Gene therapy for Crigler-Najjar Syndrome Type I
Official title: 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
Run by Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare · NCT06641154 · Data updated 2026-11-12
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 |
| Ages | 3 Months to 10 Years |
| Sex | All |
| Enrollment | 5 (estimated) |
| Sponsor | Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare Government |
| Locations | 2 sites (Moscow and 1 other locations) |
| Status | Recruiting |
| Started | November 6, 2024 |
| Main results expected | November 1, 2027 |
| Study ends | November 1, 2029 |
| Registry record updated | January 13, 2025 |
| Trial ID | NCT06641154 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a 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.
✕Probably not a fit
Patients who have undergone liver transplantation or have significant underlying liver disease will 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 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.
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 gone: While gene therapy for Crigler-Najjar Syndrome is a novel approach, previous studies in gene therapy for other genetic disorders have shown promising results.
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 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 it's running
-
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
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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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.
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Gene therapy for severe Crigler-Najjar syndrome
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.
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.