Clinical Research Study Finder

Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling. You can also look for studies that offer remote participation from home!

Find a Clinical Research Study

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Multimodal Imaging in Autism

The purpose of this study is to understand how children’s brains process sounds and language. Children with Autism Spectrum Disorder (ASD), Intellectual and Developmental Delay (IDD), or Developmental Language Disorder (DLD) often have trouble understanding sounds that are too close together compared to Typically Developing Controls (children without ASD, IDD, or DLD). Fast changes in speech can be hard for children. This may be awkward during conversations and difficult in social situations. This research will see if trouble with processing sounds contributes to language difficulties in children with ASD, IDD, and/or DLD.

BEAM-201

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. 


Weight Trajectory with ETI

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. 

Genomics of Nephrotic Syndrome

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.
Participants will be asked to provide a one time blood sample for DNA testing and information about their health will be collected. 

To be eligible, participants must have:
  1. Clinical diagnosis of nephrotic syndrome
  2. Biopsy proven diagnosis of FSGS or MCD and,
  3. History of living or deceased donor kidney transplantation

Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Relapsed AML

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. 

MAS825 Trial for NLRC4-GOF

The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF). Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood and stool testing, physical exam, an electrocardiogram (ECG), questionnaire completion, and administration of study drug. Participation will last for up to 4 years. 

MRI Assessment of NAD+

NAD+ (nicotinamide adenine dinucleotide) is an important substance found in the body that plays a role in energy metabolism and overall health. The purpose of this research study is to test the performance of a new magnetic resonance imaging (MRI) technique (spectroscopy) to measure NAD+ metabolism in brain and skeletal muscle. MRI provides pictures of the inside of the body and information about chemicals the body makes.
We are seeking to enroll males and females with Friedrich’s Ataxia (FA) as well as healthy matched volunteers, 18 years of age and above. Pregnant females excluded for safety purposes.

22LL011; Epcoritamab in B-cell Neoplasms

CHOP in collaboration with AbbVie, is conducting a study for pediatric patients from 1 year of age to 25 years of age with relapsed or refractory aggressive mature B-cell neoplasms (including diffuse large B-cell lymphoma, Burkitt and Burkitt-like lymphoma/leukemia). In this clinical trial, participants will receive the study drug epcoritamab, an immune therapy. Epcoritamab is in clinical development and is being studied for an unapproved use in pediatric patients with B-cell neoplasms. It is not approved for use by the FDA or other global regulatory health authorities. Safety and efficacy are under evaluation. For more information about this study, including specific eligibility criteria, please visit clincialtrials.gov and search for “NCT05206357”. To hear more about this and other available trials or get any questions answered please contact our Cancer Intake Specialist by phone at 267-426-0762 or email Oncointake@chop.edu. 

SLEEPY 2.0

The goal of this study is to find out if we can develop a digital health approach that can increase sleep duration and improve growth outcomes in children.


PRISM and CFN for PMD

Primary mitochondrial diseases (PMD) are a group of energy deficiency disorders that are typically progressive and range in onset and severity. Individuals with PMD experience a variety of clinical symptoms that not only affect their physical health but significantly impact their daily life.

The goal of this study is to find the best way to help adults with PMD deal with the stress of their condition, and to help them be better able to “bounce back,” or be resilient. In order to do this, we are going to test two interventions. The first is called Promoting Resilience in Stress Management (PRISM). The second is called clinical-focused narrative (CFN) intervention.

The study period will last for about one month and will consist of five online visits and a one-time online discussion group.


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