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

Last updated: October 1, 2025
Sponsor: Atamyo Therapeutics
Overall Status: Active - Not Recruiting

Phase

1

Condition

N/A

Treatment

ATA-100 (AAV9 encoding FKRP gene)

Day 0: Placebo

Day 365 (year 1): Placebo

Clinical Study ID

NCT05224505
ATA-001-FKRP
2021-004276-33
  • Ages 16-99
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

    1. Female and male ambulant patients
    1. Patients ≥ 16 years old
    1. Documented LGMDR9 diagnosis based on clinical presentation and genotypingconfirming the FKRP gene mutations
    1. Moderate diaphragmatic muscle impairment

Exclusion

Exclusion Criteria:

    1. Detectable serum neutralizing antibodies against AAV9
    1. Cardiomyopathy

Study Design

Total Participants: 6
Treatment Group(s): 6
Primary Treatment: ATA-100 (AAV9 encoding FKRP gene)
Phase: 1
Study Start date:
September 01, 2022
Estimated Completion Date:
September 30, 2029

Study Description

Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.

Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.

All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.

Connect with a study center

  • Rigshospitalet, University of Copenhagen Blegdamsvej 9

    Copenhagen, 2100
    Denmark

    Site Not Available

  • Rigshospitalet, University of Copenhagen Blegdamsvej 9

    Copenhagen 2618425, 2100
    Denmark

    Site Not Available

  • Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital

    Paris, 75013
    France

    Site Not Available

  • Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital

    Paris 2988507, 75013
    France

    Site Not Available

  • Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing

    Newcastle Upon Tyne, NE1 4LP
    United Kingdom

    Site Not Available

  • Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing

    Newcastle upon Tyne 2641673, NE1 4LP
    United Kingdom

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.