Clinical trials: Crigler Najjar Syndrome, open to volunteers
Crigler-Najjar Syndrome Type I
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar type 1 aged ≤10 years and requiring phototherapy.
- Trial name
- NCT06641154
- Testing
- GT-UGT1A1-AAV8-02
- Where
- 2 sites in Russia
- Volunteers
- 5 people
- Sponsor
- Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Crigler-Najjar Syndrome
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Clinical trial rationale: CNS is an ultra-rare (<1/1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that…
- Trial name
- NCT06518005
- Testing
- Imlifidase, GNT0003
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Genethon
Crigler-Najjar Syndrome
Gene Therapy for Severe Crigler Najjar Syndrome
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy.
- Trial name
- CareCN (NCT03466463)
- Testing
- GNT0003
- Where
- 4 sites in France, Italy and 1 more
- Volunteers
- 17 people
- Sponsor
- Genethon