Research Studies Finder

Researchers at CHOP are recruiting patients diagnosed with either acute leukemia (myeloid, lymphoid), myelodysplastic syndromes, or aplastic anemia and treated with allogeneic hematopoietic cell transplantation (HCT) within the past 6-48 months. Research has shown that adolescents and young adults who are treated with HCT are at increased risk for skeletal muscle loss. So, the purpose of this study is to investigate if a combination of exercise training and an exercise supplement can mitigate this effect. Thesupplement is called nicotinamide riboside. Participants will be randomly assigned to 1 of 4 groups:

• Exercise + Supplement
• Exercise + Placebo
• No Exercise + Supplement
• No Exercise + Placebo

The purpose of the study is to learn more about the experience of caregivers who have a child diagnosed with autism. We also want to better understand what is working well in healthcare in the US. This study is open to primary caregivers of a child (aged 3-17) who has been diagnosed with autism spectrum disorder in the last year. Participants must have immigrated to the US and identify as Hispanic or Latinx. Study staff will interview participants about their experiences as a caregiver of an autistic child. This one-time interview will last 60-90 minutes in Spanish or English.

The goal of this study is to observe patients with Homocystinuria (CBSDH) over 3 years to learn how their disease is managed under regular circumstances. This study does not involve any investigational medications, but will provide information to researchers who are currently developing a medication to treat the disease. The study has 5 main parts, all of which are provided free of charge to participants: DXA exam, blood draws, ophthalmology exam, cognitive testing, and physical exams. To learn more about the study, please contact our team via the information listed below.

The purpose of this study is to look at the safety and tolerability of RBX-121, a one-time gene therapy, in subjects with MPS II (Hunter Syndrome). You may be eligible for this study if you are a male between the ages of 4 months to 5 years old. Reimbursement for travel and research procedures will be provided. This study involves blood draws, interviews, urine collection, genetic testing, general anesthesia, lumbar punctures, MRI, gene therapy, lumbar puncture, ultrasound, ECG/echocardiogram and physical and neurological examinations. If you have questions or would like to learn more about the study, please contact the study team at MetabolismResearch@email.chop.edu.

The purpose of this study is to find out if JR-141, an experimental drug, works better than the standard treatment, idursulfase, for MPS II (Hunter Syndrome) and how safe and effective it is in the management of central nervous system symptoms (such as brain or body function loss) and other body symptoms (including lungs, ears, heart, kidneys, and eyes, as well as any type of liver, bone or joint abnormalities) related to MPS II. Since JR-141 is an investigational drug, it has not been approved by the Food and Drug Administration (FDA).

The purpose of this study is to test and find a safe dose of this investigational gene therapy PBGM01 (also known as, the study drug) in children who have been diagnosed with the early infantile or late infantile form of GM1. The study will also try to determine how PBGM01 might affect the GM1 disease course over time. The study drug is investigational, which means it has not been approved for marketing by the FDA. This study is the first time PBGM01 will be given to humans, so we do not know for sure if participants will benefit from this study.

The purpose of this study is to determine the safety and effectiveness of an experimental form of therapy, known as BEAM-201. BEAM-201 uses immune cells, called T cells, that are genetically changed to identify cancer cells. In this study, T cells are collected from a healthy human donor and have been modified in a laboratory. If the genetically changed T cells recognize and attach to cancer cells, they may have the ability to kill those cancer cells. The U.S. Food and Drug Administration (FDA) allows BEAM-201 to be used in research.Patients with T-cell Acute Lymphoblastic Leukemia (T-ALL) or T-cell Lymphoblastic Lymphoma (L-LL), where the disease has come back (relapsed) or has not responded to treatment (refractory) may be eligible for the study.

Use of elexacaftor-texacaftor-ivacaftor ("ETI"), also known as Trikafta, is associated with weight gain in most people with cystic fibrosis (CF) who take this medication. However, some people gain more or less weight than is expected, and this can be either beneficial or problematic based on the individual's starting weight. The goal of this study is to compare factors between children and young adults with CF who have either had robust or minimal weight gain after one year of treatment with ETI.This observational study requires one study visit to CHOP that will last approximately 6 hours.

For this study, we are studying the relationship between variations in the genes of the immune system called HLA and how they relate to:

1. The risk of developing nephrotic syndrome
2. The response to treatment of nephrotic syndrome and
3. The risk of recurrence of nephrotic syndrome following kidney transplantation.

1. Clinical diagnosis of nephrotic syndrome
2. Biopsy proven diagnosis of FSGS or MCD and,
3. History of living or deceased donor kidney transplantation

This study involves taking a study drug called venetoclax with or without standard of care chemotherapy. The overall goal of this study is to see if adding venetoclax to standard chemotherapy leads to improved survival for children and young adults with relapsed acute myeloid leukemia (AML). The study may last up to 9 years and will enroll male and female patients from the ages of 29 days to 21 years of age.