Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN) — Stella

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Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN) — Stella

Recruiting

Back to Crigler-Najjar Syndrome Type I trials

Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(2 sites)

Russia

Alphaviva LLC, Moscow

Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare, Moscow

Key details

Phase

Early testing / Safety & dosing

Sponsor

Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare

Enrollment target

~5 participants

Started

November 2024

Primary completion

November 2027

Age range

3 Months – 10 Years

Last updated January 2025

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