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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ML-004 for Adolescents and Adults With ASD

The purpose of this study is to test the safety of an investigational medicine called ML-004 and how it might affect communication, behavior, and other symptoms of autism spectrum disorder (ASD).  This medication is investigational because it has not been approved by the FDA for the treatment of symptoms associated with autism. 
You may qualify if you: 
  • Are 12 to 25 years old
  • Have an autism diagnosis
  • Have a designated care/study partner able to attend every visit and answer questions about the participant. This person does not need to be the participant's parent or guardian. This person should have significant contact with them and can support them for the duration of the study. 
  • Are able to swallow the study medication (tablet)

Approach Avoidance Model of Repetitive Behaviors

The goal of this research study is to learn about experiences with repetitive behaviors from autistic teens and their families. In order to learn about your experiences with repetitive behaviors, you will be invited to complete a set of online questionnaires about your (or your teen's) experiences. The goal of this study is to promote advocacy and transparency for autistic teens' lived experiences. A small subset of teens may also be asked to complete a brain MRI and additional behavioral assessments, if they are interested. Participating in the MRI and further behavioral assessments is voluntary and is not required to complete the online surveys.

Preventing Distracted Driving

The Center for Injury Research & Prevention is seeking teens age 16-19 and a parent to participate in a research study about distracted driving. Both teens and parents must be licensed drivers and use smartphones. The purpose of this research study is to figure out if a new smartphone app impacts distracted driving, and to learn parent and teen perspectives on the app. Participants will be asked to complete occasional surveys and use a smartphone app for up to 3 weeks. The entire study can be completed remotely, and no visits to CHOP are required. Families are compensated for their time in the study. 

ADHD ETA Driving Cohort Study

We are asking both parents and their child between ages 16-18 who recently received their learner’s permit to partner with us as we learn more about teen driving safety. We are looking for children both with and without an ADHD diagnosis to participate. First, parents will answer questions about themselves and their child. Then, both parents and their child will complete up to five surveys over the course of two to three years. The surveys will ask about your thoughts and experiences with driving, health, and other outcomes.

Drive Focus Study

We are asking teens with autism between the ages of 16-21 who have expressed interest in learning how to drive or have received their learner’s permit in the past 3 months, and their parent/primary caregiver to participate in an interview study. The purpose of this study is to inform the development of driver training technology for autistic individuals who are interested in learning to drive. Participants will be asked to complete one or two interviews where they are shown images of the app and/or asked to test it out on a study-provided tablet. Interviews will take approximately 60 minutes. Participants will receive compensation for participation.

ADHD ETA Naturalistic Driving Study

This study is open to teens ages 16-20 who are about to receive or recently received their driver’s license, and a parent. We are looking for teens both with and without an ADHD diagnosis. Participants must have access to a vehicle that the study team can install with data collection equipment and be residents of New Jersey or Pennsylvania. We are asking if families will partner with us as we learn more about the real-world driving behaviors of teen with and without ADHD. Families will receive compensation for their participation. 

TAPISTRY

The purpose of this study is to evaluate the effects, good or bad, of targeted therapies or immunotherapy (drugs that help the body's immune system fight cancer cells) in patients who have solid tumors with specific genetic alterations or with a high number of mutations. Patients will be assigned to cohorts (groups) based on having a specific type of genetic alteration in their tumor. The study includes cohorts for patients whose tumors have genetic alterations in the ROS1, NTRK, PIK3CA, BRAF, or RET genes or a high number of mutations in their tumor.

Hunter Syndrome JR-141 Study

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).

GM1 Gangliosidosis Gene Therapy Study

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. 

iTONE Study

iTONE will study the impact of a 6-month home exercise program on physical activity levels, skeletal muscle mass and strength, functional health status, and quality of life in children and teenagers with pulmonary hypertension.  Participants will receive home exercise prescriptions from the CHOP exercise trainers, tailored to their interests and abilities.  Participants will wear a smartwatch that tracks activity and communicates with a digital health platform connected to the study team. 


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