Actively Recruiting
Gene Therapy for Severe Crigler Najjar Syndrome
Led by Genethon · Updated on 2023-03-28
17
Participants Needed
4
Research Sites
627 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gene Therapy for Severe Crigler Najjar Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with severe Crigler-Najjar syndrome confirmed by mutations in the UGT1A1 gene and requiring phototherapy
- Male or female patients aged 9 years or older at the time of consent
- Patient able to provide informed assent and/or written consent
You will not qualify if you...
- Patients who have undergone liver transplantation
- Patients with chronic hepatitis B or C
- Patients infected with HIV
- Patients with significant underlying liver disease
- Patients with significant encephalopathy
- Patients currently participating in another investigational trial
- Patients unable or unwilling to follow the study protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Hopital Antoine BECLERE
Clamart, France, 92141
Actively Recruiting
2
ASST Papa Giovanni XXIII
Bergamo, Italy, 24127
Actively Recruiting
3
Azienda Ospedaliera Universitaria Federico II
Naples, Italy, 80131
Actively Recruiting
4
AMC
Amsterdam, Netherlands, 1105
Actively Recruiting
Research Team
G
Genethon Clinical Development Department
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
OTHER
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here