Gene therapy for severe Crigler-Najjar syndrome
Official title: 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
Run by Genethon · NCT03466463 · Data updated 2026-11-12
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
| Study type | Interventional |
|---|---|
| Ages | 9 Years and up |
| Sex | All |
| Enrollment | 17 (estimated) |
| Sponsor | Genethon Academic / other |
| Locations | 4 sites (Clamart and 3 other locations) |
| Status | Recruiting |
| Started | March 19, 2018 |
| Main results expected | March 30, 2026 |
| Study ends | March 30, 2030 |
| Registry record updated | March 28, 2023 |
| Trial ID | NCT03466463 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients aged 10 years and older with a confirmed diagnosis of severe Crigler-Najjar syndrome requiring phototherapy.
✕Probably not a fit
Patients who have undergone liver transplantation or have significant underlying liver disease may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
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.
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 gone: Other studies involving gene therapy for genetic disorders have shown promising results, indicating potential for success in this approach.
Who can join: the full rules
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 it's running
-
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
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Treatment for severe Crigler-Najjar syndrome using gene therapy
This study is testing a new gene therapy to see if it can safely lower bilirubin levels in people with severe Crigler-Najjar syndrome and help them avoid needing a liver transplant.
-
GNT0004 gene therapy for Duchenne muscular dystrophy in ambulant boys
This study tests a one-time IV gene therapy (GNT0004) to see if it slows DMD progression in ambulant boys aged 6–10.
-
Natural history of Duchenne muscular dystrophy in boys aged 4–9
This project will follow boys aged 4–9 with Duchenne muscular dystrophy for 6–36 months to collect standard clinical, imaging, and lab data and create a baseline before possible gene therapy.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.