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.

Find a Clinical Research Study

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UX053 in GSD III

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a clinical drug trial that involves giving you a study drug called UX033. The FDA has not approved this drug for treatment of glycogen storage disease type III (GSD III) but has approved us to use this drug as part of this clinical trial.

The goal of the trial is to test the study drug safety, how your body processes the drug, and how the drug affects your body. You will be asked to participate in either Part 1 or Part 2 of the study:

  • Participation in Part 1 of this study will last for 3 months, will involve 10 study visits and you will receive one dose of UX053.
  • Participation in Part 2 of this study will last for up to 48 weeks, will involve 18 study visits and you will receive five doses of UX053 or a placebo.

It is possible that treatment with UX053 may improve your blood sugar levels. Travel costs will be covered by the study and you will receive compensation for participating.

PBKR03 for Krabbe Disease

Patients with Krabbe disease do not have enough of a certain protein called galactosylceramidase due to a defective gene. The lack of this protein causes damage to the patient’s nerve cells. In this study, we will give a normal, properly working copy of that gene to your child. The procedure for replacing a defective gene by a normal one in a patient is called gene therapy. If your child joins this study, the normal galactosylceramidase gene will be delivered once into his or her cells by using a virus that has been changed so that it no longer causes disease. The name of the treatment used in this study is PBKR03. PBKR03 will be injected with a needle into a space at the top of your child’s spine (called the cisterna magna). The purpose of this clinical research study is to learn more about PBKR03. It will test if treatment with PBKR03 is safe in children diagnosed with early infantile Krabbe disease.

TransPrEP Phase 3: 3T Intervention

Transcend, Triumph, and Thrive (3T) is a HIV prevention intervention study for transgender youth ages 15-24, looking at their use of Pre-exposure prophylaxis (PrEP) The purpose of this study is to pilot a program designed to address the health needs of transgender youth. 3T was designed based on focus group data from trans youth and will enroll 80 transfeminine, transmasculine and gender non-conforming/non-binary youth ages 15-24. Participants are enrolled for 12 months, starting with the 6-month intervention component.  

 The 6-month intervention is led by Community Health Workers (CHWs). Participants meet with their CHW to develop and implement a series of personalized goals related to their social, emotional, and physical health. Participants are asked to engage with online content via a study exclusive website. Topics include mental health, career/job skills, food and housing security, trauma, transitioning, and sexual and reproductive health. Each month, there is a forum with other participants and community member guest speakers focusing on the guiding principles of the study: being your authentic self​, persevering in the face of adversity, loving oneself, advocating for oneself​, and creating & contributing to the collective trans community.  Participants may come to as many or few of these forums as they like. Participants also complete assessments (online surveys) at their enrollment, 3-month, 6-month, 9-month, and 12-month visits, and HIV and STI testing at their enrollment, 6-month, and 12-month visits. Additionally, participants are offered PrEP, at no cost, during the study. Taking PrEP is completely voluntary, and they will not receive an extra incentive if they decide to take it.  

DTX401 Gene Therapy in Glycogen Storage Disease (GSD) Type Ia

Our team at the Congenital Hyperinsulinism Center at CHOP is working on a research study to determine whether an experimental gene therapy product, DTX401, is safe and effective in people with glycogen storage disease type Ia (GSDIa). The FDA has not approved this drug for the treatment of GSDIa but has approved us to use this drug as part of this clinical trial. The purpose of this research study is to see if DTX401 can help people with GSDIa have normal blood sugar levels and fewer episodes of hypoglycemia. DTX041 works by delivering copies of the G6PC gene to your liver through a one-time infusion. Participation in this study will last for 2 years and will involve 30 study visits. It is possible that treatment with the study drug infusion may improve your blood sugar levels. Travel costs will be covered by the study and you will receive compensation for participating.


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