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Tests treatment safety and results for Alagille Syndrome
Official title Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
ClinicalTrials.gov ID: NCT07290257
What this study is testing
What is Livmarli Oral Product?
Livmarli Oral Product is an investigational medicine, being studied as a potential treatment for alagille syndrome.
Also referred to as Maralixibat.
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
- In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment. In patients with Progressive Familial Intrahepatic Cholestasis (PFIC), the key objectives are to evaluate tolerability, long-term safety (including possible liver toxicity and potential impact of chronic exposure to propylene glycol (PG)), long-term efficacy, and growth and development.
- Phase 4: studies an already-approved treatment
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 and older
You may be able to join if
- Understand and execute an Informed consent and assent (as applicable)
- For participants with ALGS ≥2 months of age at Day 1
- For participants with PFIC, ≥3 months of age at Day 1
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed...
- For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
You likely can't join if
- History of Liver Transplant
- Any Livmarli contraindications (as per SmPC)
- Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
- Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access...
- Received another IBAT inhibitor within 7 days before the first dose of Livmarli
- Baseline data before start of treatment of Livmarli are unavailable (\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and...
See the full eligibility criteria
- Understand and execute an Informed consent and assent (as applicable)
- For participants with ALGS ≥2 months of age at Day 1
- For participants with PFIC, ≥3 months of age at Day 1
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
- For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
- For the ALGS supplemental cohort, : Actively using Livmarli prior to study entry
- For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry
- History of Liver Transplant
- Any Livmarli contraindications (as per SmPC)
- Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
- Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
- Received another IBAT inhibitor within 7 days before the first dose of Livmarli
- Baseline data before start of treatment of Livmarli are unavailable (\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key how well it works (sBA, pruritus) parameter
The study team makes the final eligibility decision.
Where it's taking place
- Brussels, Belgium
- Ghent, Belgium
- Bron, Auvergne-Rhône-Alpes, France
- Toulouse, Occitanie, France
- Le Kremlin-Bicêtre, Île-de-France Region, France
- Berlin, Germany
- Essen, Germany
- Hamburg, Germany
- Athens, Greece
- Bergamo, Lombardy, Italy
- Palermo, Sicily, Italy
- Groningen, Netherlands
- Coimbra, Portugal
- Barcelona, Catalonia, Spain
- Madrid, Spain
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, 2 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Brussels, Belgium; Ghent, Belgium; Bron, Auvergne-Rhône-Alpes, France; Toulouse, Occitanie, France; Le Kremlin-Bicêtre, Île-de-France Region, France; Berlin, Germany and 9 more location(s). 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.