ATA-200 Gene Therapy Trial in Patients With LGMDR5

NCT ID: NCT05973630

Last Updated: 2026-01-29

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-02-15
• Study Completion Date: 2031-01-31

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.

Conditions

LGMD2C

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment arm

ATA-200 Dose : 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h

Group Type: EXPERIMENTAL

ATA-200

Intervention Type: BIOLOGICAL

single intravenous infusion

Interventions

ATA-200

single intravenous infusion

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 13 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Atamyo Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Child Health Research Institute

Gainsville, Florida, United States

Countries

United States

Other Identifiers

ATA-003-GSAR

Identifier Type: -

Identifier Source: org_study_id