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Gene Therapy for Severe Crigler Najjar Syndrome

NCT03466463 · Status: RECRUITING · Phase: NA · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2023-03-28

No results posted yet for this study

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 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

  • Crigler-Najjar Syndrome

Interventions

GENETIC

GNT0003

Intravenous infusion, single dose

Sponsors & Collaborators

  • Genethon

    lead OTHER

Principal Investigators

  • LABRUNE Philippe, Prof · Hopital Antoine Beclere

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
9 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-03-19
Primary Completion
2026-03-30
Completion
2030-03-30

Countries

  • France
  • Italy
  • Netherlands

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03466463 on ClinicalTrials.gov