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Tests treatment safety and results for Crigler-Najjar Syndrome
Official title Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome
ClinicalTrials.gov ID: NCT06518005
What this study is testing
What is Imlifidase?
Imlifidase is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for crigler-najjar syndrome.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 glucuronosyltransferase (UGT1A1), that causes the accumulation of neurotoxic unconjugated bilirubin (UCB). Reduction of UCB is managed with phenobarbital in mild CNS, and daily phototherapy in severe CNS.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 48 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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
You likely can't join if
- Participation in another treatment 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.
See the full eligibility criteria
- 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
- Participation in another treatment 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
The study team makes the final eligibility decision.
Where it's taking place
- Clamart, France
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 study runs about 48 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Clamart, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.