BridgeMD
Long Term Study of Livmarli
EU CTIS ID: 2024-516804-40-00
What this study is testing
The key objectives of this low-intervention clinical study in participants with ALGS who are prescribed Livmarli are: 1. To evaluate tolerability and long-term safety (including possible liver toxicity) 2. To evaluate long-term efficacy The key objectives of this low-intervention clinical study in participants with PFIC who are prescribed Livmarli are: 1. To evaluate tolerability and long-term safety (including possible liver toxicity) 2. To evaluate the impact of Livmarli on long-term clinical outcome events (surgical biliary diversion [SBD],liver transplant [LT], portal hypertension, complications of liver cirrhosis, liver carcinoma, disease progression, liver decompensation, liver-related mortality, and all-cause mortality) 3. To evaluate LT waitlist status 4. To evaluate growth and development 5. To obtain biomarkers of cholestasis, liver fibrosis (APRI and PELD), and liver function 6. To monitor pruritus severity over time 7. To evaluate long-term safety of chronic exposure to propylene glycol (PG)
- Therapeutic use (Phase IV)
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
- Understand and execute an Informed consent and assent (as applicable)
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis (Saleh et al. 2016) or a clinically and/or genetically confirmed PFIC diagnosis (Hassan and Hertel 2022)
- 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 ALGS, ≥2 months of age at Day 1
- For participants with PFIC, ≥3 months of age at Day 1
You likely can't join if
- History of LT
- 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)
- Baseline data before start of treatment of Livmarli are unavailable for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameters
- Received another IBAT inhibitor within 7 days before the first dose of Livmarli
See the full eligibility criteria
- Understand and execute an Informed consent and assent (as applicable)
- A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis (Saleh et al. 2016) or a clinically and/or genetically confirmed PFIC diagnosis (Hassan and Hertel 2022)
- 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 ALGS, ≥2 months of age at Day 1
- For participants with PFIC, ≥3 months of age at Day 1
- For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry
- History of LT
- 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)
- Baseline data before start of treatment of Livmarli are unavailable for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameters
- Received another IBAT inhibitor within 7 days before the first dose of Livmarli
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, 65+ years, 18-64 years, 0-17 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.