GNT0006 Gene Therapy Trial in Patients With LGMDR9

Last updated: July 30, 2024
Sponsor: Atamyo Therapeutics
Overall Status: Active - Recruiting

Phase

1/2

Condition

N/A

Treatment

Day 0: Placebo

Day 365 (year 1): Placebo

Day 0: GNT0006

Clinical Study ID

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

Study Summary

Phase 1-2 study including a dose escalation safety and proof of concept phase (Stage 1, open label), followed by a double-blind, randomized, placebo-controlled confirmatory phase (Stage 2)

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: 39
Treatment Group(s): 5
Primary Treatment: Day 0: Placebo
Phase: 1/2
Study Start date:
August 10, 2022
Estimated Completion Date:
October 31, 2030

Study Description

Multicenter, Phase 1-2 study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of GNT0006, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study will consist of 2 phases: an open-label dose escalation phase (Stage 1) and a double-blind placebo controlled, randomized phase (Stage 2), both with long-term follow-up (LTFU) period.

Stage 1 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.

Stage 2 After selection of the effective dose in Stage 1, thirty-three (33) ambulant patients will be randomized at the optimal selected dose and followed up to the primary efficacy timepoint, i.e., one year after investigational medicinal product (IMP) (or placebo) administration.

At one-year post-IMP administration (timepoint of primary interest for efficacy), patients enrolled in placebo group will receive active IMP while patients randomized in the active IMP group will receive a placebo infusion.

All subjects will be followed for up to 5 years after active IMP (GNT0006) administration.

Connect with a study center

  • Rigshospitalet, University of Copenhagen Blegdamsvej 9

    Copenhagen, 2100
    Denmark

    Active - Recruiting

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

    Paris, 75013
    France

    Active - Recruiting

  • Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing

    Newcastle Upon Tyne, NE1 4LP
    United Kingdom

    Active - Recruiting

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