Active — Not RecruitingPhase 1

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

Denmark, France, United Kingdom6 participantsStarted 2022-09-01

Plain-language summary

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

Who can participate

Age range16 Years – 99 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Safety and tolerability

Timeframe: Baseline through 12 months

Trial details

NCT IDNCT05224505

SponsorAtamyo Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-01

View on ClinicalTrials.gov More LGMDR9 trials