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

a study on Duchenne Muscular Dystrophy

Summary

Eligibility
for males ages 0 years and up (full criteria)
Location
at UCLA
Dates
study started
completion around

Description

Summary

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.

Official Title

Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study

Keywords

Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy, Muscular Dystrophies, fordadistrogene movaparvovec

Eligibility

You can join if…

Open to males ages 0 years and up

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

You CAN'T join if...

  • Investigator site staff directly involved in the study and their family members

Locations

  • Reed Neurological Research Center accepting new patients
    Los Angeles California 90095 United States
  • UCLA Children's Heart Center accepting new patients
    Los Angeles California 90095 United States
  • UCLA Clinical Lab Services accepting new patients
    Los Angeles California 90095 United States
  • UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center) accepting new patients
    Los Angeles California 90095 United States
  • UCLA Medical Center accepting new patients
    Los Angeles California 90095 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT05689164
Phase
Phase 3 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 250 study participants
Last Updated