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

Plain-language summary

This Phase 1 dose-escalation study assesses the tolerability and safety of ATA-100, an AAV9 vector encoding the FKRP gene, in patients with limb-girdle muscular dystrophy type R9 (LGMDR9), with five years of follow-up. Eligible participants are ambulant males and females aged 16 or older with a documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming FKRP mutations. Moderate diaphragmatic muscle impairment is required, and people with detectable serum neutralizing antibodies are excluded.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 1

Status

Active, not recruiting

Sponsor

Atamyo Therapeutics

Intervention

ATA-100 (AAV9 encoding FKRP gene)

Start date

9/1/2022

Estimated completion

9/30/2029

Countries

Denmark, France, United Kingdom

Locations

• Copenhagen, Denmark
• Paris, France
• Newcastle upon Tyne, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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