Cholestatic Liver DiseasePhase 2MERGENCT04168385
MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
A Phase 2 study testing Maralixibat in people with Cholestatic Liver Disease, run by Mirum Pharmaceuticals, Inc. at 17 sites in 8 countries.
Trial summary
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia. Read more about this study
- Who is it for?
- Ages 12 months and older
- Conditions the study is for
- Cholestatic Liver Disease
- What is being tested?
- Maralixibat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 17 sitesin 8 countries
- Study dates
- 16 Jan 2020to 4 Sep 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- A subject will be excluded from the study if any of the following exclusion criteria are met:
- Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.
- Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study.
- History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.
Trial locations and contacts
17 sites in 8 countries.
17 sites in 8 countries
United States6 sites
Baylor College of Medicine/Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital For Children
Indianapolis, Indiana, 46202
Children'S Hospital Los Angeles
Los Angeles, California, 90027
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Seattle Children's Hospital
Seattle, Washington, 98105
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham
Leeds Teaching Hospital NHS Trust
Leeds, LS1 3EX
Paediatric Liver Center, Kings College Hospital
London, SE5 9RS
Australia2 sites
The Royal Children'S Hospital Melbourne
Parkville, Victoria, 3052
Children's Hospital Westmead
Westmead, New South Wales, 2145
France2 sites
Hopital Necker-Enfants Malades
Paris, 75015
Hopital Kremlin Bicetre
Paris, 94275
Spain1 site
Hospital Universitario La Paz- Hospital Materno Infantil
Madrid, 261
About this study
Official title: MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.
Read the detailed description
This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects received maralixibat in the previous studies and will continues to receive maralixibat in this study.
- Conditions
- Cholestatic Liver Disease
- Being tested
- Maralixibat
- Keywords
- PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric
- Medical subject headings
- Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MaralixibatDrug
Also called Formerly LUM001 and SHP625
Maralixibat chloride oral solution orally twice daily (up to 1200* mcg/kg/day), and according to indication.
*equivalent to 1140 mcg/kg/day maralixibat
| Study group | Role | What they receive |
|---|---|---|
| Maralixibat | Experimental | Participants will all receive Maralixibat oral solution Receives: Drug: Maralixibat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
Measured: From informed consent through approximately 4.5 years, including 30 days after last dose.
Secondary outcomes (2)
Long-Term Effect on Pruritus
Change from Baseline in Pruritus Severity assessed using the Clinician Scratch Score (CSS), a 5-point scale where 0 indicates no evidence of scratching and 4 indicates cutaneous mutilation with bleeding, hemorrhage, and scarring.
Measured: From Baseline through Week 160, including Change from Baseline values.
Long-Term Effect on Serum Bile Acid Levels
Measured: From Baseline through Week 160, including Change from Baseline values.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2019
- Study start16 Jan 2020
- Primary completion4 Sep 2024
- Study completion4 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cholestatic Liver DiseaseStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04168385
- Sponsor's protocol ID
- MRX-800
- Acronym
- MERGE
- Status last verified
- Dec 2025
- First submitted
- 1 Oct 2019
- Last updated
- 8 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MRX-800: A Long-Term Safety Study of Maralixibat, an Apical Sodium Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
- ClinicalTrials.gov ID
- NCT04168385
- Acronym / sponsor ID
- MERGE / MRX-800
- Conditions
- Cholestatic Liver Disease
- Keywords
- PFIC, ALGS, Maralixibat, Bile Duct Diseases, Liver Diseases, Biliary Tract Diseases, Digestive System Diseases, Pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Jan 2020 (actual)
- Primary completion
- 4 Sep 2024 (actual)
- Study completion
- 4 Sep 2024 (actual)
- First posted
- 19 Nov 2019 (actual)
- Results
- Posted 8 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease including, but not limited to, Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC) and Biliary Atresia.
Detailed description
This is a multicenter, open-label study of maralixibat in subjects diagnosed with cholestatic liver disease (including, but not limited to ALGS, PFIC or Biliary Atresia) who have previously participated in a maralixibat clinical study. All subjects received maralixibat in the previous studies and will continues to receive maralixibat in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Maralixibat | Experimental | Drug: MaralixibatParticipants will all receive Maralixibat oral solution |
Who may qualify
- Ages
- 12 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (4 listed)
- A subject will be excluded from the study if any of the following exclusion criteria are met:
- Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.
- Any conditions or abnormalities (including laboratory abnormalities) which, in the opinion of the Investigator or Medical Monitor may compromise the safety of the subject or interfere with the subject participating in or completing the study.
- History of non-adherence to medical regimens, unreliability, medical condition, mental instability or cognitive impairment that, in the opinion of the Investigator or Sponsor medical monitor, could compromise the validity of informed consent, compromise the safety of the subject, or lead to non-adherence with the study protocol or inability to conduct the study procedures.
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 |
|---|---|---|
| MaralixibatAlso: Formerly LUM001 and SHP625Maralixibat chloride oral solution orally twice daily (up to 1200* mcg/kg/day), and according to indication. *equivalent to 1140 mcg/kg/day maralixibat | Drug | Maralixibat |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: From informed consent through approximately 4.5 years, including 30 days after last dose.
TEAE = Treatment-emergent Adverse Event; AESI = Adverse Event of Special Interest..
2 secondary outcomes
Long-Term Effect on Pruritus
Time frame: From Baseline through Week 160, including Change from Baseline values.
Long-Term Effect on Serum Bile Acid Levels
Time frame: From Baseline through Week 160, including Change from Baseline values.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Genetics Home Reference - PFICLink provided by the sponsor.
- Genetics Home Reference - Alagille syndromeLink provided by the sponsor.
- US FDA ResourcesLink provided by the sponsor.
- Mirum Pharmaceuticals homepageLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Cholestatic Liver Disease
Active, not recruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
Not yet recruitingPhase 3
J-LINX: Japan LINerixibat eXtension Study
Pruritus
Not yet recruitingPhase 2
Nemolizumab for the Treatment of Moderate to Severe Chronic Hepatic Pruritus
Chronic Hepatic Pruritus, Cholestatic Liver Disease
RecruitingPhase 1
Hepatic Impairment With Cirrhosis Due to Cholestatic Liver Disease
Hepatic Impairment, Cirrhosis
RecruitingPhase 1
Evaluate PK & Safety of Saroglitazar in Subjects With Moderate Hepatic Impairment Due to Cholestatic Liver Disease
Cholestatic Liver Disease
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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Maralixibat in people with Cholestatic Liver Disease, run by Mirum Pharmaceuticals, Inc. at 17 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 0 inclusion and 4 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: finished, as of the sponsor's last update on 8 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, United Kingdom, Australia, France, Belgium 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: From informed consent through approximately 4.5 years, including 30 days after last dose.). 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 8 Dec 2025.