Treatment for severe Crigler-Najjar syndrome using gene therapy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the efficacy and safety of GNT0003, a gene therapy designed to correct the genetic defect in patients with severe Crigler-Najjar syndrome, following pre-treatment with Imlifidase to allow access for those with pre-existing antibodies. The trial aims to reduce the accumulation of neurotoxic unconjugated bilirubin by providing a functional copy of the UGT1A1 gene through a recombinant viral vector. Participants will be monitored for safety and effectiveness in reducing bilirubin levels, with the goal of improving patient outcomes without the need for liver transplantation.

Why it matters

Who can join: the full rules

Main Inclusion Criteria:

1. Severe Crigler-Najjar syndrome requiring ≥ 6 hours/ day of phototherapy
2. Molecular confirmation of mutation in the UGT1A1gene by DNA sequencing
3. Detectable serum neutralizing antibodies against AAV8
4. Laboratory parameters value not clinically significant
5. Highly effective method of contraception
6. Affiliated to or a beneficiary of a health care system

Exclusion Criteria:

1. Participation in another interventional trial within 6 months prior to start of clinical trial intervention and during the whole clinical trial
2. Fibrosis score ≥ 3 (METAVIR) or 10 kPa (FibroScan®)
3. Liver transplantation
4. Significant underlying liver disease, chronic hepatitis B, C and/or infected with Human immunodeficiency virus
5. Any other clinically significant illness
6. Uncontrolled hyperlipidemia.
7. History of major thrombotic events, active peripheral vascular disease, proven hypercoagulable conditions,
8. History or presence of thrombotic thrombocytopenic purpura (TTP) or known familial history of TTP
9. Prior or current treatment with Gene therapy, cell based therapy, CRISPR/Cas9 or any other form of gene editing, imlifidase

Where it's running

• Hopital Antoine BECLERE
  Clamart, France Recruiting

Study contacts

• Principal investigator: Philippe LABRUNE, MD, PHD — APHP_Hopital Antoine BECLERE
• Study coordinator: GENETHON Clinical Development Department
• Email: clinicaldevelopment@genethon.fr
• Phone: +33 (0) 1 69 47 10 32

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Gene therapy for severe Crigler-Najjar syndrome

  This study is testing a new gene therapy called GNT0003 to see if it can safely help people aged 10 and older with severe Crigler-Najjar syndrome who need light treatment.

  Recruiting at 4 sites Genethon · Clamart and 3 other locations · NCT03466463

• GNT0004 gene therapy for Duchenne muscular dystrophy in ambulant boys

  This study tests a one-time IV gene therapy (GNT0004) to see if it slows DMD progression in ambulant boys aged 6–10.

  Recruiting at 10 sites Phase 2/3 Genethon · Brussels and 13 other locations · NCT07856329

• Natural history of Duchenne muscular dystrophy in boys aged 4–9

  This project will follow boys aged 4–9 with Duchenne muscular dystrophy for 6–36 months to collect standard clinical, imaging, and lab data and create a baseline before possible gene therapy.

  Recruiting at 12 sites Genethon · Brussels and 14 other locations · NCT03882827

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.