An interventional study of GNT0003 in Crigler-Najjar Syndrome, sponsored by Genethon. Recruiting at 4 sites in 3 countries. Open to participants aged 9 Years and older. Per ClinicalTrials.gov, last updated 2023-03-28.
Sponsored by Genethon · Not applicable, Interventional, and Other
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):
Exclusion Criteria:
2 doses of the IMP assessed in the dose escalation, open-label, phase 1/2 study
Genetic: GNT0003
Intravenous infusion, single dose
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
Decrease of total Serum bilirubin level after interruption of daily phototherapy (Efficacy); change in serum total biliirubin from baseline to week 48
Time frame: 48 weeks
Incidence of Treatment Emergent Adverse Events or Treatement Serious Adverse Events
Incidence of AE/SAE evaluated by changes in laboratory parameters, vital signs, physical examination, reported from baseline to each visit study. Clinically relevant abnormal findings on Laboratory values, Vital Signs, Physical findings will be reported as Adverse Events. Incidence and Severity of Adverse Events for each body system will be presented for each dose level and summarized overall.
Time frame: 48 weeks
Change in Health-related quality of Life for Adults from Baseline to Week 48 after GNT0003 administration
Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration. Scale 36-Item Short Form Survey (SF-36 Health Survey) for adults: from 0 (negative to health) to 100 (positive to health)
Time frame: 48 weeks
Change in Health-related quality of Life for Children from Baseline to Week 48 after GNT0003 administration
Quality of Life outcome measure: change of quality of life from Baseline to Week 48 after GNT0003 administration. PedsQL 4.0 Generic Core Scale for pediatrics: 0-100 scale where higher scores indicate better health-related quality of life
Time frame: 48 weeks
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Genethon