NCT05689164TerminatedPhase 3
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
Source: ClinicalTrials.gov · Last updated 21 Oct 2025
Reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
Brief summary (registry, in English)
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.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 7 (actual)
- First submitted
- 9 Jan 2023
- Start date
- 13 Mar 2023
- Primary completion date
- 24 Sept 2025
- Lead sponsor
- Pfizer
- Diseases
- Duchenne muscular dystrophy
- NCT number
- NCT05689164
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Investigator site staff directly involved in the study and their family members
Who the study is looking for
- Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
Age in the registry: 0 years to - · Sex: Male · Does not accept healthy volunteers
Sites (12)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Duke Lenox Baker Children's | Durham | United States | Unknown status |
| Duke Children's Health Center | Durham | United States | Unknown status |
| UCLA Children's Heart Center | Los Angeles | United States | Unknown status |
| UCLA Medical Center | Los Angeles | United States | Unknown status |
| Reed Neurological Research Center | Los Angeles | United States | Unknown status |
| UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center) | Los Angeles | United States | Unknown status |
| UCLA Clinical Lab Services | Los Angeles | United States | Unknown status |
| University of Utah Hospital | Salt Lake City | United States | Unknown status |
| University of Utah Imaging and Neurosciences Center | Salt Lake City | United States | Unknown status |
| University of Utah - Craig H. Neilsen Rehabilitation Hospital | Salt Lake City | United States | Unknown status |
| University of Utah Clinical Neurosciences Center | Salt Lake City | United States | Unknown status |
| Primary Children's Hospital | Salt Lake City | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.