Gene therapy for patients with LGMDR9
A Phase 1-2 Multicenter Study (2-stages) to Evaluate the Safety and Efficacy of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)
This study is testing a new gene therapy to see if it can help people with LGMDR9 by safely delivering a specific gene that may improve their condition.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 39 (estimated) |
| Ages | 16 Years to 99 Years |
| Sex | All |
| Sponsor | Atamyo Therapeutics Industry-sponsored |
| Locations | 3 sites (Copenhagen and 2 other locations) |
| Trial ID | NCT05224505 on ClinicalTrials.gov |
What this trial studies
This is a Phase 1-2 clinical trial evaluating the safety and efficacy of GNT0006, a gene therapy using an Adeno-Associated Virus vector to deliver the human FKRP transgene. The study consists of two phases: an open-label dose escalation phase where initial cohorts of patients receive varying doses, followed by a double-blind, randomized, placebo-controlled phase. The trial aims to assess pharmacodynamics, immunogenicity, and long-term outcomes over a period of up to five years. Patients will be monitored for efficacy one year after administration of the investigational medicinal product or placebo.
Who should consider this trial
Good fit: Ideal candidates are ambulant patients aged 16 and older with a documented diagnosis of LGMDR9 and moderate diaphragmatic muscle impairment.
Not a fit: Patients with detectable serum neutralizing antibodies against AAV9 or those with cardiomyopathy may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve muscle function and quality of life for patients with LGMDR9.
How similar studies have performed: Other studies using gene therapy approaches for similar conditions have shown promise, indicating potential for success in this novel application.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Copenhagen and 2 other locations
- Rigshospitalet, University of Copenhagen Blegdamsvej 9 — Copenhagen, Denmark (Recruiting)
- Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital — Paris, France (Recruiting)
- Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing — Newcastle Upon Tyne, United Kingdom (Recruiting)
Study contacts
- Study coordinator: John Vissing, Pr
- Email: john.vissing@regionh.dk
- Phone: +4535452562
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.