ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5

Phase 1

Recruiting

• Conditions: LGMD2C
• Interventions: Biological: ATA-200

• Registration Number: NCT05973630
• Lead Sponsor: Atamyo Therapeutics

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). Patients will be treated sequentially in 2 dose-cohorts.

Detailed Description

This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5.

The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose cohorts (C1) and (C2) will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. An initial cohort C1 of three (3) patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.

Enrollment of three (3) patients in the 2nd higher dose cohort C2 (with a 7-fold safety margin relative to the highest safe dose in the GLP toxicology study) will be initiated following review of the one-month safety data post-administration in cohort C1 by an independent Data Safety Monitoring Board (DSMB).

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 evaluation period.

Study Timeline

• Study First Submitted: 2023-07-24
• Study First Submitted QC: 2023-08-01
• Study First Posted: 2023-08-03
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-12
• Last Update Posted: 2025-02-14
• Study Start: 2025-02-15
• Primary Completion: 2026-07-30
• Study Completion: 2032-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 6

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 2	ATA-200	ATA-200 Dose level 2: 3.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
Cohort 1	ATA-200	ATA-200 Dose level 1: 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events,	0-6 months	Collection of adverse events at each visit
Incidence of 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

Trial Locations

Locations (3)

Child Health Research Institute; 🇺🇸 Gainsville, Florida, United States

Hopital Trousseau; 🇫🇷 Paris, France

Ospedale Maggiore Policlinico; 🇮🇹 Milano, Italy