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NCT06641154
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
Phase 1, PHASE2 trial testing GT-UGT1A1-AAV8-02 in Crigler-Najjar Syndrome Type I in 5 participants. Currently enrolling.
1 November 2027
Quick facts
| Lead sponsor | Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Recruiting now |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | sequential |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 6 November 2024 |
| Primary completion | 1 November 2027 |
| Estimated completion | 1 November 2029 |
| Sites | 2 locations across Russia |
Drugs / interventions tested
- GT-UGT1A1-AAV8-02 — full drug profile →
Conditions studied
- Crigler-Najjar Syndrome Type I — all drugs for Crigler-Najjar Syndrome Type I →
Sponsor
Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Who can join
Adults 3 Months to 10, any sex, with Crigler-Najjar Syndrome Type I. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar type 1 aged ≤10 years and requiring phototherapy. Patients will received a single administration of GT-UGT1A1-AAV8-02 and will be followed for safety and efficacy of approximately 60 months (5 years): * a follow-up of approximately 12 months (48 weeks) * a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
Publications & conference data
No peer-reviewed publications indexed yet for this trial.
Verify or expand the search:
- PubMed search for NCT06641154
- Europe PMC full search
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Related trials
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT06641154 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
- Last refreshed: 13 January 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06641154.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing