A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Registration Number
- NCT05689164
- Lead Sponsor
- Pfizer
- Brief 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 6
- Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
- Investigator site staff directly involved in the study and their family members
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description All participants fordadistrogene movaparvovec All participants enrolled in the study.
- Primary Outcome Measures
Name Time Method Number of participants with malignancy adverse event At least annually from 5 through 10 years after dosing in the interventional study Number of participants with clinically significant findings in echocardiogram parameters Annually from 5 through 10 years after dosing in the interventional study Number of participants with serious adverse events At least annually from 5 through 10 years after dosing in the interventional study. Percentage of participants with serious adverse events At least annually from 5 through 10 years after dosing in the interventional study. Number of participants with clinically significant findings in electrocardiogram (ECG) assessments Annually from 5 through 10 years after dosing in the interventional study. Number of participants with clinically significant findings in cardiac troponin I laboratory examinations Annually from 5 through 10 years after dosing in the interventional study Number of participants with adverse events considered related to treatment At least annually from 5 through 10 years after dosing in the interventional study. Percentage of particpants with adverse events considered related to treatment At least annually from 5 through 10 years after dosing in the interventional study
- Secondary Outcome Measures
Name Time Method Change from pre-dose in the ability to walk 10 meters unassisted Annually from 5 through 10 years after dosing in the interventional study. Change from pre-dose in the ability to climb stairs. Annually from 5 through 10 years after dosing in the interventional study Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score Annually from 5 through 10 years after dosing in the interventional study Age at loss of ambulation From 5 through 10 years after dosing in the interventional study Cause of death From 5 through 10 years after dosing in the interventional study Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure Annually from 5 through 10 years after dosing in the interventional study Applicable to non-ambulatory participants only
Age at death From 5 through 10 years after dosing in the interventional study Glucocorticoid use dose and frequency At least annually from 5 through 10 years after dosing in the interventional study Change from pre-dose in the North Star Ambulatory Assessment total score Annually from 5 through 10 years after dosing in the interventional study Applicable to a sub-set of participants only
Change from pre-dose in percent of predicted forced vital capacity (%pFVC) and percent predicted peak expiratory flow (%pPEF) Annually from 5 through 10 years after dosing in the interventional study Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram Annually from 5 through 10 years after dosing in the interventional study Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument Annually from 5 through 10 years after dosing in the interventional study Age when percent predicted forced vital capacity <30% Annually from 5 through 10 years after dosing in the interventional study
Trial Locations
- Locations (12)
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