TerminatedNCT06747273ClinicalTrials.gov record

Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States

Limb Girdle Muscular Dystrophy · Limb Girdle Muscular Dystrophy Type 2D/R3

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THE QUICK READ

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type: Interventional
Phase: Phase 1
Age range: 4 years - Not specified
Sex eligible: All sexes
Healthy volunteers: Not accepted
Participants: 4 Actual

These are starting points, not an eligibility decision. The research team must review the full requirements.

Why the study stopped

Study is being terminated due to a business decision.

About the research

The primary objective of this study is to evaluate the safety of SRP-9004.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Key Inclusion Criteria:

Ambulatory participants, defined as able to walk without assistive aid, 10MWR \<30 seconds, and NSAD total score ≥25; non-ambulatory participant, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3.
Ambulatory participants must be 4 to 20 years of age and the non-ambulatory participant must be ≥4 years of age.
All participants must be ≤70 kilograms
Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening.
Able to cooperate with muscle testing.
Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers \<1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).

Key Exclusion Criteria:

Left ventricular ejection fraction \<40% or clinical signs and/or symptoms of cardiomyopathy
FVC ≤40% of predicted value and/or requirement for nocturnal ventilation
Any other clinically significant illness, including neuromuscular (other than limb girdle muscular dystrophy type 2D/R3 \[LGMD2D/R3\]), 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.

Other inclusion/exclusion criteria apply.

Understand screening

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment: Not applicable
Masking: No masking

SRP-9004

Participants will receive a single dose of SRP-9004 on Day 1.

Drug: SRP-9004

SRP-9004 DRUG

Intravenous (IV) infusion.

Prepare your questions

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.

Primary measures

Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)

Time frame: Up to 60 months