RECRUITING

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

Conditions

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 1-2, Open-label, Dose Escalation Study to Evaluate the Safety of 2 Doses of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients with Gamma-sarcoglycanopathy (LGMDR5)

Quick Facts

Study Start:2025-02-15

Study Completion:2032-01-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05973630

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years to 13 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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	* 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

Inclusion Criteria

Exclusion 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

* 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

Contacts and Locations

Study Contact

Sophie Olivier

CONTACT

+33638682337

s.olivier@atamyo.com

Study Locations (Sites)

Child Health Research Institute

Gainsville, Florida, 32610

United States

Collaborators and Investigators

Sponsor: Atamyo Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-15

Study Completion Date2032-01-31

Study Record Updates

Study Start Date2025-02-15

Study Completion Date2032-01-31

Terms related to this study

Additional Relevant MeSH Terms

LGMD2C