NCT05689164|TerminatedPhase 3DMCFDA Drug

Study Stopped

All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003

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

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

Pfizer ClinicalTrials.gov

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2 other identifiers

C3391011NCT05689164

Study Type

interventional

Target

Locations

1 country

Sites

Timeline

RegisteredJan 2023

StartedMar 2023

CompletionSep 2025

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.

Trial Health

Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Mar 2023

Typical duration for phase_3

Geographic Reach

1 country

12 active sites

Status

terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.5 years study duration

First Submitted

Initial submission to the registry

January 9, 2023

Completed

10 days until next milestone

First Posted

Study publicly available on registry

January 19, 2023

Completed

2 months until next milestone

Study Start

First participant enrolled

March 13, 2023

Completed

2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 24, 2025

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 24, 2025

Completed

Last Updated

October 21, 2025

Status Verified

October 1, 2025

Enrollment Period

2.5 years

First QC Date

January 9, 2023

Last Update Submit

October 18, 2025

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneGenetic TherapyGene Therapy

Outcome Measures

Primary Outcomes (8)

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 Outcomes (13)

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
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
+8 more secondary outcomes

Study Arms (1)

All participants

OTHER

All participants enrolled in the study.

Biological: fordadistrogene movaparvovec

Interventions

fordadistrogene movaparvovecBIOLOGICAL

gene therapy administered in a previous study.

All participants

Eligibility Criteria

Age0 Years+

Sexmale(Gender-based eligibility)

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.

You may not qualify if:

Investigator site staff directly involved in the study and their family members

Contact the study team to confirm eligibility.