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
Recruiting
- Conditions
- 10027424Crigler-Najjar Syndrome
- Registration Number
- NL-OMON52580
- Lead Sponsor
- Généthon
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 5
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
Main Exclusion criteria
[1] Fibrosis score >= 3 (METAVIR) or 10 PKa
[2] Patients who underwent liver transplantation
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary Endpoint:<br /><br><br /><br>Dose escalation part:<br /><br>- Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent<br /><br>serious adverse events (TESAEs) up to Week 17<br /><br>- Change in laboratory parameters, vital signs and in physical examination from<br /><br>baseline to Week 17<br /><br><br /><br>Confirmatory part:<br /><br>- The proportion of patients having received the selected dose of GNT0003 with<br /><br>serum total bilirubin = 300 µmol/L at Week 48 after IMP<br /><br>infusion and without phototherapy from Week 16</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Endpoint:<br /><br><br /><br>Dose escalation part:<br /><br>- Time to GNT0003 vector clearance from blood, urine, saliva and feces<br /><br>- Number of patients with serum total bilirubin <= 300 µmol/L within 7 days<br /><br>after interruption of daily phototherapy<br /><br><br /><br>Confirmatory part:<br /><br>- Incidence of treatment-emergent adverse events (TEAEs) or treatment-emergent<br /><br>serious adverse events (TESAEs) up to Week 48<br /><br>- Change in laboratory parameters, vital signs and in the physical examination<br /><br>from baseline to Week 48<br /><br>- Change in serum total bilirubin from baseline to Week 48<br /><br>- Change in bilirubin/albumin ratio from baseline to Week 48<br /><br>- Change in Health-related quality of life, as measured by SF-36 (adult) and<br /><br>PedsQL (pediatric), at Weeks 24 and 48<br /><br>- Time to GNT0003 vector clearance from blood, urine, saliva and feces</p><br>