Muscular Dystrophy, DuchennePhase 2NCT05429372
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
A Phase 2 study testing PF-06939926 in people with Muscular Dystrophy, Duchenne, run by Pfizer at 13 sites in 2 countries.
Trial summary
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study Read more about this study
- Who is it for?
- Ages 2 to 3 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- PF-06939926
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 8 Aug 2022to 3 Oct 2025
- Phase
- Phase 2
- 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 2 to 3 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Confirmed diagnosis of DMD by prior genetic testing.
Who cannot join
- Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
- Positive test performed by Pfizer for neutralizing antibodies to AAV9.
- Any prior treatment with gene therapy.
- Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
- Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.
- Abnormality in specified laboratory tests, including blood counts, liver and kidney function.
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States10 sites
UF Health Shands Hospital
Gainesville, Florida, 32610
University of Florida
Gainesville, Florida, 32610
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146
CTSI Clinical Research Center
Salt Lake City, Utah, 84108
University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108
University of Utah Hospital & Clinics Investigational Drug Services
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 Hospital
Salt Lake City, Utah, 84132
Australia3 sites
Perth Children's Hospital
Nedlands, Western Australia, 6009
The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
About this study
Official title: A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
Read the detailed description
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- PF-06939926
- Keywords
- Early Stage Duchenne Muscular Dystrophy, DMD, gene therapy, fordadistrogene movaparvovec
- 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.
PF-06939926Genetic
Also called Fordadistrogene Movaparvovec
All participants will receive a single dose of PF-06939926 on Day 1.
| Study group | Role | What they receive |
|---|---|---|
| PF-06939926 | Experimental | Not described Receives: Genetic: PF-06939926 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Measured: Through Week 52
Number of participants with abnormal hematology test results
Blood samples will be collected from subjects for the analysis of hematology
Measured: Through Week 52
Number of participants with abnormal biochemistry test results
Blood samples will be collected from subjects for the analysis of biochemistry
Measured: Through Week 52
Number of participants with abnormal urine analysis
Urine samples will be collected from subjects for the analysis of urine
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes in neurological examinations
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes in body weight
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes in vital signs
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Measured: Through Week 52
Number of participants with abnormal and clinically relevant changes on echocardiogram
Measured: Through Week 52
Secondary outcomes (12)
Distribution of mini-dystrophin expression in muscle
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
Measured: At Week 9, Week 52 and Year 5 (if available)
Level of mini-dystrophin expression in muscle
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
Measured: At Week 9, Week 52 and Year 5 (if available)
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Measured: Through 5 years
Number of participants with abnormal hematology test results
Blood samples will be collected from subjects for the analysis of hematology
Measured: Through 5 years
Number of participants with abnormal biochemistry test results
Blood samples will be collected from subjects for the analysis of biochemistry
Measured: Through 5 years
Number of participants with abnormal urine analysis
Urine samples will be collected from subjects for the analysis of urine
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes in neurological examinations
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes in body weight
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes in vital signs
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Measured: Through 5 years
Number of participants with abnormal and clinically relevant changes on echocardiogram
Measured: Through 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2022
- Study start8 Aug 2022
- Primary completion3 Oct 2025
- Study completion3 Oct 2025
Why it stopped: All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, DuchenneStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 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
- NCT05429372
- Sponsor's protocol ID
- C3391008
- Other IDs
- 2021-003379-33
- Status last verified
- Oct 2025
- First submitted
- 8 Oct 2021
- 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
- A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
- ClinicalTrials.gov ID
- NCT05429372
- Acronym / sponsor ID
- C3391008
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Early Stage Duchenne Muscular Dystrophy, DMD, gene therapy, fordadistrogene movaparvovec
- Phase
- Phase 2
- 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 one combined study: C3391003
- Enrolment
- 10 participants (actual)
- Lead sponsor
- PfizerIndustry (pharma, biotech, devices)
- Study start
- 8 Aug 2022 (actual)
- Primary completion
- 3 Oct 2025 (actual)
- Study completion
- 3 Oct 2025 (actual)
- First posted
- 23 Jun 2022 (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 study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
Detailed description
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PF-06939926 | Experimental | Genetic: PF-06939926 |
Who may qualify
- Ages
- 2 to 3 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Confirmed diagnosis of DMD by prior genetic testing.
Key exclusion criteria (6 listed)
- Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
- Positive test performed by Pfizer for neutralizing antibodies to AAV9.
- Any prior treatment with gene therapy.
- Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
- Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.
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 |
|---|---|---|
| PF-06939926Also: Fordadistrogene MovaparvovecAll participants will receive a single dose of PF-06939926 on Day 1. | Genetic | PF-06939926 |
Primary outcome
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Time frame: Through Week 52
Number of participants with abnormal hematology test results
Time frame: Through Week 52
Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal biochemistry test results
Time frame: Through Week 52
Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal urine analysis
Time frame: Through Week 52
Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in neurological examinations
Time frame: Through Week 52
Number of participants with abnormal and clinically relevant changes in body weight
Time frame: Through Week 52
Number of participants with abnormal and clinically relevant changes in vital signs
Time frame: Through Week 52
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Time frame: Through Week 52
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Time frame: Through Week 52
Number of participants with abnormal and clinically relevant changes on echocardiogram
Time frame: Through Week 52
12 secondary outcomes
Distribution of mini-dystrophin expression in muscle
Time frame: At Week 9, Week 52 and Year 5 (if available)
Level of mini-dystrophin expression in muscle
Time frame: At Week 9, Week 52 and Year 5 (if available)
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Time frame: Through 5 years
Number of participants with abnormal hematology test results
Time frame: Through 5 years
Number of participants with abnormal biochemistry test results
Time frame: Through 5 years
Number of participants with abnormal urine analysis
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes in neurological examinations
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes in body weight
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes in vital signs
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Time frame: Through 5 years
Number of participants with abnormal and clinically relevant changes on echocardiogram
Time frame: Through 5 years
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 one 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.
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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
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PF-06939926 in people with Muscular Dystrophy, Duchenne, run by Pfizer at 13 sites in 2 countries.
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 6 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 13 sites in United States, Australia. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 one 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?
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events (time frame: Through Week 52). The study lists 10 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.