A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Stopped early · Phase 3
Conditions studied: Duchenne Muscular Dystrophy
In brief
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
Key facts
- Study ID
- NCT05689164
- Run by
- Pfizer
- People needed
- 7
- Starts
- 2023-03-13
- Expected to finish
- 2025-09-24
- Last updated by the study team
- 2025-10-21
Who can join
Age: any. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
You may not qualify if…
- Investigator site staff directly involved in the study and their family members
Where it is running
- Reed Neurological Research Center — Los Angeles, California, United States
- UCLA Children's Heart Center — Los Angeles, California, United States
- UCLA Clinical Lab Services — Los Angeles, California, United States
- UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center) — Los Angeles, California, United States
- UCLA Medical Center — Los Angeles, California, United States
- Duke Lenox Baker Children's — Durham, North Carolina, United States
- Duke Children's Health Center — Durham, North Carolina, United States
- University of Utah Imaging and Neurosciences Center — Salt Lake City, Utah, United States
- University of Utah Hospital — Salt Lake City, Utah, United States
- Primary Children's Hospital — Salt Lake City, Utah, United States
- University of Utah Clinical Neurosciences Center — Salt Lake City, Utah, United States
- University of Utah Craig H. Neilsen Rehabilitation Hospital — Salt Lake City, Utah, United States
Full record on ClinicalTrials.gov
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