Gene therapy for Crigler-Najjar Syndrome Type I
A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GT-UGT1A1-AAV8-02 (AAV Vector Expressing the UGT1A1 Transgene) in Patients with Crigler-Najjar Syndrome Type I Requiring Phototherapy
This study is testing a new gene therapy for young children with Crigler-Najjar Syndrome Type I to see if it can safely help them manage their severe jaundice and improve their health.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 5 (estimated) |
| Ages | 3 Months to 10 Years |
| Sex | All |
| Sponsor | Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare Government |
| Locations | 2 sites (Moscow and 1 other locations) |
| Trial ID | NCT06641154 on ClinicalTrials.gov |
What this trial studies
This Phase 1/2 clinical trial evaluates the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in children aged 10 years or younger with Crigler-Najjar Syndrome Type I who require phototherapy. Participants will receive a single administration of the gene therapy and will be monitored for safety and efficacy over a period of approximately 60 months. The study aims to address the underlying genetic cause of the disease, which leads to severe jaundice and neurological disorders due to the inability to process bilirubin. The trial is conducted in a multinational setting, focusing on a rare inherited condition with limited treatment options.
Who should consider this trial
Good fit: Ideal candidates are children aged 3 months to 10 years with a confirmed diagnosis of severe Crigler-Najjar Syndrome Type I requiring phototherapy.
Not a fit: Patients who have undergone liver transplantation or have significant underlying liver disease will not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could provide a long-term solution for managing Crigler-Najjar Syndrome Type I, potentially reducing the need for phototherapy and improving quality of life.
How similar studies have performed: While gene therapy for Crigler-Najjar Syndrome is a novel approach, previous studies in gene therapy for other genetic disorders have shown promising results.
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 3 months (no more 10 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
Moscow and 1 other locations
- Alphaviva LLC — Moscow, Russia (Recruiting)
- Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare — Moscow, Russia (Recruiting)
Study contacts
- Study coordinator: Denis V. Rebrikov, Dr., Professor,
- Email: d_rebrikov@oparina4.ru
- Phone: +7 (495) 531-44-44
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.