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

October 18, 2025 updated by: Pfizer

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
7

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Medical Center
      • Los Angeles, California, United States, 90095
        • Reed Neurological Research Center
      • Los Angeles, California, United States, 90095
        • UCLA Children's Heart Center
      • Los Angeles, California, United States, 90095
        • UCLA Clinical Lab Services
      • Los Angeles, California, United States, 90095
        • UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • Duke Lenox Baker Children's
      • Durham, North Carolina, United States, 27710
        • Duke Children's Health Center
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Primary Children's Hospital
      • Salt Lake City, Utah, United States, 84108
        • University of Utah Imaging and Neurosciences Center
      • Salt Lake City, Utah, United States, 84112
        • University of Utah Hospital
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Clinical Neurosciences Center
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Craig H. Neilsen Rehabilitation Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 second and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Other: All participants
All participants enrolled in the study.
Biological: fordadistrogene movaparvovec
gene therapy administered in a previous study.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Number of participants with serious adverse events
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study.
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.
At least 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.
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
At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with malignancy adverse event
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study
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.
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
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
Annually from 5 through 10 years after dosing in the interventional study

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change from pre-dose in the ability to walk 10 meters unassisted
Time Frame: Annually from 5 through 10 years after dosing in the interventional study.
Annually from 5 through 10 years after dosing in the interventional study.
Change from pre-dose in the ability to climb stairs.
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
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
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the North Star Ambulatory Assessment total score
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
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)
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
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
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
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%
Time Frame: Annually from 5 through 10 years after dosing in the interventional study
Annually from 5 through 10 years after dosing in the interventional study
Age at loss of ambulation
Time Frame: From 5 through 10 years after dosing in the interventional study
From 5 through 10 years after dosing in the interventional study
Age at death
Time Frame: From 5 through 10 years after dosing in the interventional study
From 5 through 10 years after dosing in the interventional study
Cause of death
Time Frame: From 5 through 10 years after dosing in the interventional study
From 5 through 10 years after dosing in the interventional study
Glucocorticoid use dose and frequency
Time Frame: At least annually from 5 through 10 years after dosing in the interventional study
At least annually from 5 through 10 years after dosing in the interventional study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Pfizer

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Pfizer CT.gov Call Center, Pfizer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 13, 2023

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 24, 2025

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 24, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 9, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 9, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 19, 2023

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 21, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 18, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • C3391011
  • NCT05689164 (Registry Identifier: ClinicalTrials.gov)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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