ATA-200 Gene Therapy Trial in Patients With LGMDR5

Phase 1Active Not RecruitingNCT05973630

Atamyo Therapeutics4 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

LGMD2C

Interventions

ATA-200BIOLOGICAL

single intravenous infusion

Eligibility

Sex: ALLMin age: 6 YearsMax age: 13 Years

Medical Language ↔ Plain English

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

Locations (1)

Child Health Research Institute

Gainsville, Florida, United States

Outcomes

Primary Outcomes

Incidence of adverse events

Collection of adverse events at each visit

Time frame: 0-6 months

Incidence of treatment-emergent adverse events,

Collection of adverse events at each visit

Time frame: 0-6 months

Incidence of serious adverse events

Collection of adverse events at each visits

Time frame: 0-6 months

Data from ClinicalTrials.gov

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