Search Results
MT2021-01: PTCy + Sirolimus/VIC-1911 as GVHD prophylaxis in myeloablative PBSC transplantation
The primary objective of this study is to determine the optimal dose of VIC-1911 when given in combination with standard immunosuppressive therapy in adult patients undergoing myeloablative stem cell transplantation.
Establishing patient derived xenografts for MPNST
We hope to use patient samples of MPNSTs to learn more about tumors in order to identify new drugs and drug combinations that could help patients with MPNST in the future. For this research, we will ask participants to provide samples of their tumors and blood while they are undergoing biopsies or tumor resections. We will study the tumors by implanting samples into mice and treating them with therapeutic drugs. We will also perform genetic assessment and other analysis of the tumors and blood. Recruiting participants with MPNST over the age of 12
• at least 12 years old
• a suspected or previously confirmed Malignant Peripheral Nerve Sheath Tumor (MPNST) with or without neurofibromatosis type 1 syndrome
• undergoing diagnostic biopsies or tumor resection surgeries
MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders
The TrialNet Natural History Study of the Development of Type 1 Diabetes
TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.
• 2.5 to 45 years old and have an immediate family member with type 1 diabetes (child, parent, or sibling)
• 2.5 to 20 years old who have an extended family member with type 1 diabetes (cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
• already have diabetes
• history of being treated with insulin or oral diabetes medications
• using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
• have any known serious diseases
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
Pilot study to determine safety of lower extremity injection of agitated saline for echocardiography of the heart.
Prospective study to compare safety of upper versus lower extremity injection agitated saline (bubble study) using echocardiography of the heart.
• 18 years or older
• "bubble study" i.e. echocardiography with agitated saline injection ordered
• enrolled in another drug or medical device study within 30 days of study enrollment.
• liver failure in past 6 months
MT2023-31: A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT) (HURCULES)
This research study is designed to compare a new gene therapy, known as OTL-203 (study drug), with a standard treatment called “allogeneic hematopoietic stem cell transplant” (allo-HSCT), to find out which is better for the treatment of MPS-IH.
• at least 28 days old to no more than 30 months old
• confirmed laboratory diagnosis of MPS-IH
• evidence of altered GAG metabolism
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• previous allo-HSCT or gene therapy
• diagnosis of HIV, Hepatitis B, Hepatitis C, or Mycoplasma
• history of uncontrolled seizures
• contraindications for MRI scans
• study staff will review additional exclusion criteria
MT2022-52: Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) with Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases
Stem cell transplants (sometimes referred to as a bone marrow transplants) have been done for over 40 years but research continues to further refine the method to reduce side effects without affecting transplant success. The purpose of this study is to improve on transplant outcomes while reducing the potential side effects based on what has been learned from previous transplant studies using a reduced intensity preparative regimen. Information collected during this study (transplant outcomes and side effects) will be compared with the outcomes of the previous reduced intensity conditioning transplant study that enrolled more than 300 patients since 2002.
• up to 75 years of age
• have a matched related donor
• additional criteria for diagnosis, and physical status (study staff will review)
• women who are pregnant or breast feeding
• active central nervous system malignancy
• untreated active infection
• additional criteria for exclusion (study staff will review)
MT2023-23: A Phase 2, Open-Label, Multi-Center Study of Innate Cell Engager AFM13 in Combination with Allogeneic Natural Killer Cells (AB-101) in Subjects with Recurrent or Refractory Hodgkin Lymphoma and CD30-Positive Peripheral T-Cell Lymphoma (LuminICE-203)
The purpose of this study is to learn about the effectiveness and safety of a new study drug called AFM13 when used in combination with a new cell therapy called AB-101. AFM13 is an antibody designed to bind to cancer cells and to “natural killer” cells. AB-101 refers to natural killer cells that were obtained from human umbilical cord blood. Natural killer cells are part of your immune system and their primary function is fighting infections and cancer. AFM13 binds the natural killer cells and links them with the cancer cells, so they can eliminate the cancer cells.
• diagnosis of relapsed or refractory classical Hodgkin Lymphoma (HL) or select subtypes of relapsed or refractory Peripheral T Cell Lymphoma (PTCL)
• must have received previous therapy (study staff will review)
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• active central nervous system (CNS) involvement
• active Hepatitis B or C or HIV infection
• history of any other systemic cancer, unless previously treated with curative intent and the subject has been disease free for 2 years or longer
• active acute or chronic graft vs. host disease (GVHD) or GVHD requiring immunosuppressive treatment
A Phase 3, Open-Label, Multi-Center, Randomized Study Evaluating the Efficacy and Safety of TAR-200 in Combination with Cetrelimab or TAR-200 Alone Versus Intravesical Bacillus Calmette-Guerin (BCG) in Participants with BCG-naive High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) (SunRISe-3)
The purpose of this study is to compare the effects (both good and bad) of an investigational drug delivery system (TAR-200) in combination with cetrelimab or TAR-200 alone to the effects of study drug comparator intravesical (medicine that is put directly into the bladder instead of being taken like a pill or put into veins) BCG in patients with HR-NMIBC. Cetrelimab is a medicine that may treat certain cancers by working with the immune system (it is also known as immunotherapy). Immunotherapy is the use of medicines to help a person’s own immune system recognize and destroy cancer cells.
• diagnosis of high grade non-muscle invasive bladder cancer (HR-NMIBC) (high-grade Ta, any T1 or carcinoma in-situ [CIS])
• have not received Bacillus Calmette Guerin (BCG)
• cancer must be surgically removed
• able to walk and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• more extensive bladder cancer (muscle invasive, locally advanced, nonresectable, or metastatic urothelial carcinoma (that is, greater than and equal to [>=] T2))
• history of clinically significant polyuria with recorded 24-hour urine volumes greater than 4000 milliliters (mL)
• Indwelling catheters are not permitted; however, intermittent catheterization is acceptable
• additional exclusion criteria (study staff will review)
A randomized phase II trial of adjuvant Pembrolizumab versus observation following curative resection for stage I non-small cell lung cancer (NSCLC) with primary tumors between 1-4 cm: Big Ten Cancer Research Consortium BTCRC-LUN18-153
This is a research study to find out if giving a drug called pembrolizumab after lung cancer surgery does a better job at keeping the cancer from coming back than surgery alone.
• at least 18 years old
• diagnosis of non-small cell lung cancer (NSCLC)
• tumor size between 1 and 4 cm in size
• had a complete surgical resection of stage I NSCLC between 4-12 weeks ago
• able to walk and carry out basic activities of living
• women are willing to use highly effective birth control for 120 days after last dose of study drug
• certain laboratory values are required (study staff will review)
• chemotherapy, radiation therapy, or immunotherapy for the treatment of this lung cancer
• active additional cancer that is progressing or has required treatment within the past 3 years
• diagnosis of immunodeficiency or receiving chronic steroid therapy
• women who are pregnant or breast feeding
• other active diseases (study staff will review)
HM2017-24 : Phase I/II Study of Nivolumab in Combination with Ruxolitinib in Relapsed or Refractory Classical Hodgkin Lymphoma: BTCRC-HEM-027
Participants who take part in this study will receive a study drug called ruxolitinib with a standard drug called nivolumab. The study is being done to measure the percentage of tumor (lymphoma) that shrinks after receiving ruxolitinib in combination with nivolumab. This study will also measure the length of time the lymphoma is inactive and how safe the combination is to administer to participants. Ruxolitinib is a pill that is taken twice every day. Nivolumab is given as an infusion in the clinic once every 4 weeks.
• age 18 or older
• able to walk and do all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• confirmed diagnosis of classical Hodgkin lymphoma that has reoccurred or not responded to treatment
• women and men who are of child bearing age must use required birth control
• there are additional criteria for prior treatment and laboratory results (study staff will review)
• inability to swallow oral medication or any condition that affects absorption of oral medications
• women who are pregnant or breast feeding
• additional criteria about current medical history (study staff will review)
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.
• 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
• 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)
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.
HM2023-07: Phase 3 Randomized Study Comparing Talquetamab SC in Combination With Daratumumab SC and Pomalidomide (Tal-DP) or Talquetamab SC in Combination With Daratumumab SC (Tal-D) Versus Daratumumab SC, Pomalidomide and Dexamethasone (DPd), in Participants With Relapsed or Refractory Multiple Myeloma who Have Received at Least 1 Prior Line of Therapy (MonumenTAL-3)
Talquetamab is an experimental medication being studied to see if it may be beneficial in the treatment of multiple myeloma.
• diagnosed with multiple myeloma that has not responded to treatment or has reoccurred after at least one treatment
• able to care for self with some assistance
• active cancer in central nervous system or clinical symptoms
• maximum amount of steriods taken in the past two weeks (study staff will review)
• disease hasn't responded to this type of drug (anti-CD38 monoclonal antibody)
Dissecting the role of acetaldehyde in oral carcinogenesis
The goal of this study is to better understand how drinking alcohol may lead to oral cancers. Acetaldehyde, a chemical formed when the body breaks down alcohol, is believed to play an important role. This study will measure acetaldehyde and DNA damage levels in the mouth of participants after a low dose of alcohol. The levels will be compared between three groups, all having different degrees of risk for developing oral cancer, in order to identify DNA damage that might be crucial to cancer formation.
• 21 to 45 years of age alcohol drinker
• Experiences flushing (reddening or warming of face) when you drink OR have Fanconi Anemia
• 18 to 45 years of age non-drinkers
• Tobacco or nicotine users
MT2020-27: Phase I/II Trial Using E7777 to Enhance Regulatory T-Cell Depletion Prior to Tisagenlecleucel (Kymriah) Therapy for Relapsed/Refractory Diffuse Large B-Cell Lymphoma (DLBCL)
This purpose of this study is to identify a safe dose level for the study drug, E7777, when given with standard tisagenlecleucel therapy (also known by its brand name, Kymriah, is an immunotherapy that is made from the participants own blood cells) in participants with Diffuse Large B-Cell Lymphoma (DLBCL). Up to three dose levels of E7777 will be tested.
• diagnosis of a relapse or refractory large B cell lymphoma, for which treatment with Kymriah is planned
• received two or more lines of systemic therapy
• able to walk and do all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• participants of child bearing age must use birth control for 30 days following completion of treatment
• additional inclusion criteria (study staff will review)
• women who are pregnant or breast feeding
• CNS involvement by malignancy
• eye disease or complaints visual acuity impairment, color or shape distortion, or blurred vision - potential participants are required to have an eye exam as part of screening
• additional exclusion criteria (study staff will review)
COG AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
• patients must be > 365 days and < 25 years of age
• participant has newly diagnosed B-ALL or MPAL with ?25% blasts on a bone marrow (BM) aspirate or newly diagnosed B-LLy
• see link to clinicaltrials.gov for complete inclusion criteria
• patients with Down syndrome are not eligible
• patients with acute undifferentiated leukemia (AUL) are not eligible
• female patients who are pregnant, since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
• lactating women who plan to breastfeed their infants while on study and for 2 months after the last dose of inotuzumab ozogamicin.
• see link to clinicaltrials.gov for complete exclusion criteria
A Phase 3, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the& Efficacy, Safety, and Pharmacokinetics of Baricitinib in Children from 6 Years& Less than 18 Years of Age with Alopecia Areata
HM2022-48: A Phase 1/2 Dose Escalation Study of the BCL-2 Inhibitor ZN-d5 and the Wee1 Inhibitor ZN-c3 in Subjects with Acute Myeloid Leukemia
This study is being performed to determine the safety and tolerability of ZN-c3 alone and the combination of ZN-c3 and ZN-d5 in Acute Myeloid Leukemia (AML). We want to identify the best doses of the study drugs and learn if either drug effects the blood levels of the other. We will also assess how effective the Study Drugs are in treating AML and explore whether certain aspects of AML can predict whether leukemia responds to the study drug(s).
• adults with Acute Myeloid Leukemia (AML) (including secondary or therapy-related), relapsed from or refractory to one or more prior lines of therapy
• able to walk and do selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• women of childbearing potential must not be pregnant and must use effective birth control during the study and for 6 months after the last dose of study drugs
• men must agree to use a condom when having intercourse during the study and for 3 months after the last dose of study drugs
• active central nervous system (CNS) involvement
• significant cardiovascular disease
• active hepatitis B or hepatitis C infection
• additional exclusion criteria (study staff will review)
Computer Aided Diagnostic System for Prostate Cancer Detection Using Quantitative Multiparametric MRI
In a partnership with Invenshure/Flywheel (Minneapolis, MN), this study aims to improve and integrate prostate specific quantitative imaging methods and develop a pipeline to introduce CBS-CAD into the clinic.
• at least 18 years old
• under clinical observation/work-up for suspected prostate cancer but not yet diagnosed
• able to read, write, and speak in English
• Control Participants: prior prostate biopsy, history of prostate cancer and/or concerns of rising prostate specific antigen, or contraindication to MRI
• Patient Participants: history of prostate cancer related procedures including but not limited to prostate biopsy, surgery, radiation therapy or hormone therapy, or contraindication to MRI
Time Restricted Eating as a Viable Alternative to Caloric Restriction for Treating Hyperglycemia in a Population with Type 2 (T2DM) diabetes (SFS3)
Feasibility study to test our overall hypothesis that time restricted eating (TRE) presents a viable alternative to caloric restriction for improving glycemic measures and reducing weight in overweight/obese patients with metformin-only treated Type 2 diabetes (T2DM).
• adults who are overweight/obese and have type 2 diabetes treated only with metformin
• 18-65 years old
• BMI between 25-40 kg/m2
• HbA1c between 6.5-8.5%
• self reported weight must be stable (+/- 5 pounds) for at least 3 months prior to the study
• own a smartphone
• women who are pregnant or are planning to become pregnant
• eating disorders
MT2023-06: A CLINICAL STUDY TO ASSESS THE EFFICACY AND SAFETY OF LERIGLITAZONE IN ADULT MALE SUBJECTS WITH CEREBRAL ADRENOLEUKODYSTROPHY (CALYX)
This study has 2 parts: a double-blind period and an open-label extension. In the double-blind period of this study, the study medicine will be compared to a placebo. A placebo is a treatment that looks and tastes exactly like the study medicine but does not contain any active ingredient. In this study, you will receive leriglitazone or placebo. Whether you receive leriglitazone or placebo will be decided randomly (by chance, like flipping a coin). In this study, 1 out of every 2 subjects (50%) will receive leriglitazone and 1 out of every 2 subjects (50%) will receive placebo. To make this study fair, you and the study doctor will not be told which treatment you will receive, this is called “blinding”. In the open-label extension, all subjects will receive leriglitazone.
• diagnosis of progressive cerebral adrenoleukodystrophy (cALD), defined as GdE with brain lesions
• bone marrow transplantation (HSCT) is not recommended patient is not willing to undergo HSCT
• no major cognitive impairment
• see link to clinicaltrials.gov for additional inclusion criteria
• or treatment with ex-vivo gene therapy (eli-Cel).
• known type 1 or type 2 diabetes
• see link to clinicaltrials.gov for additional exclusion criteria
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
The investigational therapy in this study is referred to as Restorative Microbiota Therapy (RMT). It is prepared by extracting healthy bacteria from the stool of healthy human donors and making it into capsules taken by mouth. The donor stool samples are rigorously tested for harmful bacteria and viruses before processing. There is scientific evidence to suggest that RMT might make immunotherapy more effective. The primary goal of the study is to test if RMT makes durvalumab + tremelimumab treatment with chemotherapy more effective to control lung cancer.
• confirmed adenocarcinoma of the lung that is stage IIIB/C or stage IV that can't be surgically removed
• prior chemotherapy or immunotherapy as adjuvant therapy for lung cancer is permitted as long as it has been more than 6 months from last dose
• people who have 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
• restricted in strenuous physical activity but can walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• weigh at least 30 kg (66 lbs.)
• contact study staff for additional requirements
• women who are pregnant or breast feeding
• unable to swallow medications
• additional medical and mental health diagnosis (study staff will review)
Randomized Clinical Evaluation of the AccuCinch Ventricular Restoration System in Patients who Present with Symptomatic Heart Failure with Reduced Ejection Fraction (HFrEF)
The objective of this study is to evaluate the safety and efficacy of the AccuCinch Ventricular Restoration System in patients with symptomatic heart failure with reduced ejection fraction (HFrEF).
• at least 18 years old
• Ejection Fraction: between 20% and 40% measured by transthoracic echocardiography (TTE)
• diagnosis and treatment for heart failure should be established at least 90 days before entering the study & should be on stable, optimal medical therapy for at least 30 days
• myocardial infarction or any percutaneous cardiovascular intervention, cardiovascular surgery, or carotid surgery within 90 days prior to consent
• any planned cardiac surgery or interventions within the next 180 days
• women who are pregnant, planning to become pregnant, or are breast feeding
• additional cardiac and medical diagnosis will exclude participation (study staff will review)
STUDY OF PHIL EMBOLIC SYSTEM IN THE TREATMENT OF INTRACRANIAL DURAL ARTERIOVENOUS FISTULAS (PHIL dAVF)
• 22 to 80 years old
• diagnosis of intracranial arteriovenous dural fistula (dAVF)
• multiple dAVFs to be treated
• history of life threatening allergy to contrast media (unless treatment for allergy is tolerated)
• women who are pregnant
A Phase IIa, Randomized, Parallel, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Enpatoran in Dermatomyositis and Polymyositis Participants receiving Standard of Care
This research is studying enpatoran (M5049) as a possible treatment for dermatomyositis (DM) and polymyositis (PM). The study will last for approximately 31 weeks (upto 9 study visits) and an additional optional 24 weeks (upto 5 study visits) if you agree to participate in the extension period for a total of up to 14 months (55 weeks). It will include approximately up to 3 telephone calls from the study center. The main activities in the study include: providing consent to participate, performing tests to check your health throughout the study (such as physical exams, electrocardiogram (ECG) testing, collection of urine and blood draws for laboratory testing), study drug dosing and recording relevant information in the study diary, and performing tests, completing questionnaires and assess signs and symptoms of your condition. This is a Phase 2a study. and it is anticipated that approximately 40 people will participate in this study.
MT2023-10: A Phase 1 Study of FT522 in Combination with Rituximab in Participants with Relapsed/Refractory B-Cell Lymphoma
We are studying FT522 - a new product that is made by modifying cells in a laboratory - both with and without additional drugs, to see if it can help treat people with B-cell lymphoma. This study is for people who have had at least one treatment for their lymphoma, but the cancer either returned or did not respond to the treatment. We are testing this product to see what side effects it might have, as well as to see whether it is effective at treating B-cell lymphoma.
• diagnosis of B-cell lymphoma (BCL)
• at least 1 prior systemic regimen of treatment
• men and women participants of childbearing potential who engage in heterosexual intercourse must agree to use specified method(s) of contraception
• women who are pregnant or breastfeeding
• capable of only limited selfcare; confined to bed or chair more than 50% of waking hours
• body weight less than 50 kg (110 lb.)
• additional medical diagnosis (study staff will review)
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 research is being done to determine whether the investigational drug tabelecleucel (allogeneic Epstein-Barr virus-specific cytotoxic T lymphocytes [EBV-CTLs]) can help people with EBV-associated diseases.
• diagnosis of Epstein-Barr Virus (EBV) disorder
• able to walk and do all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• see link to clinicaltrials.com for additional inclusion criteria
• women who are breastfeeding or pregnant
• 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
• additional exclusion criteria apply (study staff will review)
MT2022-41 A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial) (BEACON)
BEAM-101 is an experimental new therapy being developed for treating people with SCD and vaso-occlusive crises. The goal of this study is to see if BEAM-101 is safe and effective for people in the study. The study sponsor and study doctors would also like to see if individuals who are treated with BEAM-101 require fewer blood transfusions and experience fewer vasoocclusive crises requiring hospitalization, compared to before they received BEAM-101. This study will also measure the levels of fetal hemoglobin along with measures that assess quality of life and ability to function following treatment with BEAM-101.
• 18 to 35 years old
• documented diagnosis of sickle cell disease with specific genotypes (study staff will review)
• disease is severe
• HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
• previous transplant
• history of an overt stroke