Duchenne Muscular DystrophyPhase 3NCT05689164
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
A Phase 3 study testing fordadistrogene movaparvovec in people with Duchenne Muscular Dystrophy, run by Pfizer at 12 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- fordadistrogene movaparvovec
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 12 sitesUnited States
- Study dates
- 13 Mar 2023to 24 Sep 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
Who cannot join
- Investigator site staff directly involved in the study and their family members
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
United States12 sites
Duke Lenox Baker Children's
Durham, North Carolina, 27705
Duke Children's Health Center
Durham, North Carolina, 27710
Reed Neurological Research Center
Los Angeles, California, 90095
UCLA Children's Heart Center
Los Angeles, California, 90095
UCLA Clinical Lab Services
Los Angeles, California, 90095
UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)
Los Angeles, California, 90095
UCLA Medical Center
Los Angeles, California, 90095
University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108
University of Utah Hospital
Salt Lake City, Utah, 84112
Primary Children's Hospital
Salt Lake City, Utah, 84113
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132
University of Utah Craig H. Neilsen Rehabilitation Hospital
Salt Lake City, Utah, 84132
About this study
Official title: 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- fordadistrogene movaparvovec
- Keywords
- Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
fordadistrogene movaparvovecBiological
gene therapy administered in a previous study.
| Study group | Role | What they receive |
|---|---|---|
| All participants | Other | All participants enrolled in the study. Receives: Biological: fordadistrogene movaparvovec |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with serious adverse events
Measured: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of participants with serious adverse events
Measured: At least annually from 5 through 10 years after dosing in the interventional study.
Number of participants with adverse events considered related to treatment
Measured: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of particpants with adverse events considered related to treatment
Measured: At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with malignancy adverse event
Measured: 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
Measured: 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
Measured: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in echocardiogram parameters
Measured: 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
Measured: Annually from 5 through 10 years after dosing in the interventional study.
Change from pre-dose in the ability to climb stairs.
Measured: 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
Measured: 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
Measured: 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)
Measured: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram
Measured: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument
Measured: 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
Measured: Annually from 5 through 10 years after dosing in the interventional study
Age when percent predicted forced vital capacity <30%
Measured: Annually from 5 through 10 years after dosing in the interventional study
Age at loss of ambulation
Measured: From 5 through 10 years after dosing in the interventional study
Age at death
Measured: From 5 through 10 years after dosing in the interventional study
Cause of death
Measured: From 5 through 10 years after dosing in the interventional study
Glucocorticoid use dose and frequency
Measured: At least annually from 5 through 10 years after dosing in the interventional study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2023
- Study start13 Mar 2023
- Primary completion24 Sep 2025
- Study completion24 Sep 2025
Why it 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
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by PfizerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pfizer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pfizer CT.gov Call Center, Study director, Pfizer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05689164
- Sponsor's protocol ID
- C3391011
- Other IDs
- NCT05689164
- Status last verified
- Oct 2025
- First submitted
- 9 Jan 2023
- Last updated
- 21 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 21 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
- ClinicalTrials.gov ID
- NCT05689164
- Acronym / sponsor ID
- C3391011
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason 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
- Enrolment
- 7 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 13 Mar 2023 (actual)
- Primary completion
- 24 Sep 2025 (actual)
- Study completion
- 24 Sep 2025 (actual)
- First posted
- 19 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All participants | Other | Biological: fordadistrogene movaparvovecAll participants enrolled in the study. |
Who may qualify
- Ages
- 0 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
Key exclusion criteria (1 listed)
- Investigator site staff directly involved in the study and their family members
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| fordadistrogene movaparvovecgene therapy administered in a previous study. | Biological | All participants |
Primary outcome
Number of participants with serious adverse events
Time frame: 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.
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.
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
Number of participants with malignancy adverse event
Time frame: 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.
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
Number of participants with clinically significant findings in echocardiogram parameters
Time frame: Annually from 5 through 10 years after dosing in the interventional study
13 secondary outcomes
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.
Change from pre-dose in the ability to climb stairs.
Time frame: 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
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
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
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
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
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
Age when percent predicted forced vital capacity <30%
Time frame: 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
Age at death
Time frame: 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
Glucocorticoid use dose and frequency
Time frame: At least annually from 5 through 10 years after dosing in the interventional study
Results and safety
The study was stopped early (reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2025.
- To obtain contact information for a study center near you, click here.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
Related trials for Duchenne Muscular Dystrophy
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Active, not recruitingObservational study
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Active, not recruitingPhase 1
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pfizer CT.gov Call Center study director
Pfizer
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing fordadistrogene movaparvovec in people with Duchenne Muscular Dystrophy, run by Pfizer at 12 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 21 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with serious adverse events (time frame: At least annually from 5 through 10 years after dosing in the interventional study.). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2025.