Gene Therapy for Severe Crigler Najjar Syndrome
Recruiting
N/A
Interventional Study
Crigler-Najjar Syndrome
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 9 and older
- Sex
- Any
- Study type
- Interventional
- Purpose
- Other
- Participants needed
- 17 (estimated)
- Sponsor
- Genethon · Other
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About This Trial
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. Patients will received a single administration of GNT0003 and will be followed for safety and efficacy of approximately 60 months (5 years):
* a follow-up of approximately 12 months (48 weeks)
* a…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy
* Male or female at least 9 years at the date of signature of informed consent
* Patient able to give informed assent and/or consent …
Contacts
Genethon Clinical Development Department
00 33 (0)1 69 47 10 32
clinical_development@genethon.fr
CONTACT