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CareCN gene therapy study in severe Crigler-Najjar syndrome
NL-OMON52580Phase 2Recruiting
- Sponsor
- Généthon
- Start
- May 2018
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label trial of GNT-0003 in Crigler-Najjar syndrome (CNS) (2 arms), sponsored by Généthon. Primary endpoints: Incidence of treatment-emergent adverse events and serious adverse events, Change in safety assessments and 1 more. Started May 2018; status: recruiting.
02
Drugs
GNT-0003
03
Conditions
Crigler-Najjar syndrome (CNS)
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