Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT05224505

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

LGMDR9 Muscular Dystrophy, Limb-Girdle, Type 2I

Age range

16 year–99 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Rigshospitalet, University of Copenhagen Blegdamsvej 9, Copenhagen, Denmark

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ATA-100 (AAV9 encoding FKRP gene)

Biological

Single intravenous infusion on Day 0

Primary outcomes

  1. Safety and tolerability

    Time frame: Baseline through 12 months

    Incidence of Adverse Events and significant laboratory changes

Secondary outcomes

  1. Combined endpoint (global testing)

    Time frame: Baseline through 12 months

    Change in North Star Assessment for musculary Dystrophy (NSAD) and Forced Vital capacity (FVC %) from baseline

  2. Timed Up and Go (TUG) test

    Time frame: Baseline through 12 months

    Secondary endpoint

  3. Change from baseline in velocity as measured by 10MWT

    Time frame: Baseline through 12 months

    10-meter walk test

  4. 2-minute walk distance test

    Time frame: Baseline through 12 months

    Secondary endpoint

  5. Cardiac MRI

    Time frame: Baseline through 12 months

    To measure cardiac function (left ejection fraction)

  6. Muscle MRI

    Time frame: Baseline through 12 months

    To measure change from baseline in fat repartition fraction in thigh and leg skeletal muscles

  7. Muscle Biopsy

    Time frame: Baseline through 12 months

    Quantification of FKRP positive muscle fibers

  8. Muscle Biopsy

    Time frame: Baseline through 12 months

    Percentage of glycosylation

  9. Patient reported outcome and quality of life assessment

    Time frame: Baseline through 12 months

    Quality of Life in genetic Neuromuscular Disease (QoL-gNMD), with a range from 0 to 78, the higher the score the worse the quality of life

  10. Patient reported outcome and quality of life assessment

    Time frame: Baseline through 12 months

    ACTIVLIM, scale measuring level of limitation in performing daily activities (total score ranging from 0 to 44, with the lower score the highest limitation)

Sponsors and collaborators

Lead sponsor

Atamyo Therapeutics

Industry

Registry information

Official study title

A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)

Important dates

Study start
2022
Primary completion
2025
Study completion
2029
First posted
Feb 4, 2022
Registry last updated
Oct 6, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.