An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies
Latest Information Update: 06 Dec 2024
At a glance
- Drugs GNT 0003 (Primary) ; Imlifidase (Primary)
- Indications Crigler-Najjar syndrome
- Focus Therapeutic Use
- Sponsors Genethon
Most Recent Events
- 28 Nov 2024 Status changed from not yet recruiting to recruiting.
- 29 Jul 2024 Status changed to not yet recruiting.
- 02 Feb 2024 According to Hansa Biopharma AB media release, initiation of clinical study with imlifidase prior to GNT-0003 will be in 2024.