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)

Status

Active, not recruiting

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05973630

Enrollment

Registered

2023-08-03

Start date

2025-02-15

Completion date

2031-01-31

Last updated

2026-01-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

LGMD2C

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).

Detailed description

This is a multicenter Phase 1b assessing the safety and tolerability of intravenous ATA-200 for the treatment of LGMDR5. The study will enroll ambulant patients with LGMDR5. Patients will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. Patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies. Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months. All subjects will be followed up for an additional 4.5 years after completion of the initial evaluation period.

Interventions

BIOLOGICALATA-200

single intravenous infusion

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Years to 13 Years

Healthy volunteers

Inclusion criteria

* 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

Exclusion criteria

* 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

Design outcomes

Primary

Measure	Time frame	Description
Incidence of adverse events	0-6 months	Collection of adverse events at each visit
Incidence of treatment-emergent adverse events,	0-6 months	Collection of adverse events at each visit
Incidence of serious adverse events	0-6 months	Collection of adverse events at each visits

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026