Gene Therapy for Crigler-Najjar Syndrome

Phase 1

• Conditions: Severe Crigler Najjar syndrome in patients requiring daily phototherapy (= 6h/day); 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-IT
• Lead Sponsor: GENETHO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-05
• Last Update Posted: 11 January 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

[1] Patients with severe Crigler-Najjar syndrome requiring phototherapy
[2] Molecular confirmation of mutations in the UGT1A1 gene by DNA sequencing
Are the trial subjects under 18? yes
Number of subjects for this age range:
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 kPa
[2] Patients who underwent liver transplantation

Study & Design

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