Gene Therapy for Severe Crigler Najjar Syndrome

Plain-language summary

This study looks at a gene therapy called GNT0003 in patients with severe Crigler-Najjar syndrome. Patients at least 9 years of age with confirmed mutations in the UGT1A1 gene who require phototherapy may take part. It is run as a gene therapy study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Recruiting

Sponsor

Genethon

Intervention

GNT0003

Start date

3/19/2018

Estimated completion

3/30/2030

Countries

France, Italy, Netherlands

Related genes

Locations

• Clamart, France
• Bergamo, Italy
• Naples, Italy
• Amsterdam, Netherlands

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

* 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 Criteria:

* 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