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A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

NCT ID: NCT05876780

Last Updated: 2024-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-19

Study Completion Date

2028-08-28

Brief Summary

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The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.

Detailed Description

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Conditions

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Limb Girdle Muscular Dystrophy

Keywords

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Ambulatory Non-ambulatory Gene-Delivery LGMD2E/R4 β-SG deficiency LGMD Performance of Upper Limb (PUL)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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SRP-9003

Participants will receive single IV infusion of SRP-9003 on Day 1.

Group Type EXPERIMENTAL

SRP-9003

Intervention Type GENETIC

Single IV infusion of SRP-9003

Interventions

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SRP-9003

Single IV infusion of SRP-9003

Intervention Type GENETIC

Other Intervention Names

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scAAVrh74.MHCK7.hSGCB

Eligibility Criteria

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Inclusion Criteria

* Cohort 1 only: Ambulatory per protocol specified criteria. - Cohort 2 only: Non-ambulatory per protocol specified criteria and 4 to 50 years of age.
* Possesses 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-sarcoglycan gene Deoxyribonucleic acid (DNA) (SGCB) gene mutations based on documented clinical findings.
* Ability to cooperate with muscle testing.

Exclusion Criteria

* Presence of any other clinically significant illness or medical condition, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, neuromuscular (other than LGMD2E/R4), 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 gene transfer 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.
* Any contraindication to use of corticosteroid.
Minimum Eligible Age

4 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Sarepta Therapeutics, Inc.

Locations

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Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Countries

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United States

Other Identifiers

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SRP-9003-102

Identifier Type: -

Identifier Source: org_study_id