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: 18 Aug 2023
Price :
$35
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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
- 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.