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A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

NCT05689164

TerminatedPhase 3

Sponsor: Pfizer

Conditions: Duchenne Muscular Dystrophy

Interventions: fordadistrogene movaparvovec

Countries: United States

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.

Eligibility overview

Sex: MALE

Age: 0 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.

Exclusion Criteria:

* Investigator site staff directly involved in the study and their family members
Locations (12)
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Los Angeles, California, United States
  • Durham, North Carolina, United States
  • Durham, North Carolina, United States
  • Salt Lake City, Utah, United States
  • Salt Lake City, Utah, United States
  • Salt Lake City, Utah, United States
  • Salt Lake City, Utah, United States
  • Salt Lake City, Utah, United States