CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy

CareCN: A phase I/II, open-label, study to evaluate the safety and efficacy of an intravenous injection of GNT0003 (Adeno-associated Viral Vector expressing the UGT1A1 transgene) in patients with severe Crigler-Najjar syndrome requiring phototherapy - Care CN

Status

Recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON52580

Enrollment

Registered

2017-10-09

Start date

2018-05-14

Completion date

Unknown

Last updated

2024-09-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Crigler-Najjar Syndrome

Interventions

One intravenous infusion via peripheral vein of 200 mL of GNT0003 diluted in saline and human serum albumin

Eligibility

Age

2 Years to 99 Years

Inclusion criteria

Inclusion criteria: Main Inclusion criteria [1] Patients with severe Crigler-Najjar syndrome requiring daily phototherapy (>= 6h/day) [2] Molecular confirmation of mutations in the UGT1A1 gene by DNA sequencing

Exclusion criteria

Exclusion criteria: Main Exclusion criteria [1] Fibrosis score >= 3 (METAVIR) or 10 PKa [2] Patients who underwent liver transplantation

Design outcomes

Primary

Measure	Time frame
Primary Endpoint: Dose escalation part: - Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 17 - Change in laboratory parameters, vital signs and in physical examination from baseline to Week 17 Confirmatory part: - The proportion of patients having received the selected dose of GNT0003 with serum total bilirubin = 300 µmol/L at Week 48 after IMP infusion and without phototherapy from Week 16	—

Measure

Time frame

Primary Endpoint: Dose escalation part: - Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent serious adverse events (TESAEs) up to Week 17 - Change in laboratory parameters, vital signs and in physical examination from baseline to Week 17 Confirmatory part: - The proportion of patients having received the selected dose of GNT0003 with serum total bilirubin = 300 µmol/L at Week 48 after IMP infusion and without phototherapy from Week 16

—

Secondary

Measure	Time frame
Secondary Endpoint: Dose escalation part: - Time to GNT0003 vector clearance from blood, urine, saliva and feces - Number of patients with serum total bilirubin	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)