Status:
RECRUITING
Gene Therapy for Severe Crigler Najjar Syndrome
Lead Sponsor:
Genethon
Conditions:
Crigler-Najjar Syndrome
Eligibility:
All Genders
9+ years
Phase:
NA
Brief Summary
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 phototherap...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
- Patients with significant encephalopathy
- Participation in any other investigational trial during this trial
- Patients unable or unwilling to comply with the protocol requirements
Key Trial Info
Start Date :
March 19 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 30 2030
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT03466463
Start Date
March 19 2018
End Date
March 30 2030
Last Update
March 28 2023
Active Locations (4)
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1
Hopital Antoine BECLERE
Clamart, France, 92141
2
ASST Papa Giovanni XXIII
Bergamo, Italy, 24127
3
Azienda Ospedaliera Universitaria Federico II
Napoli, Italy, 80131
4
AMC
Amsterdam, Netherlands, 1105