Alagille SyndromePhase 2ICONICNCT02160782
Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)
A Phase 2 study testing LUM001 (Maralixibat) in people with Alagille Syndrome, run by Mirum Pharmaceuticals, Inc. at 9 sites in 6 countries.
Trial summary
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX). 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 (Maralixibat)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 31 participants
- Where
- 9 sitesin 6 countries
- Study dates
- 28 Oct 2014to 28 May 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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or female between the ages of 12 months and 18 years inclusive.
- Diagnosis of ALGS.
- Evidence of cholestasis (one or more of the following):
- Total serum bile acid > 3x ULN for age.
- Conjugated bilirubin > 1 mg/dL.
- Fat soluble vitamin deficiency otherwise unexplainable.
Show all 28Show fewer
- GGT > 3x ULN for age.
- Intractable pruritus explainable only by liver disease.
- Females of childbearing potential must have a negative serum pregnancy test during Screening.
- Males and females of child-bearing potential who are sexually active, or are not currently sexually active during the study, but become sexually active during the period of the study and 30 days following the last dose of study drug, must agree and use acceptable contraception during the trial.
- Participant is expected to have a consistent caregiver(s) for the duration of the study.
- Informed consent and assent (per IRB/IEC) as appropriate.
- Access to phone for scheduled calls from study site.
- Caregivers (and age-appropriate participants) must be willing and able to use an eDiary device during the study.
- Caregivers (and age-appropriate participants) must digitally accept the licensing agreement in the eDiary software.
- Caregivers (and age-appropriate participants) must complete at least 10 eDiary reports (morning or evening) during each of two consecutive weeks of the screening period (maximum possible reports = 14 per week).
- Average daily score >2 on the Itch Reported Outcome (ItchRO™) questionnaire (maximum possible daily score of 4) for two consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed.
- Inclusion Criteria for participants to be eligible for the 52-week optional follow-up treatment period:
- Completed the protocol through the Week 48 visit with no safety concerns. Participants who were discontinued due to safety reasons can be rechallenged if blood tests are back to relatively normal values for this patient population and participant does not meet any of the protocol's stopping rules. The decision will be made by the investigator in consultation with the sponsor medical monitor.
- Participants who have undergone a surgical disruption of the enterohepatic circulation will not be eligible to enter into the follow up treatment period.
- Participants who were discontinued for other reasons will be considered for the 52-week optional follow-up treatment period on an individual basis. The decision will be made by the investigator in consultation with the sponsor medical monitor.
- Inclusion Criteria for participants with LUM001dosing interruption <7 days, or >=7 days:
- The Participant has either: completed the protocol through the Week 48 visit with no major safety concerns OR discontinued due to safety reasons judged unrelated to the study drug, and laboratory results have returned to levels acceptable for this patient population or individual and participant does not meet any of the protocol's stopping rules at the time of entry into the follow-up period. The decision will be made by the investigator in consultation with the sponsor medical monitor. [Participants who were discontinued for other reasons will be considered on an individual basis.]
- Females of childbearing potential must have a negative urine or serum pregnancy test (beta- human chorionic gonadotropin [β-hCG]) at the time of entry into the long-term optional follow-up treatment period.
- Males and females of child-bearing potential who are sexually active, or are not currently sexually active during the study, but become sexually active during the period of the study and 30 days following the last dose of study drug, must agree and use acceptable contraception during the trial.
- Informed consent and assent (per IRB/EC) as appropriate.
- Access to phone for scheduled calls from study site.
- Caregivers (and age-appropriate participants) must be willing and able to use an eDiary device during the study.
Who cannot join
- Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
- Surgical interruption of the enterohepatic circulation.
- Previous liver transplant
- Decompensated cirrhosis (ALT >15 x ULN, INR >1.5 [unresponsive to vitamin K therapy], albumin <3.0 g/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy).
- History or presence of other concomitant liver disease.
- History or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism or excretion of drugs, including bile salt metabolism in the intestine (eg, inflammatory bowel disease).
Show all 20Show fewer
- History or presence of gallstones or kidney stones.
- Known diagnosis of human immunodeficiency virus (HIV) infection.
- Cancers, except for in situ carcinoma, or cancers treated at least 5 years prior to Screening with no evidence of recurrence.
- Recent medical history or current status that suggests that the participant may be unable to complete the study.
- Any female who is pregnant or lactating or who is planning to become pregnant during the study period.
- Known history of alcohol or substance abuse.
- Administration of bile acid or lipid binding resins within 28 days prior to screening and throughout the trial.
- Known hypersensitivity to LUM001 or any of its components.
- Receipt of investigational drug, biologic, or medical device within 28 days prior to screening, or 5 half-lives of the study agent, whichever is longer.
- History of non-adherence to medical regimens, unreliability, mental instability or incompetence that could compromise the validity of informed consent or lead to nonadherence with the study protocol based upon investigator judgment.
- Any other conditions or abnormalities which, in the opinion of the investigator or sponsor medical monitor, may compromise the safety of the participant, or interfere with the participant participating in or completing the study.
- Participants weighing over 50 kg at screening.
- Exclusion Criteria for participants with LUM001 dosing interruption >=7 days:
- All exclusion criteria mentioned above apply upon entry into the long-term optional follow-up period, with the exception of participants weighing over 50 kg at screening.
Trial locations and contacts
9 sites in 6 countries.
9 sites in 6 countries
France3 sites
Hopital Femme Mere Enfant De Lyon
Bron, 69677
Hopital Necker-Enfants Malades
Paris, 75015
Hopital Kremlin Bicetre
Paris, 94275
Australia2 sites
The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Children's Hospital Westmead
Westmead, New South Wales, 2145
Spain1 site
Hospital Universitario La Paz- Hospital Materno Infantil
Madrid, 261
United Kingdom1 site
Paediatric Liver Center, Kings College Hospital
London, SE5 9RS
About this study
Official title: Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX).
Read the detailed description
The study is divided into 6 parts: a 6-week open-label, dose escalation period, a 12-week open-label stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, a 26-week long-term stable dosing period, and an a 52-week optional follow-up treatment period, and a long-term optional follow-up treatment period for eligible participants who choose to stay on treatment with LUM001.
- Conditions
- Alagille Syndrome
- Being tested
- LUM001 (Maralixibat), 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.
LUM001 (Maralixibat)Drug
LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD). To be administered Twice Daily (BID) for patients who are eligible.
PlaceboDrug
Placebo will be administered orally once daily during randomized withdrawal period
| Study group | Role | What they receive |
|---|---|---|
| LUM001 (Maralixibat) | Experimental | LUM001, also known as Maralixibat (MRX) will be administered orally once a day (QD) up to 400 microgram per kilogram per day (mcg/kg/day) up to Week 52, followed by an increase in dose orally twice a day (BID) during long-term follow-up based on efficacy (serum bile acid [sBA] level and ItchRO[Obs] score) and safety assessment.
Note: 400 mcg/kg maralixibat chloride is equivalent to 380 mcg/kg free maralixibat. Receives: Drug: LUM001 (Maralixibat) |
| Placebo | Placebo comparator | Placebo will be administered orally once a day during randomized withdrawal period (Week 19 to Week 22) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18
The primary efficacy endpoint of this study was the mean change from Week 18 to Week 22 (the RWD period) of fasting sBA levels in participants who had a reduction in sBA ≥50% from baseline to Week 12 or Week 18 (Modified Intent-to-Treat [MITT] Population). Five participants in the MRX group and 10 participants in the placebo group met the prespecified sBA reduction criteria.
Measured: Week 18 to Week 22
Secondary outcomes (13)
Change From Baseline to Week 18 in Fasting sBA Levels
This secondary efficacy endpoint is the mean change from baseline to Week 18 in fasting sBA levels
Measured: Baseline to Week 18
Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs)
This secondary efficacy endpoint is the change from baseline to Week 18 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).
Measured: Baseline to Week 18
Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt)
This secondary efficacy endpoint is the change from baseline to Week 18 in pruritus as measured by ItchRO(Pt) weekly average morning score
Measured: Baseline to Week 18
Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs)
This secondary efficacy endpoint is the change from Week 18 to Week 22 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).
Measured: Week 18 to Week 22
Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Pt)
This secondary efficacy endpoint is the change from Week 18 to Week 22 in pruritus as measured by ItchRO(Pt) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).
Measured: Week 18 to Week 22
Change From Baseline to Week 18 in Alkaline Phosphatase
This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALP
Measured: Baseline to Week 18
Change From Week 18 to Week 22 in Alkaline Phosphatase
This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in ALP
Measured: Week 18 to Week 22
Change From Baseline to Week 18 in Alanine Aminotransferase
This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALT
Measured: Baseline to Week 18
Change From Week 18 to Week 22 in Alanine Aminotransferase
This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in alanine aminotransferase (ALT)
Measured: Week 18 to Week 22
Change From Baseline to Week 18 in Total Bilirubin
This secondary efficacy endpoint is the mean change from baseline to Week 18 in total bilirubin
Measured: Baseline to Week 18
Change From Week 18 to Week 22 in Total Bilirubin
This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in total bilirubin
Measured: Week 18 to Week 22
Change From Baseline to Week 18 in Direct Bilirubin
This secondary efficacy endpoint is the mean change from baseline to Week 18 in direct bilirubin
Measured: Baseline to Week 18
Change From Week 18 to Week 22 in Direct Bilirubin
This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in direct bilirubin
Measured: Week 18 to Week 22
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2014 (estimated)
- Study start28 Oct 2014
- Primary completion28 May 2020
- Study completion28 May 2020
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 (3)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
- Model description
- Open-label single arm study with a randomized placebo-controlled parallel group period
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Mirum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02160782
- Sponsor's protocol ID
- LUM001-304
- Other IDs
- 2013-005373-43
- Acronym
- ICONIC
- Status last verified
- Jun 2021
- First submitted
- 9 Jun 2014
- Last updated
- 14 Jul 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
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 42002216Kamath BM, Goldstein A, Howard R, Garner W, Vig P, Marden JR, Billmyer E, Anderson A, Kirson N, Jacquemin E, Gonzales E. Maralixibat Treatment Response in Alagille Syndrome is Associated with Improved Health-Related Quality of Life. J Pediatr. 2023 Jan;252:68-75.e5. doi: 10.1016/j.jpeds.2022.09.001. Epub 2022 Sep 10.
PubMed 36096175Gonzales E, Hardikar W, Stormon M, Baker A, Hierro L, Gliwicz D, Lacaille F, Lachaux A, Sturm E, Setchell KDR, Kennedy C, Dorenbaum A, Steinmetz J, Desai NK, Wardle AJ, Garner W, Vig P, Jaecklin T, Sokal EM, Jacquemin E. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021 Oct 30;398(10311):1581-1592. doi: 10.1016/S0140-6736(21)01256-3. Epub 2021 Oct 28.
PubMed 34755627
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term, Open-Label Study With a Double-Blind, Placebo-Controlled, Randomized Drug Withdrawal Period of LUM001 (Maralixibat), an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients With Alagille Syndrome
- ClinicalTrials.gov ID
- NCT02160782
- Acronym / sponsor ID
- ICONIC / LUM001-304
- Conditions
- Alagille Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Mirum Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Oct 2014 (actual)
- Primary completion
- 28 May 2020 (actual)
- Study completion
- 28 May 2020 (actual)
- First posted
- 11 Jun 2014 (estimated)
- Results
- Posted 14 Jul 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a long-term, open-label study with a double-blind, placebo-controlled, randomized drug withdrawal period in children with Alagille Syndrome (ALGS) designed to evaluate the safety and efficacy of LUM001 (Also known as maralixibat or MRX).
Detailed description
The study is divided into 6 parts: a 6-week open-label, dose escalation period, a 12-week open-label stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, a 26-week long-term stable dosing period, and an a 52-week optional follow-up treatment period, and a long-term optional follow-up treatment period for eligible participants who choose to stay on treatment with LUM001.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Open-label single arm study with a randomized placebo-controlled parallel group period
| Arm | Type | Receives |
|---|---|---|
| LUM001 (Maralixibat) | Experimental | Drug: LUM001 (Maralixibat)LUM001, also known as Maralixibat (MRX) will be administered orally once a day (QD) up to 400 microgram per kilogram per day (mcg/kg/day) up to Week 52, followed by an increase in dose orally twice a day (BID) during long-term follow-up based on efficacy (serum bile acid [sBA] level and ItchRO[Obs] score) and safety assessment. Note: 400 mcg/kg maralixibat chloride is equivalent to 380 mcg/kg free maralixibat. |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo will be administered orally once a day during randomized withdrawal period (Week 19 to Week 22) |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (28 listed)
- Male or female between the ages of 12 months and 18 years inclusive.
- Diagnosis of ALGS.
- Evidence of cholestasis (one or more of the following):
- Females of childbearing potential must have a negative serum pregnancy test during Screening.
- Males and females of child-bearing potential who are sexually active, or are not currently sexually active during the study, but become sexually active during the period of the study and 30 days following the last dose of study drug, must agree and use acceptable contraception during the trial.
Key exclusion criteria (20 listed)
- Chronic diarrhea requiring ongoing intravenous fluid or nutritional intervention.
- Surgical interruption of the enterohepatic circulation.
- Previous liver transplant
- Decompensated cirrhosis (ALT >15 x ULN, INR >1.5 [unresponsive to vitamin K therapy], albumin <3.0 g/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy).
- History or presence of other concomitant liver disease.
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 |
|---|---|---|
| LUM001 (Maralixibat)LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD). To be administered Twice Daily (BID) for patients who are eligible. | Drug | LUM001 (Maralixibat) |
| PlaceboPlacebo will be administered orally once daily during randomized withdrawal period | Drug | Placebo |
Primary outcome
Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18
Time frame: Week 18 to Week 22
The primary efficacy endpoint of this study was the mean change from Week 18 to Week 22 (the RWD period) of fasting sBA levels in participants who had a reduction in sBA ≥50% from baseline to Week 12 or Week 18 (Modified Intent-to-Treat [MITT] Population). Five participants in the MRX group and 10 participants in the placebo group met the prespecified sBA reduction criteria.
13 secondary outcomes
Change From Baseline to Week 18 in Fasting sBA Levels
Time frame: Baseline to Week 18
Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs)
Time frame: Baseline to Week 18
Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt)
Time frame: Baseline to Week 18
Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs)
Time frame: Week 18 to Week 22
Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Pt)
Time frame: Week 18 to Week 22
Change From Baseline to Week 18 in Alkaline Phosphatase
Time frame: Baseline to Week 18
Change From Week 18 to Week 22 in Alkaline Phosphatase
Time frame: Week 18 to Week 22
Change From Baseline to Week 18 in Alanine Aminotransferase
Time frame: Baseline to Week 18
Change From Week 18 to Week 22 in Alanine Aminotransferase
Time frame: Week 18 to Week 22
Change From Baseline to Week 18 in Total Bilirubin
Time frame: Baseline to Week 18
Change From Week 18 to Week 22 in Total Bilirubin
Time frame: Week 18 to Week 22
Change From Baseline to Week 18 in Direct Bilirubin
Time frame: Baseline to Week 18
Change From Week 18 to Week 22 in Direct Bilirubin
Time frame: Week 18 to Week 22
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2021.
- 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.
- Derived from this study: PubMed 36096175Kamath BM, Goldstein A, Howard R, Garner W, Vig P, Marden JR, Billmyer E, Anderson A, Kirson N, Jacquemin E, Gonzales E. Maralixibat Treatment Response in Alagille Syndrome is Associated with Improved Health-Related Quality of Life. J Pediatr. 2023 Jan;252:68-75.e5. doi: 10.1016/j.jpeds.2022.09.001. Epub 2022 Sep 10.
- Derived from this study: PubMed 34755627Gonzales E, Hardikar W, Stormon M, Baker A, Hierro L, Gliwicz D, Lacaille F, Lachaux A, Sturm E, Setchell KDR, Kennedy C, Dorenbaum A, Steinmetz J, Desai NK, Wardle AJ, Garner W, Vig P, Jaecklin T, Sokal EM, Jacquemin E. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021 Oct 30;398(10311):1581-1592. doi: 10.1016/S0140-6736(21)01256-3. Epub 2021 Oct 28.
- Individual participant dataSharing planned: No
Related trials for Alagille Syndrome
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingObservational study
Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation
Exocrine Pancreatic Insuficiency
RecruitingObservational study
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
Alagille Syndrome
RecruitingObservational study
A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment
Alagille Syndrome
Active, not recruitingPhase 3
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Alagille Syndrome
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
9 sites in 6 countries: see every site and its status.
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 9 sites in 6 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 28 inclusion and 20 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 14 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 9 sites in France, Australia, Belgium, Poland, Spain and 1 more country. 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 14 Jul 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 Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 (time frame: Week 18 to Week 22). 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 NCT02160782, which also lists 3 linked publications.
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 14 Jul 2021.