ATA-200 Gene Therapy Trial in Patients With LGMDR5
A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)
1 other identifier
interventional
4
1 country
1
Brief Summary
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Feb 2025
Longer than P75 for phase_1
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 24, 2023
CompletedFirst Posted
Study publicly available on registry
August 3, 2023
CompletedStudy Start
First participant enrolled
February 15, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 31, 2031
January 29, 2026
January 1, 2026
1.7 years
July 24, 2023
January 27, 2026
Conditions
Outcome Measures
Primary Outcomes (3)
Incidence of adverse events
Collection of adverse events at each visit
0-6 months
Incidence of treatment-emergent adverse events,
Collection of adverse events at each visit
0-6 months
Incidence of serious adverse events
Collection of adverse events at each visits
0-6 months
Study Arms (1)
Treatment arm
EXPERIMENTALATA-200 Dose : 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
Interventions
Eligibility Criteria
You may qualify if:
- Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
- Ambulant male or female patients aged 6 to less than 12 years of age at screening
- Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
You may not qualify if:
- Detectable neutralizing antibodies against AAV8
- Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50%
- Respiratory assistance
- Concomitant medical condition that might interfere with LGMDR5 evolution
- Acute illness within 4 weeks of anticipated IMP administration
- Current participation in another clinical trial with investigational medicinal product
- Previous participation in gene and cell therapy trials
- Any condition that would contraindicate immunosuppressant treatment
- Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
- Any vaccination 1 month prior to planned IMP administration
- Serology consistent with HIV exposure or active hepatitis B or C infection
- Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Child Health Research Institute
Gainsville, Florida, 32610, United States
MeSH Terms
Conditions
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 24, 2023
First Posted
August 3, 2023
Study Start
February 15, 2025
Primary Completion (Estimated)
October 30, 2026
Study Completion (Estimated)
January 31, 2031
Last Updated
January 29, 2026
Record last verified: 2026-01