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

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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Biological: fordadistrogene movaparvovec


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 0 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: All participants<br>All participants enrolled in the study. | Biological: fordadistrogene movaparvovec<br>* gene therapy administered in a previous study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 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 | 
| 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 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 clinically significant findings in echocardiogram parameters |  | Annually from 5 through 10 years after dosing in the interventional study | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 the North Star Ambulatory Assessment total score | Applicable to a sub-set of participants only | 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 | 
| Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure | Applicable to non-ambulatory participants only | 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 | 
| Age at loss of ambulation |  | 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 | 
| Cause of 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy