abemaciclib, females should use contraception for at least 3 weeks following the last abemaciclib. For other study drugs, highly effective contraceptive precautions (and avoiding sperm donation) must be used according to their label.
Life expectancy of at least 8 weeks and able to complete at least 1 cycle of treatment.
Caregivers and participants willing to make themselves available for the duration of the trial.
Exclusion Criteria:
Received allogenic bone marrow or solid organ transplant.
Received live vaccination.
Intolerability or hypersensitivity to any of the study treatments or its components.
Diagnosed and/or treated additional malignancy within 3 years prior to enrollment that may affect the interpretation of results, with the exception of curatively treated basal cell carcinoma of the skin, squamous cell carcinoma of the skin, and/or curatively resected in situ cervical and/or breast cancers.
Pregnant or breastfeeding.
Active systemic infections or viral load.
Serious and/or uncontrolled preexisting medical condition(s) that would preclude participation in this study.
Parts A and C only: Have a bowel obstruction.
Prior treatment with drugs known to be strong inhibitors or inducers of isoenzyme cytochrome P450 3A (CYP3A) or strong inhibitors of uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) if the treatment cannot be discontinued or switched to a different medication at least 5 half-lives prior to starting study drug.
Received prior treatment with cyclin-dependent kinase (CDK) 4 & 6 inhibitor.
Part C only: Received prior systemic therapy for relapsed/refractory neuroblastoma.
Currently enrolled in any other clinical study involving an investigational product or non-approved use of a drug or device.
Has received an experimental treatment in a clinical trial within the last 30 days or 5 half-lives, whichever is longer.A Randomized Double Blind Phase II Trial of Restorative Microbiota Therapy (RMT) or Placebo in Combination with Durvalumab (MEDI4736) and Tremelimumab With Chemotherapy in Treatment Naive Advanced or Metastatic Adenocarcinoma Non-Small Cell Lung Cancer
This is a randomized, active-controlled, parallel-group, double-blind Phase II trial, of oral restorative microbiota therapy (RMT) or placebo combined with intravenous (IV) durvalumab (MEDI4736) plus tremelimumab and chemotherapy in patients with treatment naïve advanced or metastatic adenocarcinoma non-small cell lung cancer (NSCLC). The primary objectives include: -To evaluate the efficacy of restorative microbiota therapy (RMT) in combination with durvalumab and tremelimumab plus chemotherapy compared with placebo in combination with durvalumab and tremelimumab plus chemotherapy using PFS per RECIST 1.1 as assessed by the investigator -To evaluate the safety and feasibility of restorative microbiota therapy (RMT) in combination with durvalumab and tremelimumab plus chemotherapy in patients with untreated advanced or metastatic adenocarcinoma non-small cell lung cancer (NSCLC)
Amit Kulkarni
STUDY00007800
Inclusion Criteria:
Histologically or cytologically confirmed adenocarcinoma of the lung that is unresectable stage IIIB/C or stage IV, does not have an EGFR sensitizing (activating) mutation or ALK or ROS1 translocation. BRAF, RET, NTRK, MET ex 14 splice site mutation
Measurable disease based on RECIST 1.1
Tumor sample requirements
Mandatory provision of an unstained, archived tumor tissue sample in a quantity sufficient to allow for biomarker and genomic analysis. A minimum of 10 unstained slides should be available for evaluation.
Known PD-L1 TC expression status assayed by Ventana SP263. Patients who have known PDL-1 as assayed by PharmDx 22C3 assay may be eligible; however, available archival tissue will be used to assay with Ventana SP263 test.
Prior chemotherapy or immunotherapy as adjuvant therapy for lung cancer is permitted as long as it has been >6 months from last dose at the time of enrollment. Local treatment of isolated lesions, excluding target lesions, for palliative intent is acceptable (e.g. by local surgery or radiotherapy). Prior systemic therapy for advanced/metastatic NSCLC makes the patient ineligible for this study.
Patients with treated brain metastasis are eligible as long as they have stable symptoms, are more than 2 weeks from completion of therapy, and do not require more than 10mg of daily prednisone or equivalent.
ECOG Performance status of 0 or 1
Body weight of >30 kg
Adequate organ function within 14 days of study enrollment defined as:
Hemoglobin ≥ 9.0 g/dL
Absolute neutrophil count ≥1,500/mcL
Platelets ≥ 100,000/mcL
Total bilirubin ≤1.5x upper limit of normal (ULN) - this will not apply to patients with confirmed Gilbert's syndrome (persistent or recurrent hyperbilirubinemia that is predominantly unconjugated in the absence of hemolysis or hepatic pathology), who will be allowed only in consultation with their physician.
AST (SGOT) and ALT (SGPT) ≤2.5 x institutional ULN unless liver metastases are present, in which case it must be ≤5x ULN
Measured creatinine clearance (CL) >40 mL/min or calculated creatinine CL>40 mL/min by the Cockcroft-Gault formula (Cockcroft and Gault 1976) or by 24-hour urine collection for determination of creatinine clearance:
Expected life expectancy of at least 12 weeks in the opinion of the enrolling investigator as documented in the medical record
Women of childbearing potential and men with partners of child-bearing potential must agree to use effective contraception for the time of screening to the duration of treatment and 3 months after the last dose of study drug
Provide voluntary written consent prior to the performance of any research related tests or procedures.
Exclusion Criteria:
Not pregnant or breast feeding. Evidence of post-menopausal status, or negative urine or serum pregnancy test for female pre-menopausal patients. Women will be considered postmenopausal if they have amenorrhea for 12 months without an alternative medical explanation
Dysphagia or inability to swallow medications
Squamous cell, large cell, or NSCLC NOS (not otherwise specified) histology or mixed tumors
Has untreated brain metastasis or active leptomeningeal carcinomatosis
Has a known sensitivity to any component of therapeutic agents used in this study
Receipt of any immunotherapy or investigational drug within 4 weeks prior to the first dose of study drug; and in the case of monoclonal antibodies 6 weeks prior to the first dose of study drug
Prior treatment with any other anti-PD-1, or PD-L1, including durvalumab or an anti-PD-L2 agent or an antibody or a small molecule targeting other immuno-regulatory receptors except as adjuvant therapy for NSCLC so long as it has been greater than six months since the last treatment
Current or prior use of immunosuppressive medication within 28 days before the first dose of study drug, with the exceptions of intranasal and inhaled corticosteroids or systemic corticosteroids at physiological doses, which are not to exceed 10 mg/day of prednisone, or an equivalent corticosteroid
Recent major surgery within 4 weeks prior to entry into the study (excluding the placement of vascular access) that would prevent administration of study drug
Active or prior documented autoimmune disease within the past 2 years requiring systemic treatment (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Patients with vitiligo, Grave's disease, or psoriasis not requiring systemic treatment (within the past 2 years) are not excluded. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment.
Active documented inflammatory bowel disease (eg, Crohn's disease, ulcerative colitis) who require immunosuppression or patients who exceed 10 mg/day of prednisone, or an equivalent corticosteroid are excluded
History of primary immunodeficiency
History of organ transplant that requires therapeutic immunosuppression
Taking daily probiotics (patients with last probiotic > 4weeks prior to first dose of RMT are eligible)
History of Human Immunodeficiency Virus (HIV) (known HIV 1/2 antibodies positive) who are on anti-retroviral treatment for < 6 months and absolute CD4 count<500 (patients with HIV not meeting these criteria are eligible)
Has known active Hepatitis B or C. Active Hepatitis B is defined as a known positive HBsAg, and positive hepatitis B virus quantification assay (patients with history of Hepatitis B who have seroconversion i.e. Hepatitis B core antibody positive and Hepatitis B surface antibody positive are eligible). Active Hepatitis C is defined by a known positive Hep C Ab result and positive quantitative HCV RNA results (Patients with Hepatitis C who are on anti-viral suppressive therapy and negative quantitative HCV RNA results are eligible)
Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, uncontrolled hypertension, unstable angina pectoris, cardiac arrhythmia, active peptic ulcer disease or gastritis, active bleeding diatheses
Myocardial infarction or stroke within 3 months prior to enrollment
History of systolic or diastolic heart failure with New York Heart Association (NYHA) class III or IV symptoms (refer to Appendix II)
Has active or prior history of (non-infectious) pneumonitis that required steroids or patients with interstitial lung disease
Psychiatric illness/social situations that would limit compliance with study requirements or compromise the ability of the patient to give written informed consent
Known history of active tuberculosis. Patients with prior history of latent TB could be included if they have been treated previously with isoniazid.
Patients who are on chronic systemic antibiotic therapy (antibiotics for ≥60 consecutive days within 12 weeks of enrollment). Patients who receive systemic antibiotics between enrollment and start of RMT are eligible
Receipt of live attenuated vaccination within 30 days prior to study entry or within 30 days of receiving RMT
History of another primary malignancy (excluding non-melanoma skin cancer) within 5 years prior to starting RMT, except if the patient has undergone potentially curative therapy with no evidence of disease recurrence for 5 years since initiation of that therapy
Any condition that, in the opinion of the investigator, would interfere with evaluation of the study drug or interpretation of patient safety or study results Clinics and Surgery Center (CSC)
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
STUDY00012932
Inclusion Criteria:
• ages 12-17
• BMI 35 kg/m2 or more or Body Mass Index 120% or more for 95th percentile for age and sex
Exclusion Criteria:
• Type 1 or Type 2 diabetes
• use of medications for obesity in the past 6 months
• any treatment with growth hormone
• bariatric surgery -major mental health diagnosis (study staff will review)
• pregnant or plan to become pregnant
• significant medical diagnosis (study staff will review)
Diabetes & Endocrine, Children's Health
Obesity, Overweight, weight loss
Minnesota KPMP CKD and Resilient Diabetes Recruiting Site (KPMP)
The Kidney Precision Medicine Project (KPMP) is a prospective cohort study, whose goal is to use kidney biopsies, along with clinical medical data, to develop new disease classification systems, and treatments for acute kidney injury (AKI) and chronic kidney disease (CKD). The University of Minnesota KPMP site will focus on recruiting participants with CKD associated with type 1 diabetes (CKD-T1D), type 2 diabetes (CKD-T2D), or hypertension (CKD-HTN), in addition to recruiting participants with resilient type 1 diabetes (Res-T1D). People with Res-T1D have had type 1 diabetes for many years and no clinical signs of CKD. Both AKI and CKD are conditions that impose a significant global health burden. Yet, no effective therapies currently exist for AKI, and only a few are available for CKD. To address this need, KPMP will obtain kidney biopsy tissue, blood, urine, and stool samples, and medical data from study participants. The network will utilize state-of-the-art methods to perform analysis on the samples collected to define kidney disease subgroups in molecular terms by identifying critical cells, pathways and targets for novel therapies.
Patrick Nachman
STUDY00013784
Inclusion Criteria:
• at least 18 years old
• diagnosis acute or chronic kidney (renal) disease with diabetes mellitus (type 1 or 2) OR associated with hypertension
• persistent kidney damage based on specific lab values at least 3 months apart (study staff will review)
Exclusion Criteria:
• Body Mass Index (BMI) greater than 40 kg/m2
• any allergic reaction to iodinated contrast
• receiving chemotherapy or radiation to treat cancer
• transplant recipient (includes solid transplant and bone marrow)
• unwilling to receive blood transfusion (if needed)
• women who are pregnant
Kidney, Prostate & Urinary
Clinics and Surgery Center (CSC), Acute Kidney Failure, Acute Kidney Insufficiency, Acute Renal Failure, Chronic Kidney Disease
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.
Antoinette Moran
STUDY00019323
Inclusion Criteria:
• 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
Exclusion Criteria:
• 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
Children's Health, Diabetes & Endocrine
T1D, new onset, type 1 diabetes,