CareCN: A phase I/II, open label, escalating dose study to evaluate safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy
Phase of Trial: Phase I/II
Latest Information Update: 10 Dec 2018
At a glance
- Drugs GNT 0003 (Primary)
- Indications Crigler-Najjar syndrome
- Focus Adverse reactions; First in man; Therapeutic Use
- Acronyms CareCN
- Sponsors Genethon
- 10 Dec 2018 According to a Genethon media release, the company has treated the first patient in this study.
- 27 Sep 2018 Planned End Date changed from 31 Aug 2020 to 31 Dec 2020.
- 27 Sep 2018 Planned primary completion date changed from 31 Aug 2020 to 31 Dec 2020.