Status:
RECRUITING
Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
Lead Sponsor:
Genethon
Collaborating Sponsors:
Hansa Biopharma AB
Conditions:
Crigler-Najjar Syndrome
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 glucuro...
Eligibility Criteria
Inclusion
- Main
- Severe Crigler-Najjar syndrome requiring ≥ 6 hours/ day of phototherapy
- Molecular confirmation of mutation in the UGT1A1gene by DNA sequencing
- Detectable serum neutralizing antibodies against AAV8
- Laboratory parameters value not clinically significant
- Highly effective method of contraception
- Affiliated to or a beneficiary of a health care system
Exclusion
- Participation in another interventional trial within 6 months prior to start of clinical trial intervention and during the whole clinical trial
- Fibrosis score ≥ 3 (METAVIR) or 10 kPa (FibroScan®)
- Liver transplantation
- Significant underlying liver disease, chronic hepatitis B, C and/or infected with Human immunodeficiency virus
- Any other clinically significant illness
- Uncontrolled hyperlipidemia.
- History of major thrombotic events, active peripheral vascular disease, proven hypercoagulable conditions,
- History or presence of thrombotic thrombocytopenic purpura (TTP) or known familial history of TTP
- Prior or current treatment with Gene therapy, cell based therapy, CRISPR/Cas9 or any other form of gene editing, imlifidase
Key Trial Info
Start Date :
November 8 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 30 2030
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT06518005
Start Date
November 8 2024
End Date
September 30 2030
Last Update
December 3 2024
Active Locations (1)
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1
Hopital Antoine BECLERE
Clamart, France, 92141