Duchenne Muscular DystrophyPhase 4ENHANCENCT07542314
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
A Phase 4 study testing ELEVIDYS, Sirolimus and Glucocorticoids in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 1 site in United States.
Trial summary
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent. Read more about this study
- Who is it for?
- Ages 4 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- ELEVIDYS, Sirolimus and Glucocorticoids
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Oct 2026to 31 Aug 2027
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.
- Cohort 1 only: Is eligible for commercial ELEVIDYS.
- Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.
- Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive).
- Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements.
- Either has a parent or legal guardian who is willing to provide informed consent, or is ≥ 18 years of age and able to provide informed consent independently.
Who cannot join
- Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).
- Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
- Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:
- The participant's ability to comply with the protocol-required procedures, and/or
- The participant's well-being or safety, and/or
- The clinical interpretability of the data collected from the participant
Show all 11Show fewer
- Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
- Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.
- Cohort 1 only: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients.
- Cohort 1 only: Any wounds or recent injuries that, in the opinion of the Investigator, would be at risk of dehiscence or impaired healing in the setting of sirolimus administration.
- Other inclusion/exclusion criteria may apply, per protocol.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4, contact
United States1 site, 1 recruiting
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030
About this study
Official title: Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics
- Keywords
- Duchenne Muscular Dystrophy, DMD, ELEVIDYS, Delandistrogene moxeparvovec-rokl, SRP-9001
- 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.
ELEVIDYSDrug
Also called Delandistrogene moxeparvovec-rokl, SRP-9001
Administered via an intravenous infusion.
SirolimusDrug
Administered orally.
GlucocorticoidsDrug
Administered orally.
AntibioticsDrug
Administered orally.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: ELEVIDYS | Other | Participants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally. Receives: Drug: ELEVIDYS; Drug: Sirolimus; Drug: Glucocorticoids; Drug: Antibiotics |
| Cohort 2: ELEVIDYS | No intervention | Participants who have previously received ELEVIDYS in a commercial setting after prophylactic treatment with sirolimus and corticosteroids will participate in one study visit during which a muscle biopsy will be performed. No study treatment will be administered in this cohort. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1: Number of Participants with ALI
Measured: 12 weeks
Secondary outcomes (9)
Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs)
Measured: Day 1 up to Week 24
Cohort 1: Number of Participants with Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Interstitial Lung Disease/Non-infectious Pneumonitis
Measured: Day 1 up to Week 24
Cohort 1: Number of Participants with Hepatic Adverse Events, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Measured: Day 1 up to Week 24
Cohort 1: Number of Participants with Severe ALI
Measured: Day 1 up to Week 24
Cohort 1: Number of Participants with ALI
Measured: Day 1 up to Week 24
Cohort 1: Duration of ALI
Measured: Day 1 up to Week 24
Cohort 1: Amount of Steroid Use
Measured: Day 1 up to Week 24
Cohort 1: Duration of Steroid Use
Measured: Day 1 up to Week 24
Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Apr 2026
- Study start30 Oct 2026 (estimated)
- Primary completion31 Aug 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07542314
- Sponsor's protocol ID
- SRP-9001-402
- Acronym
- ENHANCE
- Status last verified
- Oct 2026
- First submitted
- 2 Apr 2026
- Last updated
- 6 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 4 Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Patients With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting (ENHANCE)
- ClinicalTrials.gov ID
- NCT07542314
- Acronym / sponsor ID
- ENHANCE / SRP-9001-402
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, DMD, ELEVIDYS, Delandistrogene moxeparvovec-rokl, SRP-9001
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Oct 2026 (estimated)
- Primary completion
- 31 Aug 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 21 Apr 2026 (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 primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: ELEVIDYS | Other | Drug: ELEVIDYS, Drug: Sirolimus, Drug: Glucocorticoids, Drug: AntibioticsParticipants will receive ELEVIDYS in a commercial setting on Day 1. Participants will also receive sirolimus, glucocorticoids and antibiotics orally. |
| Cohort 2: ELEVIDYS | No intervention | –Participants who have previously received ELEVIDYS in a commercial setting after prophylactic treatment with sirolimus and corticosteroids will participate in one study visit during which a muscle biopsy will be performed. No study treatment will be administered in this cohort. |
Who may qualify
- Ages
- 4 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Cohort 1 only: Is male at birth, ambulatory, and ≥ 4 years of age at the time of dosing.
- Cohort 1 only: Is eligible for commercial ELEVIDYS.
- Cohort 2 only: Is male at birth and has previously received ELEVIDYS in a commercial setting after pre-treatment with sirolimus and corticosteroids.
- Cohort 1 only: Participants who are sexually active must agree to use, for the entire duration of the study, a condom and the female sexual partner must also use a medically acceptable form of birth control (eg, oral contraceptive).
- Has (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with the study visit schedule and all other protocol requirements, or is ≥ 18 years of age and personally able to understand and comply with the protocol requirements.
Key exclusion criteria (11 listed)
- Cohort 1 only: Contraindicated to receive ELEVIDYS per the United States Package Insert (USPI).
- Cohort 1 only: Has serological evidence of current, chronic, or active human immunodeficiency virus, hepatitis C, or hepatitis B infection.
- Has a medical condition or confounding circumstances (eg, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:
- Cohort 1 only: Has a symptomatic infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks prior to Day 1.
- Cohort 1 only: Has received a live virus vaccine within 4 weeks or inactive vaccine within 2 weeks of the Day 1 visit or expects to receive a vaccination during the first 3 months after Day 1.
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 |
|---|---|---|
| ELEVIDYSAlso: Delandistrogene moxeparvovec-rokl, SRP-9001Administered via an intravenous infusion. | Drug | Cohort 1: ELEVIDYS |
| SirolimusAdministered orally. | Drug | Cohort 1: ELEVIDYS |
| GlucocorticoidsAdministered orally. | Drug | Cohort 1: ELEVIDYS |
| AntibioticsAdministered orally. | Drug | Cohort 1: ELEVIDYS |
Primary outcome
Cohort 1: Number of Participants with ALI
Time frame: 12 weeks
9 secondary outcomes
Cohort 1: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Interstitial Lung Disease/Non-infectious Pneumonitis
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Hepatic Adverse Events, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with Severe ALI
Time frame: Day 1 up to Week 24
Cohort 1: Number of Participants with ALI
Time frame: Day 1 up to Week 24
Cohort 1: Duration of ALI
Time frame: Day 1 up to Week 24
Cohort 1: Amount of Steroid Use
Time frame: Day 1 up to Week 24
Cohort 1: Duration of Steroid Use
Time frame: Day 1 up to Week 24
Cohort 1: Quantity of ELEVIDYS Dystrophin Protein Expression as Measured by Western Blot
Time frame: Week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2026.
Related trials for Duchenne Muscular Dystrophy
Enrolling by invitationObservational study
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
RecruitingObservational study
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Duchenne Muscular Dystrophy (DMD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
RecruitingPhase 1/2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
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.
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4, contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing ELEVIDYS, Sirolimus and Glucocorticoids in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 1 site 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 6 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 6 Oct 2026. 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 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4, (1-888-SAREPTA (1-888-727-3782), SareptAlly@sarepta.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cohort 1: Number of Participants with ALI (time frame: 12 weeks). 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 6 Oct 2026.