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A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

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Trial Profile

A Phase I/II, Open Label, Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 11 Apr 2024

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At a glance

  • Drugs GNT 0003 (Primary)
  • Indications Crigler-Najjar syndrome
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Acronyms CareCN
  • Sponsors Genethon

    • Most Recent Events

    • 17 Aug 2023 Results (n=5 of the dose-escalation portion of a phase 1-2) assessing safety and efficacy of a single intravenous infusion of an adeno-associated virus serotype 8 vector encoding UGT1A1 in patients with the Crigler-Najjar syndrome published in the New England Journal of Medicine
    • 23 Mar 2023 Planned End Date changed from 27 Dec 2026 to 30 Mar 2030.
    • 23 Mar 2023 Planned primary completion date changed from 20 Jun 2022 to 30 Mar 2026.

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