Alagille SyndromePhase 2ITCHNCT02057692
Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome
A Phase 2 study testing LUM001 in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 13 sites in 2 countries.
Trial summary
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS. Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Alagille Syndrome
- What is being tested?
- LUM001
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 13 sitesin 2 countries
- Study dates
- 24 Nov 2014to 16 Nov 2016
- 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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of Alagille Syndrome
- Evidence of cholestasis
- Moderate to severe pruritus
- Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures
Who cannot join
- Surgical disruption of the enterohepatic circulation
- Liver transplant
- History or presence of other concomitant liver disease
- Females who are pregnant or lactating
- Known HIV infection
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States12 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
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
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
University of Utah
Salt Lake City, Utah, 84113
University of California at San Francisco
San Francisco, California, 94143
Seattle Children's Hospital
Seattle, Washington, 98105
About this study
Official title: The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
- Conditions
- Alagille Syndrome
- Being tested
- LUM001, Placebo
- Medical subject headings
- Alagille Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LUM001Drug
LUM001 administered orally
PlaceboDrug
Placebo administered orally
| Study group | Role | What they receive |
|---|---|---|
| LUM001 | Experimental | LUM001 for oral administration Receives: Drug: LUM001 |
| Placebo | Placebo comparator | Placebo administered orally once each day Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
Measured: Baseline, Week 13/Early Termination
Secondary outcomes (3)
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level
Fasting sBA level was measured by using a liquid chromatography mass spectrometry method.
Measured: Baseline, Week 13/Early Termination
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels
Liver enzyme levels of alanine aminotransferase, alkaline phosphatase, aspartate aminotransferase and gamma glutamyl transferase were reported here.
Measured: Baseline, Week 13/Early Termination
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations
Liver enzyme levels of total bilirubin and direct bilirubin were reported here.
Measured: Baseline, Week 13/Early Termination
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2014 (estimated)
- Study start24 Nov 2014
- Primary completion16 Nov 2016
- Study completion16 Nov 2016
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Childhood Liver Disease Research and Education Network
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Mirum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02057692
- Sponsor's protocol ID
- LUM001-301
- Acronym
- ITCH
- Status last verified
- Mar 2019
- First submitted
- 5 Feb 2014
- Last updated
- 26 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Hoskins BJ, Mogul DB, Chen C, Karnsakul W. Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials. J Pediatr. 2026 Jul;294:115108. doi: 10.1016/j.jpeds.2026.115108. Epub 2026 Apr 17.
PubMed 42002216
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Evaluation of the Intestinal Bile Acid Transport (IBAT) Inhibitor LUM001 in the Reduction of Pruritus in Alagille Syndrome, a Cholestatic Liver Disease
- ClinicalTrials.gov ID
- NCT02057692
- Acronym / sponsor ID
- ITCH / LUM001-301
- Conditions
- Alagille Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Childhood Liver Disease Research and Education Network
- Study start
- 24 Nov 2014 (actual)
- Primary completion
- 16 Nov 2016 (actual)
- Study completion
- 16 Nov 2016 (actual)
- First posted
- 7 Feb 2014 (estimated)
- Results
- Posted 23 Jan 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is a randomized, double-blind, placebo-controlled study in children with Alagille Syndrome (ALGS). The study will investigate the effects of LUM001, compared to placebo, on pruritus, serum bile acids, liver enzymes, and other biochemical markers in patients with ALGS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LUM001 | Experimental | Drug: LUM001LUM001 for oral administration |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo administered orally once each day |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Diagnosis of Alagille Syndrome
- Evidence of cholestasis
- Moderate to severe pruritus
- Ability to understand and willingness to sign informed consent/assent prior to initiation of any study procedures
Key exclusion criteria (5 listed)
- Surgical disruption of the enterohepatic circulation
- Liver transplant
- History or presence of other concomitant liver disease
- Females who are pregnant or lactating
- Known HIV infection
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 |
|---|---|---|
| LUM001LUM001 administered orally | Drug | LUM001 |
| PlaceboPlacebo administered orally | Drug | Placebo |
Primary outcome
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus
Time frame: Baseline, Week 13/Early Termination
Pruritus was assessed using Itch report outcome measure (ItchRO[Obs]), administered as an electronic diary (eDiary) which was completed by the participants twice daily (morning and evening). ItchRO(Obs) score ranged from 0 to 4, with the higher score indicating increasing itch severity. The highest score between the morning and evening ItchRO(Obs) reports represented the daily score: a measure of the worst itching over the previous 24-hour period.
3 secondary outcomes
Change From Baseline to Endpoint (Week 13/Early Termination) in Fasting Serum Bile Acid (sBA) Level
Time frame: Baseline, Week 13/Early Termination
Change From Baseline to Endpoint (Week 13/Early Termination) in Liver Enzyme Levels
Time frame: Baseline, Week 13/Early Termination
Change From Baseline to Endpoint (Week 13/Early Termination) in Total and Direct Bilirubin Concentrations
Time frame: Baseline, Week 13/Early Termination
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42002216Hoskins BJ, Mogul DB, Chen C, Karnsakul W. Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials. J Pediatr. 2026 Jul;294:115108. doi: 10.1016/j.jpeds.2026.115108. Epub 2026 Apr 17.
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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. This study is not recruiting at the moment.
Study Director study director
Mirum
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LUM001 in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 13 sites in 2 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 4 inclusion and 5 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 26 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Canada. 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. Some participants in this study may receive a placebo. 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 23 Jan 2018). 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?
Change From Baseline to Endpoint (Week 13/Early Termination) in Pruritus (time frame: Baseline, Week 13/Early Termination). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02057692, which also lists 1 linked publication.
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 26 Mar 2019.