Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
Overview
- Phase
- Phase 3
- Intervention
- fordadistrogene movaparvovec
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Pfizer
- Enrollment
- 7
- Locations
- 12
- Primary Endpoint
- Number of participants with malignancy adverse event
- Status
- Terminated
- Last Updated
- 6 months ago
Overview
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.
Investigators
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
Arms & Interventions
All participants
All participants enrolled in the study.
Intervention: fordadistrogene movaparvovec
Outcomes
Primary Outcomes
Number of participants with malignancy adverse event
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in echocardiogram parameters
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with serious adverse events
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of participants with serious adverse events
Time Frame: 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
Time Frame: 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
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with adverse events considered related to treatment
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of particpants with adverse events considered related to treatment
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study
Secondary Outcomes
- 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)
- 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)
- 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)
- 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)