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Clinical trial

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

NCT06518005 is registered in ClinicalTrials.gov with Genethon as lead sponsor.

NCT NCT06518005
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Genethon
Phase
Phase 2
Overall status
Recruiting
Start date
November 8, 2024
Completion date
September 30, 2030
Enrollment
3 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

crigler-najjar syndrome