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Alagille SyndromePhase 2IMAGINE-IINCT02117713

An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

A Phase 2 study testing LUM001 (Maralixibat) in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 11 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Read more about this study

Who is it for?
Ages 1 year to 18 years
Conditions the study is for
Alagille Syndrome
What is being tested?
LUM001 (Maralixibat)
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
34 participants
Where
11 sitesin 2 countries
Study dates
16 Mar 2015to 1 Jun 2020
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 1 year to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Male or female, 12 months to 18 years of age.
  • Competent to provide informed consent and assent (per institutional review board/Ethics Committee [IRB/EC]), as appropriate.
  • Completed participation in the LUM001-301 protocol.
  • Females of childbearing potential must have a negative urine pregnancy test [beta human chorionic gonadotropin (beta-hCG)] at the Baseline Visit.
  • Sexually active females must be prepared to use an effective method of contraception during the trial.
  • Effective methods of contraception are considered to be:
Show all 20Show fewer
  • Hormonal (for example, contraceptive pill, patch, intramuscular implant or injection); or
  • Barrier method, for example, (a) condom with spermicide, or (b) diaphragm, with spermicide; or
  • Intrauterine device (IUD).
  • Participants above the age of assent and caregivers and children must be able to read and understand English or Spanish.
  • Caregivers (and age appropriate participants) must have access to phone for scheduled calls from study site.
  • Caregivers (and age appropriate participants) must be willing and able to complete a daily electronic diary (ItchRO) during the first consecutive 12 weeks of the study and then for 4 consecutive weeks following the Week 24 and Week 44 visits.
  • Caregivers (and age appropriate participants) must digitally accept the licensing agreement in the ItchRO electronic diary software at the outset of the study.
  • Eligible participants must be able to adhere to local Ethics Committee or Institutional Review Board (IRB) blood volume limits for laboratory testing.
  • The participant has completed the protocol either through Week 144, or the End of Trial visit, or has received permission from the sponsor and the Premier Medical monitor to re-enter the study in the long-term, optional follow-up treatment period 2.
  • Females of child-bearing potential must have a negative urine or serum pregnancy test (beta-HCG]) at the time of entry into the long-term optional follow-up treatment period 2.
  • Male and female participants of child-bearing potential who are sexually active, or are not currently sexually active, but become sexually active during the study or for 30 days following the last dose of study drug, must agree to use acceptable contraception during the study.
  • Informed consent and assent (per IRB/EC) as appropriate.
  • Caregivers (and age appropriate participants) must have access to phone for scheduled calls from study site.
  • Caregivers (and age appropriate participants) must be willing to follow the rules of eDiary completion.

Who cannot join

  • Experienced an adverse event or serious adverse event (SAE) related to the study drug during the LUM001-301 protocol that led to the discontinuation of the participant from the core study.
  • Any conditions or abnormalities (including laboratory abnormalities) which in the opinion of the Investigator, Medical Monitor or ChiLDReN Protocol Chair, may compromise the safety of the participant, or interfere with the participant participating in or completing the study.
  • History or known presence of gallstones or kidney stones.
  • History of non-adherence during the participant's participation in the LUM001-301 protocol. Non-adherence is defined by dosing compliance (dosing compliance is calculated by [the total number of doses that were actually taken by the participant] divided by [the total number of doses that should have been taken by the participant] multiplied by 100) of less than 80% in the LUM001-301 protocol.
  • Unlikely to comply with the study protocol, or unsuitable for any other reason, as judged by the investigator.
  • All above exclusion criteria will apply upon re-entry into the long-term, optional follow-up treatment period 2.

Trial locations and contacts

11 sites in 2 countries.

11 sites in 2 countries

United States10 sites

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • 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, 19147

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224

  • University of Utah

    Salt Lake City, Utah, 84113

  • University of California at San Francisco Children's Hospital

    San Francisco, California, 94143

  • Seattle Children's Hospital

    Seattle, Washington, 98105

Other trials in United States

Canada1 site

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

Other trials in Canada

About this study

Official title: A Multicenter Extension Study to Evaluate the Long-Term Safety and Durability of the Therapeutic Effect of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in the Treatment of Cholestatic Liver Disease in Pediatric Subjects With Alagille Syndrome

This is a multicentre, extension study of LUM001 in children diagnosed with Alagille Syndrome who have completed participation in a core LUM001 treatment protocol. The primary objective is to evaluate long-term safety and tolerability of LUM001. Efficacy will be assessed by evaluating the effect of LUM001 on the biochemical markers and pruritus associated with Alagille Syndrome.

Being tested
LUM001 (Maralixibat)
Medical subject headings
Alagille Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LUM001 (Maralixibat)Drug

    Dosing of LUM001 also known as Maralixibat (MRX) with the objective of achieving optimal control of pruritus at a dose level that is tolerated by the participant and up to a maximum daily dose of 280 micrograms per kilogram (mcg/kg).

Study groups in NCT02117713
Study groupRoleWhat they receive
LUM001 (Maralixibat)ExperimentalParticipant will receive LUM001 also known as Maralixibat (MRX) administered orally once per day.
Receives: Drug: LUM001 (Maralixibat)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA)

    This primacy efficacy endpoint is the mean change from MRX baseline to week 48 in fasting sBA levels.

    Measured: Baseline to Week 48

Secondary outcomes (10)

  1. Change From MRX Baseline to Week 216 in Fasting Serum Bile Acid (sBA)

    The secondary endpoint of this study was the mean change from MRX baseline to week 216 fasting in sBA levels.

    Measured: Baseline to week 216

  2. Change From Baseline to Week 218 in Pruritus

    This secondary efficacy endpoint is the mean change from MRX baseline over time to week 218 in pruritus as measured by ItchRO(Obs) weekly average morning severity score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe). Results reported here are the long-term results.

    Measured: Baseline to Week 218

  3. Change From Baseline to Week 216 in Alanine Aminotransferase

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALT levels.

    Measured: Baseline to week 216

  4. Change From Baseline to End of Treatment in Alkaline Phosphatase

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in ALP levels.

    Measured: Baseline to Week 216

  5. Change From MRX Baseline to Week 216 in Aspartate Aminotransferase

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in AST levels.

    Measured: Baseline to week 216

  6. Change From MRX Baseline to Week 216 in Clinician Xanthoma Severity Score

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in clinician xanthoma severity scores. It is based on a 0-4 scale to rate the number of lesions present and the degree to which the lesions interfere or limit activities. Clinician xanthoma severity scores range from 0 to 4, with a score of zero representing no evidence of xanthomatosis and a score of 4 representing xanthoma so severe that it is disabling.

    Measured: Baseline to week 216

  7. Change From Baseline to Week 216/LOFC Clinician Scratch Scale (CSS) Score

    This secondary efficacy endpoint is the mean change from MRX baseline over time to week 216/LOCF in pruritus as measured by the Clinician Scratch Scale (CSS). The Clinician Scratch Scale uses a 5-point scale, where 0 = none; 1 = rubbing or mild scratching when undistracted; 2 = active scratching without evident skin abrasions; 3 = abrasion evident; 4 = cutaneous mutilation, haemorrhage and scarring evident.

    Measured: Baseline to Week 216

  8. Change From MRX Baseline to Week 216 in Gamma Glutamyltransferase

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in GGT.

    Measured: Baseline to Week 216

  9. Mean Change From MRX Baseline to Week 216 in Total Bilirubin

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in total bilirubin.

    Measured: Baseline to week 216

  10. Mean Change From MRX Baseline to Week 216 in Direct Bilirubin

    This secondary efficacy endpoint is the mean change from MRX baseline to week 216 in direct bilirubin.

    Measured: Baseline to week 216

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Apr 2014 (estimated)
  2. Study start16 Mar 2015
  3. Primary completion1 Jun 2020
  4. Study completion1 Jun 2020

Resources

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
Double blind
Who is masked
Participant, Investigator
Enrolment
34 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02117713
Sponsor's protocol ID
LUM001-305
Other IDs
SHP625-305
Acronym
IMAGINE-II
Status last verified
Jun 2021
First submitted
16 Apr 2014
Last updated
1 Jul 2021
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

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

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing LUM001 (Maralixibat) in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 11 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 20 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: finished, as of the sponsor's last update on 1 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 11 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. 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 22 Jun 2021). 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 MRX Baseline to Week 48 in Fasting Serum Bile Acid (sBA) (time frame: Baseline to Week 48). 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 NCT02117713, 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 1 Jul 2021.

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