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Efficacy and safety trial of GNT0003 following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar syndrome presenting pre-existing anti-AAV8 antibodies.
EU CTIS ID: 2023-510405-18-00
What this study is testing
To assess the efficacy of a single intravenous administration of GNT0003 following imlifidase pre-treatment in participants with severe CNS requiring phototherapy and pre-existing AAV8 antibodies
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female aged 16 years and older at the time of screening.
- 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.
You likely can't join if
- 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.
See the full eligibility criteria
Who can join
- Male or female aged 16 years and older at the time of screening.
- 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 (of GNT-018-IDES clinical trial and GNT-018-IDES-PS performance study) which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
What rules you out
- 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, immunosuppressants, 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
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.