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Trial intelligence / Profile preview
GNT0003 gene therapy for severe Crigler-Najjar syndrome
NL-OMON60014Phase 2Recruiting
- Sponsor
- Généthon
- Start
- Apr 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 treatment-emergent serious adverse events, Change in safety assessments and 1 more. Started Apr 2018; status: recruiting.
02
Drugs
GNT-0003
03
Conditions
Crigler-Najjar syndrome (CNS)
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