ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

NCT05224505 · tracked via the Priya Life Science UK tracker

Sponsor

Atamyo Therapeutics

Phase

Phase 1

Started

2022-09-01

Last updated

2025-10-06

Condition(s) studied

LGMDR9

Investigational drug(s) / intervention(s)

ATA-100 (AAV9 encoding FKRP gene)

ATA-100 (AAV9 encoding FKRP gene): Single intravenous infusion on Day 0

Study summary

Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up

Eligibility

Sex

ALL

Min age

16 Years

Max age

99 Years

Healthy volunteers

Inclusion Criteria: * 1\. Female and male ambulant patients * 2\. Patients ≥ 16 years old * 3\. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations * 4\. Moderate diaphragmatic muscle impairment Exclusion Criteria: * 1\. Detectable serum neutralizing antibodies against AAV9 * 2\. Cardiomyopathy

Primary outcome measure(s)

Safety and tolerability — Baseline through 12 months
Incidence of Adverse Events and significant laboratory changes

Trial sites (3)

Facility	City	Region
Rigshospitalet, University of Copenhagen Blegdamsvej 9	Copenhagen	Denmark
Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital	Paris	France
Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing	Newcastle upon Tyne	United Kingdom

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05224505 on ClinicalTrials.gov ↗ ← All trials in the UK