Gene Therapy for Crigler Najjar Syndrome

Phase 1

• Conditions: severe Crigler Najjar syndrome in patients requiring phototherapy; MedDRA version: 20.0Level: PTClassification code 10011386Term: Crigler-Najjar syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2017-000506-37-NL
• Lead Sponsor: Genethon

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-10-16
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

1. Patients with severe Crigler-Najjar syndrome requiring phototherapy
2. Molecular confirmation of mutation in the UGT1A1 gene by DNA sequencing

Are the trial subjects under 18? yes
Number of subjects for this age range: 9
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 7
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion Criteria

1. Fibrosis score = 3 (METAVIR) or 10 PKa
2. Patients who underwent liver transplantation

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified