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

Pfizer

Status and phase: Active, not recruiting; Phase 3

Conditions: Duchenne Muscular Dystrophy

Treatments: Biological: fordadistrogene movaparvovec

Study type: Interventional

Funder types: Industry

Identifiers

NCT05689164

C3391011

NCT05689164 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: Male

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

6 participants in 1 patient group

All participants

Other group

Description:

All participants enrolled in the study.

Treatment:

Biological: fordadistrogene movaparvovec