Gene Therapy for Severe Crigler Najjar Syndrome
ConditionCrigler-Najjar Syndrome
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age9+
SponsorGenethon
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 long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
Eligibility criteria
Qualifiers
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 in writing
Disqualifiers
Patients who underwent liver transplantation
Patients with chronic hepatitis B or C
Patients infected with Human immunodeficiency virus (HIV)
Patients with significant underlying liver disease
Trial design
Treatments tested in this trial
- GNT0003
Treatment groups
17 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov