A Phase I/II, Open Label, Escalating Dose Study to Evaluate Safety and Efficacy of an Intravenous Injection of GNT0003 (AAV Vector Expressing the UGT1A1 Transgene) in Patients With Severe Crigler-Najjar Syndrome Requiring Phototherapy

Status: Recruiting
Location: See all (4) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

This is a Phase 1/2, multinational, open-label, escalating-dose 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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 9
Healthy Volunteers: No
View:

• 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

Locations
Other Locations
France
Hopital Antoine BECLERE
Recruiting
Clamart
Italy
ASST Papa Giovanni XXIII
Recruiting
Bergame
Azienda Ospedaliera Universitaria Federico II
Recruiting
Napoli
Netherlands
AMC
Recruiting
Amsterdam
Contact Information
Primary
Genethon Clinical Development Department
clinical_development@genethon.fr
00 33 (0)1 69 47 10 32
Time Frame
Start Date: March 19, 2018
Estimated Completion Date: December 27, 2026
Participants
Target number of participants: 17
Treatments
Experimental: GNT0003
2 doses of the IMP assessed in a dose escalation, open-label, phase 1/2 study
Sponsors
Leads: Genethon

This content was sourced from clinicaltrials.gov

Similar Clinical Trials