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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-13

Study Completion Date

2025-09-24

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.

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Detailed Description

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Conditions

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Duchenne Muscular Dystrophy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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All participants

All participants enrolled in the study.

Group Type OTHER

fordadistrogene movaparvovec

Intervention Type BIOLOGICAL

gene therapy administered in a previous study.

Interventions

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fordadistrogene movaparvovec

gene therapy administered in a previous study.

Intervention Type BIOLOGICAL

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

Minimum Eligible Age

0 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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