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Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

StatusRecruiting
PhasePhase 2
SponsorGenethon
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated December 3, 2024 (before its estimated September 30, 2026 completion). Its status may not reflect the trial's current state.

Record History

  1. Notable Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Oct 15, 2024 to Nov 8, 2024
    • Site details revised
    v3
  2. Minor Interventions: Imlifidase and GNT0003 (Drug) to GNT0003 (Drug), Imlifidase (Drug)
    • Study arms: 1 to 2
    v2
  3. Minor Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
9 trials monitored
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Clinical trial rationale: CNS is an ultra-rare (\<1/1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that causes the accumulation of neurotoxic unconjugated bilirubin (UCB). Reduction of UCB is managed with phenobarbital in mild CNS, and daily phototherapy in severe CNS. There is no authorized curative medical treatment for CNS. Liver transplantation is currently the only curative treatment for severe CNS. GNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene. Imlifidase (IgG-degrading enzyme) has demonstrated its efficacy in highly sensitized adult kidney transplant patients. To give participants with pre-existing anti-AAV8 antibodies access to gene therapy treatments, this trial aims to demonstrate the safety and efficacy of GNT0003 following imlifidase pre-treatment in adult participants with severe CNS requiring daily phototherapy and presenting with pre-existing anti-AAV8 antibodies. Primary objective: to assess efficacy of a single intravenous administration of GNT0003 following imlifidase pre-treatment in participants with severe CNS requiring phototherapy and pre-existing AAV8 antibodies Secondary objective: to collect data on safety and tolerability of GNT0003 and imlifidase, efficacy of imlifidase, pharmacokinetic and pharmacodynamic profile of GNT0003, and Quality of Life. The trial will include 3 parts: * A baseline period for at least 3 months * A treatment period * A follow-up period: * Initial post-treatment follow-up over 48 weeks * Long-term follow-up for 4 additional years This trial will be conducted in accordance with the International Conference on Harmonization Guideline for Good Clinical Practice and the Declaration of Helsinki. Participants must be consented using the approved Informed Consent Form before any procedures specified in the protocol are performed.
Trial Details
NCT Number NCT06518005
Lead Sponsor Genethon
Collaborators: Hansa Biopharma AB
Conditions
(from sponsor)
Crigler-Najjar Syndrome
Related terms Crigler-Najjar Syndrome
Enrollment 3 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov