Gene Therapy for Severe Crigler Najjar Syndrome
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: GNT0003.
- Who it may be relevant to
- Registry conditions: Crigler-Najjar Syndrome. Basic parameters: from 9 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France, Italy, Netherlands
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase I/II, Open Label, 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
Overview
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.
Interventions
- Genetic GNT0003
Intravenous infusion, single dose
Primary outcome measures
- Proportion of patients having received the selected dose of GNT0003 with serum bilirubin ≤ 300µmol/L within 48 meeks after GNT0003 administration and without phototherapy from week 16 [Time frame: 48 weeks]
- Incidence of Treatment Emergent Adverse Events or Treatement Serious Adverse Events [Time frame: 48 weeks]
Secondary outcome measures (2)
- Change in Health-related quality of Life for Adults from Baseline to Week 48 after GNT0003 administration [Time frame: 48 weeks]
- Change in Health-related quality of Life for Children from Baseline to Week 48 after GNT0003 administration [Time frame: 48 weeks]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Other
Study locations
Italy · 2 centers
- ASST Papa Giovanni XXIII — Bergamo
- Azienda Ospedaliera Universitaria Federico II — Naples
France · 1 center
- Hopital Antoine BECLERE — Clamart
Netherlands · 1 center
- AMC — Amsterdam
Publications
- D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. Gene Therapy in Patients with the Crigler-Najjar Syndrome. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. PMID 37585628
Identifiers
NCT: NCT03466463 · GNT-012-CRIG