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Muscular Dystrophies, Limb-GirdlePhase 3COMPASSNCT06952686

A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants

A Phase 3 study testing SRP-9005 and Corticosteroid in people with Muscular Dystrophies, Limb-Girdle, run by Sarepta Therapeutics, Inc. at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy. Read more about this study

Who is it for?
Ages 4 years and older
Conditions the study is for
Muscular Dystrophies, Limb-Girdle
What is being tested?
SRP-9005 and Corticosteroid
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
30 Jun 2025to 30 Mar 2032
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 4 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Ambulatory, defined as able to walk without assistive aid, 10MWR <30 seconds, and NSAD total score ≥20
  • Non-ambulatory, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3
  • Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic gamma-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to pre-Infusion screening. Results to be confirmed by sponsor at a CLIA/CAP/ISO15189 certified laboratory prior to dosing.
  • Have AAVrh74 antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunoassay

Who cannot join

  • Has a symptomatic infection (for example, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks before study treatment infusion
  • Part A: Has left ventricular ejection fraction (LVEF) <50% on the screening echocardiogram (ECHO) (without use of cardiac medication) or clinical signs and/or symptoms of cardiomyopathy or any history of cardiac disease
  • Part B: Has LVEF <40% on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy
  • Has FVC <40% of predicted value at screening and/or requirement for nocturnal ventilation
  • Serological evidence of current, chronic, or active human immunodeficiency virus infection, or hepatitis B or C infection or active viral or bacterial infection based on clinical observations
  • Any prior treatment with gene therapy, cell-based therapy (for example, stem cell transplantation), clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9), or any other form of gene editing
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  • Treatment with human growth factor within 3 months of Day 1
  • Treatment with any investigational medication within 6 months of the screening visit
  • Is unable to undergo or tolerate a cardiac MRI procedure for any reason
  • Note: Other inclusion/exclusion criteria may apply.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23510

Other trials in United States

About this study

Official title: A Seamless Phase 1/3, Multicenter, Single Dose Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Subjects (COMPASS)

This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy.

Being tested
SRP-9005, Corticosteroid
Keywords
SRP-9005, Type 2C/R5, LGMD2C/R5, Pediatric, Non-ambulatory, Ambulatory, Phase 1, Phase 3
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-9005Biological

    Also called rAAVrh74.MHCK7.hSGCG

    Solution for single IV infusion

  • CorticosteroidDrug

    Also called Prednisone or equivalent

    Oral tablet (prophylactic)

Study groups in NCT06952686
Study groupRoleWhat they receive
SRP-9005ExperimentalParticipants will receive a single intravenous (IV) infusion of SRP-9005.
Receives: Biological: SRP-9005; Drug: Corticosteroid

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs)

    Measured: Baseline through Month 60

  2. Part A and B: Change from Baseline in gamma-Sarcoglycan (SG) Expression at Day 60 Post-dose in Ambulatory Participants as Measured by Immunofluorescence (IF) Percent Positive Fibers (PPF)

    Measured: Baseline, Day 60

Secondary outcomes (29)

  1. Part A and B: Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score

    Measured: Baseline through Month 60

  2. Part A and B: Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score

    Measured: Baseline through Month 60

  3. Part A and B: Change from Baseline through Month 60 in Time to Rise From Floor

    Measured: Baseline through Month 60

  4. Part A and B: Change from Baseline Through Month 60 in Time to Ascend 4 Steps

    Measured: Baseline through Month 60

  5. Part A and B: Change from Baseline Through Month 60 in Time to Complete the 10-meter Walk/Run (10MWR)

    Measured: Baseline through Month 60

  6. Part A and B: Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)

    Measured: Baseline through Month 60

  7. Part A and B: Change from Baseline Through Month 60 in Timed Up and Go

    Measured: Baseline through Month 60

  8. Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by IF Percent Fiber Intensity (PFI)

    Measured: Baseline, Day 60

  9. Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by Western Assay

    Measured: Baseline, Day 60

  10. Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PPF

    Measured: Baseline to Day 60

  11. Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PFI

    Measured: Baseline to Day 60

  12. Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by Western Assay

    Measured: Baseline to Day 60

  13. Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PPF

    Measured: Baseline to Month 24

  14. Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PFI

    Measured: Baseline to Month 24

  15. Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by Western Assay

    Measured: Baseline to Month 24

  16. Part A and B: Change from Baseline Through Month 60 in Creatine Kinase Levels

    Measured: Baseline through Month 60

  17. Part A and B: Loss of Ambulation

    Measured: Baseline through Month 60

  18. Part A and B: Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imaging (MRI)

    Measured: Baseline through Month 60

  19. Part A and B: Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95th Percentile (SV95C) (Ambulatory Participants Only)

    Measured: Baseline through Month 60

  20. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)

    Measured: Baseline through Month 60

  21. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity Percent Predicted (FVC%p)

    Measured: Baseline through Month 60

  22. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)

    Measured: Baseline through Month 60

  23. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second % Predicted (FEV1%p)

    Measured: Baseline through Month 60

  24. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)

    Measured: Baseline through Month 60

  25. Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)

    Measured: Baseline through Month 60

  26. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Inspiratory Pressure (MIP%p)

    Measured: Baseline through Month 60

  27. Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)

    Measured: Baseline through Month 60

  28. Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Expiratory Pressure (MEP%p)

    Measured: Baseline through Month 60

  29. Part B: Number of Participants with TEAEs

    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.

  1. First posted1 May 2025
  2. Study start30 Jun 2025 (estimated)
  3. Primary completion30 Nov 2030 (estimated)
  4. Study completion30 Mar 2032 (estimated)

Why it stopped: Study was Withdrawn per Sponsor decision.

Resources

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
Model description
This study comprises 2 parts: Part A is the Phase 1 portion, Part B is the Phase 3 expansion portion.
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06952686
Sponsor's protocol ID
SRP-9005-101
Acronym
COMPASS
Status last verified
Jul 2025
First submitted
23 Apr 2025
Last updated
6 Aug 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing SRP-9005 and Corticosteroid in people with Muscular Dystrophies, Limb-Girdle, 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 4 inclusion and 10 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: withdrawn, as of the sponsor's last update on 6 Aug 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 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 withdrawn before enrolling participants, so there are no results.

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?

Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs) (time frame: Baseline through Month 60). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06952686.

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 Aug 2025.

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