Limb-girdle Muscular DystrophyPhase 3EMERGENENCT06246513
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
A Phase 3 study testing SRP-9003 and Glucocorticoid in people with Limb-girdle Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 10 sites in 6 countries.
Trial summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2). Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Limb-girdle Muscular Dystrophy
- What is being tested?
- SRP-9003 and Glucocorticoid
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 10 sitesin 6 countries
- Study dates
- 15 Jan 2024to 30 Nov 2029
- Phase
- Phase 3
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Cohort 1, only ambulatory participants:
- Able to walk without assistive aid
- 10MWR <30 seconds
- NSAD ≥25
- Cohort 2, only non-ambulatory participants:
- 10MWR ≥30 seconds or unable to perform
Show all 10Show fewer
- PUL 2.0 entry scale score ≥3
- Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
- Able to cooperate with muscle testing
- Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
Who cannot join
- Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
- Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
- Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
- Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)
- Other inclusion/exclusion criteria apply.
Trial locations and contacts
10 sites in 6 countries.
10 sites in 6 countries
United States4 sites
Nationwide Childrens Hospital
Columbus, Ohio, 43205
University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla, California, 92037
Children's Hospital of The King's Daughter
Norfolk, Virginia, 23507
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Belgium2 sites
NMRC Gent (UZ Gent)
Ghent, 9000
University Hospital Leuven (UZ Leuven)
Leuven, Vlaams Brabant, 3000
Germany1 site
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen, North Rhine-Westphalia, 45147
Italy1 site
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, 20122
About this study
Official title: A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
- Conditions
- Limb-girdle Muscular Dystrophy
- Being tested
- SRP-9003, Glucocorticoid
- Keywords
- Limb Girdle Muscular Dystrophy Type 2E/R4, LGMD2E/R4, Pediatric, SRP-9003, scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec, Non-ambulatory, Ambulatory
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRP-9003Biological
Also called scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec
Solution for single IV infusion
GlucocorticoidDrug
Also called Prednisone or equivalent
Oral tablet (prophylactic)
| Study group | Role | What they receive |
|---|---|---|
| SRP-9003 | Experimental | Participants will receive a single intravenous (IV) infusion of SRP-9003. Receives: Biological: SRP-9003; Drug: Glucocorticoid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Measured: Baseline, Day 60
Secondary outcomes (13)
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay
Measured: Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)
Measured: Baseline, Day 60
Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)
Measured: Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score
Measured: Baseline through Month 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score
Measured: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test
Measured: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test
Measured: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test
Measured: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test
Measured: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test
Measured: Baseline through Month 60
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
Measured: Baseline through Month 60
Change From Baseline Through Month 60 in Creatine Kinase Level
Measured: Baseline through Month 60
Time to Change of Loss of Ambulation
Measured: Baseline through Month 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2024
- Study start15 Jan 2024
- Primary completion4 Mar 2025
- Study completion30 Nov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb-girdle 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06246513
- Sponsor's protocol ID
- SRP-9003-301
- Other IDs
- 2022-503112-17-00
- Acronym
- EMERGENE
- Status last verified
- Feb 2026
- First submitted
- 30 Jan 2024
- Last updated
- 2 Mar 2026
- 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 2 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4
- ClinicalTrials.gov ID
- NCT06246513
- Acronym / sponsor ID
- EMERGENE / SRP-9003-301
- Conditions
- Limb-girdle Muscular Dystrophy
- Keywords
- Limb Girdle Muscular Dystrophy Type 2E/R4, LGMD2E/R4, Pediatric, SRP-9003, scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec, Non-ambulatory, Ambulatory
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 15 Jan 2024 (actual)
- Primary completion
- 4 Mar 2025 (actual)
- Study completion
- 30 Nov 2029 (estimated)
- First posted
- 7 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 multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| SRP-9003 | Experimental | Biological: SRP-9003, Drug: GlucocorticoidParticipants will receive a single intravenous (IV) infusion of SRP-9003. |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Cohort 1, only ambulatory participants:
- Cohort 2, only non-ambulatory participants:
- Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
- Able to cooperate with muscle testing
- Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
Key exclusion criteria (5 listed)
- Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
- Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
- Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
- Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)
- Other inclusion/exclusion criteria 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 |
|---|---|---|
| SRP-9003Also: scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovecSolution for single IV infusion | Biological | SRP-9003 |
| GlucocorticoidAlso: Prednisone or equivalentOral tablet (prophylactic) | Drug | SRP-9003 |
Primary outcome
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Time frame: Baseline, Day 60
13 secondary outcomes
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay
Time frame: Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)
Time frame: Baseline, Day 60
Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)
Time frame: Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score
Time frame: Baseline through Month 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score
Time frame: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test
Time frame: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test
Time frame: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test
Time frame: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test
Time frame: Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test
Time frame: Baseline through Month 60
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
Time frame: Baseline through Month 60
Change From Baseline Through Month 60 in Creatine Kinase Level
Time frame: Baseline through Month 60
Time to Change of Loss of Ambulation
Time frame: Baseline through Month 60
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 2 Mar 2026.
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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.
10 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SRP-9003 and Glucocorticoid in people with Limb-girdle Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 10 sites in 6 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 10 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: under way, not looking for participants, as of the sponsor's last update on 2 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Belgium, Germany, Italy, Spain and 1 more country. 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?
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?
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?
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers (time frame: Baseline, Day 60). 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 2 Mar 2026.