Duchenne Muscular DystrophyPhase 1NCT06241950
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
A Phase 1 study testing delandistrogene moxeparvovec and imlifidase in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 1 site in Spain.
Trial summary
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks. Read more about this study
- Who is it for?
- Ages 4 to 9 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- delandistrogene moxeparvovec and imlifidase
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSpain
- Study dates
- 29 Jan 2024to 10 Oct 2025
- Phase
- Phase 1
- 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 4 to 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Ambulatory per protocol specified criteria.
- Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
- Ability to cooperate with motor assessment testing.
- Has elevated rAAVrh74 antibody titers per protocol-specified requirements.
- A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein.
- Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
Who cannot join
- Previous treatment with imlifidase.
- Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for receiving the study drugs or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
- Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Note: Other inclusion or exclusion criteria could apply.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- delandistrogene moxeparvovec, imlifidase
- Keywords
- Gene-Delivery, DMD, Ambulatory, Pediatric, Duchenne, Pre-existing antibodies, rAAVrh74 Antibody, Seropositive, Seropositivity, Titer
- 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.
delandistrogene moxeparvovecGenetic
Also called SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Single IV infusion of delandistrogene moxeparvovec
imlifidaseBiological
Also called Idefirix
IV infusion of Imlifidase
| Study group | Role | What they receive |
|---|---|---|
| Delandistrogene Moxeparvovec after Imlifidase Infusion | Experimental | Participants may receive 1 or 2 doses of imlifidase, depending on rAAVrh74 antibody titer results. Then, based on their rAAVrh74 antibody titer, eligible participants may receive 1 dose of delandistrogene moxeparvovec infusion. Receives: Genetic: delandistrogene moxeparvovec; Biological: imlifidase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
Measured: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity
Measured: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF)
Measured: Baseline, Week 12
Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration
Measured: Week 12
Secondary outcomes (5)
Maximum Observed Plasma Concentration (Cmax) of Imlifidase
Measured: Up to Day 7
Total IgG in Serum After Imlifidase Administration
Measured: Up to Week 12
rAAVrh74 Antibody Titers After Imlifidase Administration
Measured: Up to Hour 120
Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration
Measured: Up to Day 7
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Measured: Up to Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2024
- Study start29 Jan 2024
- Primary completion10 Oct 2025
- Study completion10 Oct 2025
Why it stopped: Study is being terminated due to a business decision.
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 Sarepta Therapeutics, Inc.The 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Hansa Biopharma AB
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06241950
- Sponsor's protocol ID
- SRP-9001-104
- Other IDs
- 2022-003407-15
- Status last verified
- Nov 2025
- First submitted
- 26 Jan 2024
- Last updated
- 20 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74
- ClinicalTrials.gov ID
- NCT06241950
- Acronym / sponsor ID
- SRP-9001-104
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Gene-Delivery, DMD, Ambulatory, Pediatric, Duchenne, Pre-existing antibodies, rAAVrh74 Antibody, Seropositive
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study is being terminated due to a business decision.
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Hansa Biopharma AB
- Study start
- 29 Jan 2024 (actual)
- Primary completion
- 10 Oct 2025 (actual)
- Study completion
- 10 Oct 2025 (actual)
- First posted
- 5 Feb 2024 (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.
This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Delandistrogene Moxeparvovec after Imlifidase Infusion | Experimental | Genetic: delandistrogene moxeparvovec, Biological: imlifidaseParticipants may receive 1 or 2 doses of imlifidase, depending on rAAVrh74 antibody titer results. Then, based on their rAAVrh74 antibody titer, eligible participants may receive 1 dose of delandistrogene moxeparvovec infusion. |
Who may qualify
- Ages
- 4 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Ambulatory per protocol specified criteria.
- Has a definitive diagnosis of DMD prior to Screening based on documentation of clinical findings and confirmatory genetic testing.
- Ability to cooperate with motor assessment testing.
- Has elevated rAAVrh74 antibody titers per protocol-specified requirements.
- A pathogenic frameshift mutation, nonsense mutation or premature stop codon or pathogenic variant in the DMD gene that is expected to lead to absence of dystrophin protein.
Key exclusion criteria (5 listed)
- Previous treatment with imlifidase.
- Presence of any other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for receiving the study drugs or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
- Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
- Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
- Note: Other inclusion or exclusion criteria could apply.
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 |
|---|---|---|
| delandistrogene moxeparvovecAlso: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYSSingle IV infusion of delandistrogene moxeparvovec | Genetic | Delandistrogene Moxeparvovec after Imlifidase Infusion |
| imlifidaseAlso: IdefirixIV infusion of Imlifidase | Biological | Delandistrogene Moxeparvovec after Imlifidase Infusion |
Primary outcome
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content
Time frame: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity
Time frame: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF)
Time frame: Baseline, Week 12
Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration
Time frame: Week 12
5 secondary outcomes
Maximum Observed Plasma Concentration (Cmax) of Imlifidase
Time frame: Up to Day 7
Total IgG in Serum After Imlifidase Administration
Time frame: Up to Week 12
rAAVrh74 Antibody Titers After Imlifidase Administration
Time frame: Up to Hour 120
Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Serum, After Delandistrogene Moxeparvovec Administration
Time frame: Up to Day 7
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Time frame: Up to Week 104
Results and safety
The study was stopped early (reason given: Study is being terminated due to a business decision.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
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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.
Medical Director study director
Sarepta Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing delandistrogene moxeparvovec and imlifidase in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 1 site in Spain.
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 6 inclusion and 5 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 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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: Study is being terminated due to a business decision.). 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?
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression as Measured by Western Blot Adjusted by Muscle Content (time frame: Baseline, Week 12). The study lists 4 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 20 Nov 2025.