Gene therapy for severe Crigler-Najjar syndrome
A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy
This study is testing a new gene therapy called GNT0003 to see if it can safely help people aged 10 and older with severe Crigler-Najjar syndrome who need light treatment.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 17 (estimated) |
| Ages | 9 Years and up |
| Sex | All |
| Sponsor | Genethon Academic / other |
| Locations | 4 sites (Clamart and 3 other locations) |
| Trial ID | NCT03466463 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety and efficacy of GNT0003, a gene therapy administered via intravenous infusion, in patients aged 10 years and older with severe Crigler-Najjar syndrome who require phototherapy. Participants will receive a single dose of GNT0003 and will be monitored for safety and efficacy over a period of approximately 60 months. The study follows guidelines for long-term follow-up of patients receiving gene therapy, ensuring comprehensive assessment of outcomes.
Who should consider this trial
Good fit: Ideal candidates are patients aged 10 years and older with a confirmed diagnosis of severe Crigler-Najjar syndrome requiring phototherapy.
Not a fit: Patients who have undergone liver transplantation or have significant underlying liver disease may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve the quality of life for patients with severe Crigler-Najjar syndrome by reducing or eliminating the need for phototherapy.
How similar studies have performed: Other studies involving gene therapy for genetic disorders have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy * Male or female at least 9 years at the date of signature of informed consent * Patient able to give informed assent and/or consent in writing Exclusion Criteria: * Patients who underwent liver transplantation * Patients with chronic hepatitis B or C * Patients infected with Human immunodeficiency virus (HIV) * Patients with significant underlying liver disease * Patients with significant encephalopathy * Participation in any other investigational trial during this trial * Patients unable or unwilling to comply with the protocol requirements
Where this trial is running
Clamart and 3 other locations
- Hopital Antoine BECLERE — Clamart, France (Recruiting)
- ASST Papa Giovanni XXIII — Bergamo, Italy (Recruiting)
- Azienda Ospedaliera Universitaria Federico II — Naples, Italy (Recruiting)
- Amc — Amsterdam, Netherlands (Recruiting)
Study contacts
- Principal investigator: LABRUNE Philippe, Prof — Hopital Antoine Beclere
- Study coordinator: Genethon Clinical Development Department
- Email: clinical_development@genethon.fr
- Phone: 00 33 (0)1 69 47 10 32
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.