Alagille SyndromePhase 4NCT07290257
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)
A Phase 4 study testing Livmarli Oral Product in people with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, run by Mirum Pharmaceuticals, Inc. at 15 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 2 months and older
- Conditions the study is for
- Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
- What is being tested?
- Livmarli Oral Product
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 230 participants
- Where
- 15 sitesin 8 countries
- Study dates
- 25 Sep 2025to 22 Dec 2030
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 efficacy (sBA, pruritus) parameter
Trial locations and contacts
15 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
15 sites in 8 countries
France3 sites, 3 recruiting
- Recruiting
Hospices Civils de Lyon - Hopital Femme Mère Enfant
Bron, Auvergne-Rhône-Alpes, 69500
Mathias Ruiz, +33 472 35 70 50, mathias.ruiz@chu-lyon.fr
- Recruiting
Bicetre University Hospital
Le Kremlin-Bicêtre, Île-de-France Region, 94270
Emmanuel Gonzales, +33 1 45 21 37 88, emmanuel.gonzales@aphp.fr
- Recruiting
CHU de Toulouse - Hôpital des Enfants
Toulouse, Occitanie, 31049
Nolwenn Laborde, +33.(0)5 34558566, laborde.n@chu-toulouse.fr
Germany3 sites, 3 recruiting
- Recruiting
Charite Berlin
Berlin, 13353
Philip Bufler, philip.bufler@charite.de
- Recruiting
Children's University Hospital Essen
Essen, 45149
Elke Lainka, +49 (0)201 723 3360, Elke.Lainka@uk-essen.de
- Recruiting
University Hospital Hamburg-Eppendorf
Hamburg, 20246
Sebastian Schulz-Jürgensen, +49 40 7410 52702, s.schulz-juergensen@uke.de
Belgium2 sites, 2 recruiting
- Recruiting
Cliniques Universitaires Saint Luc (UCLouvain)
Brussels, 1200
Stephenne Xavier, +32 2 764 13 77, xavier.stephenne@uclouvain.be
- Recruiting
University Hospital Gent (UZ Gent)
Ghent, 9000
Ruth De Bruyne, +32 9 332 39 66, ruth.debruyne@ugent.be
Italy2 sites, 2 recruiting
- Recruiting
AO Ospedale PAPA GIOVANNI XXIII
Bergamo, Lombardy, 24126
Lorenzo D'Antiga, ldantiga@asst-pg23.it
- Recruiting
Istituto mediterraneo trapianti - ISMETT
Palermo, Sicily, 90127
Giusy Ranucci, granucci@ismett.edu
Spain2 sites, 2 recruiting
- Recruiting
Hospital Universitairo Vall D'Hebron
Barcelona, Catalonia, 08035
Jesus Quintero, jesus.quintero@vallhebron.cat
- Recruiting
Hospital Universitario La Paz
Madrid, 28046
Esteban Frauca Remacha, esteban.frauca@salud.madrid.org
Greece1 site, 1 recruiting
- Not yet recruiting
2nd Paediatric Clinic P &A Kyriakou Children's Hospital
Athens, 11527
Aglaia Zellos
Netherlands1 site, 1 recruiting
- Recruiting
University Medical Center Groningen (UMCG)
Groningen, 9713GZ
Henkjan Verkade, +31 50 3614147, h.j.verkade@umcg.nl
Portugal1 site, 1 recruiting
- Not yet recruiting
Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo
Coimbra, 3000-602
Isabel Goncalves Costa
About this study
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.
Read the detailed description
This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.
Participants will be treated according to standard of care for the duration of the study.
- Being tested
- Livmarli Oral Product
- Keywords
- ALGS, Liver Diseases, Alagille Syndrome, Livmarli, Maralixibat, Cholestasis, Pruritus, Itching, IBAT inhibitor, PFIC
- Medical subject headings
- Alagille Syndrome, Cholestasis, progressive familial intrahepatic 1, Liver Diseases, Cholestasis, Pruritus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Livmarli Oral ProductDrug
Also called Maralixibat
Participants will be treated according to standard of care for the duration of the study and Schedule of Assessments.
| Study group | Role | What they receive |
|---|---|---|
| Livmarli | Experimental | ALGS: Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli.
PFIC: Patients with Progressive Familial Intrahepatic Cholestasis treated with Livmarli. The study will enroll a minimum of 130 patients with PFIC Receives: Drug: Livmarli Oral Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events (AEs)
Number and proportion of participants with AEs.
Measured: Up to 7 days after the last dose of Livmarli.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Total and direct bilirubin
Measured: Once every 3-4 months, for 5 years.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Change in LFTs from Baseline: Aminotransferases and GGT.
Measured: Once every 3-4 months, for 5 years.
Fat-Soluble Vitamins (FSV) Concentration
Change in FSV levels (vitamins A, D, and E) from Baseline.
Measured: Once every 3-4 months, for 5 years.
International Normalized Ratio (INR)
Change in INR level from Baseline.
Measured: Once every 3-4 months, for 5 years.
Long-Term Clinical Outcomes
Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.
Measured: Once every 3-4 months, for 5 years.
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC
Change in PG level from Baseline.
Measured: Once every 3-4 months, for 5 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2025
- Study start25 Sep 2025
- Primary completion22 Sep 2030 (estimated)
- Study completion22 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- More trials run by Mirum Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a single-group, open-label study enrolling participants with ALGS and PFIC treated with Livmarli. For ALGS, the primary cohort will include at least 45 treatment-naïve participants. A supplemental ALGS cohort will include participants previously treated with Livmarli, with no enrollment limit. For PFIC, the study will enroll at least 130 participants across PFIC subtypes, and all participants enrolled for at least 1 year will be included in the interim analysis.
- Masking
- None (open label)
- Enrolment
- 230 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07290257
- Sponsor's protocol ID
- MRX-803
- Status last verified
- Jun 2026
- First submitted
- 17 Sep 2025
- Last updated
- 23 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- Acronym / sponsor ID
- MRX-803
- Keywords
- ALGS, Liver Diseases, Alagille Syndrome, Livmarli, Maralixibat, Cholestasis, Pruritus, Itching
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 230 participants (estimated)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 Sep 2025 (actual)
- Primary completion
- 22 Sep 2030 (estimated)
- Study completion
- 22 Dec 2030 (estimated)
- First posted
- 18 Dec 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a multicenter, open-label, low-intervention clinical study in participants diagnosed with either ALGS with cholestatic pruritus or PFIC who are treated with Livmarli.
Participants will be treated according to standard of care for the duration of the study.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a single-group, open-label study enrolling participants with ALGS and PFIC treated with Livmarli. For ALGS, the primary cohort will include at least 45 treatment-naïve participants. A supplemental ALGS cohort will include participants previously treated with Livmarli, with no enrollment limit. For PFIC, the study will enroll at least 130 participants across PFIC subtypes, and all participants enrolled for at least 1 year will be included in the interim analysis.
| Arm | Type | Receives |
|---|---|---|
| Livmarli | Experimental | Drug: Livmarli Oral ProductALGS: Primary cohort with a minimum of 45 previously untreated (treatment naïve). Supplemental cohort of participants who have previously received Livmarli. PFIC: Patients with Progressive Familial Intrahepatic Cholestasis treated with Livmarli. The study will enroll a minimum of 130 patients with PFIC |
Who may qualify
- Ages
- 2 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Livmarli Oral ProductAlso: MaralixibatParticipants will be treated according to standard of care for the duration of the study and Schedule of Assessments. | Drug | Livmarli |
Primary outcome
Adverse Events (AEs)
Time frame: Up to 7 days after the last dose of Livmarli.
Number and proportion of participants with AEs.
Liver function tests (LFTs) - measuring enzyme activity or concentration
Time frame: Once every 3-4 months, for 5 years.
Change in LFTs from Baseline: Total and direct bilirubin
Liver function tests (LFTs) - measuring enzyme activity or concentration
Time frame: Once every 3-4 months, for 5 years.
Change in LFTs from Baseline: Aminotransferases and GGT.
Fat-Soluble Vitamins (FSV) Concentration
Time frame: Once every 3-4 months, for 5 years.
Change in FSV levels (vitamins A, D, and E) from Baseline.
International Normalized Ratio (INR)
Time frame: Once every 3-4 months, for 5 years.
Change in INR level from Baseline.
Long-Term Clinical Outcomes
Time frame: Once every 3-4 months, for 5 years.
Number of participants with surgical biliary diversion, liver transplantation, liver transplant waitlist status change, clinically evident portal hypertension, complications of liver cirrhosis, liver carcinoma, liver decompensation, and death.
Laboratory Test for Monitoring of Propylene Glycol Toxicity in Participants with PFIC
Time frame: Once every 3-4 months, for 5 years.
Change in PG level from Baseline.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
15 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Livmarli Oral Product in people with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis, run by Mirum Pharmaceuticals, Inc. at 15 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in France, Germany, Belgium, Italy, Spain and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trials Mirum (+16506674085, clinicaltrials@mirumpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events (AEs) (time frame: Up to 7 days after the last dose of Livmarli.). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2026.