Efficacy and safety trial of GNT0003 following imlifidase pre-treatment in adult participants with severe Crigler-Najjar syndrome presenting pre-existing anti-AAV8 antibodies.
- Conditions
- Crigler-Najjar syndromeMedDRA version: 20.0Level: PTClassification code: 10011386Term: Crigler-Najjar syndrome Class: 100000004850Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- CTIS2023-510405-18-00
- Lead Sponsor
- Genethon
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 3
Male or female must be = 18 years old or the legal age of consent in the jurisdiction in which the trial is taking place at the time of signing the informed consent., Participant with severe Crigler-Najjar syndrome requiring = 6 hours/ day of phototherapy., Participant with molecular confirmation of mutation in the UGT1A1 gene by DNA sequencing., Participant with detectable serum neutralizing antibodies against AAV8., Participant with laboratory parameters value not clinically significant, as assessed by the investigator, and meeting the following criteria, a.Hematology, clinical chemistry and coagulation = grade 1 (as per Common Terminology Criteria for Adverse Events [CTCAE] criteria) (including but not limited to: activated partial thromboplastin time =1.5 x ULN, creatinine increased =1.5 x ULN, Platelet count decreased =75,000/mm3, lymphocyte count increased =20 000/mm3). b.Alanine amino-transferase (ALT), aspartate amino-transferase (AST), gamma-glutamyl transferase (GGT) = grade 3 (as per CTCAE criteria: GGT, ALT and/or AST =20.0 x ULN if baseline was normal; =20.0 x baseline if baseline was abnormal)., Participants must agree to use a highly effective method of contraception from screening visit (V1) to at least 48 weeks after start of IMP administration., Signed the study performance informed consent., Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Participation in another interventional trial during this clinical trial, including investigational trial involving gene or cell therapeutics within 6 months prior to start of IMP administration and during the whole clinical trial; participation in non-interventional registries or epidemiological studies is allowed., Participant presents or has a history of thrombotic thrombocytopenic purpura (TTP) or known familial history of TTP, Fibrosis score = 3 (METAVIR) or 10 kPa based on: a)Fibrotest: performed within 12 months prior to screening visit 1* Or b)FibroScan®: performed within 12 months prior to screening visit 1* Or c)Liver biopsy taken within 24 months prior to screening visit 1*, Participant with significant underlying liver disease., Presence of any other clinically significant illness., Participant with a history of major thrombotic events, active peripheral vascular disease, or proven hypercoagulable conditions., Participant with known hypersensitivity to imlifidase and its excipients., Contraindication to corticosteroids, sirolimus, imlifidase, or antihistamines., Treatment with any of the prior/concomitant therapies according to the time frames specified in the protocol. At any time : Gene therapy, Cell based therapy (e.g., stem cell transplantation), CRISPR/Cas9, or any other form of gene editing, imlifidase, Participant who underwent liver transplantation
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method