NCT02160782 (ICONIC) is a Phase 2 clinical trial of LUM001 (Maralixibat) in Alagille Syndrome, sponsored by Mirum Pharmaceuticals, Inc..

Who is sponsoring NCT02160782?

NCT02160782 is sponsored by Mirum Pharmaceuticals, Inc..

What drugs are being tested in NCT02160782?

Interventions in NCT02160782: LUM001 (Maralixibat), Placebo.

What condition does NCT02160782 study?

NCT02160782 studies Alagille Syndrome.

Is NCT02160782 still recruiting?

NCT02160782 is currently completed. Last updated 14 July 2021.

Are results published for NCT02160782?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2021-07-14 · How we verify

NCT02160782: ICONIC

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS)

Completed Phase 2 Results posted Last updated 14 July 2021

What this trial tests

Phase 2 trial testing LUM001 (Maralixibat) in Alagille Syndrome in 31 participants. Completed in 28 May 2020.

Timeline

28 October 2014

Primary endpoint
28 May 2020

28 May 2020

Quick facts

Lead sponsor	Mirum Pharmaceuticals, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	treatment
Enrollment	31
Start date	28 October 2014
Primary completion	28 May 2020
Estimated completion	28 May 2020
Sites	9 locations across France, Belgium, United Kingdom, Poland, Australia, Spain

Drugs / interventions tested

LUM001 (Maralixibat) — full drug profile →
Placebo

Conditions studied

Alagille Syndrome — all drugs for Alagille Syndrome →

Sponsor

Mirum Pharmaceuticals, Inc. — full company profile →

Who can join

Adults 12 Months to 18, any sex, with Alagille Syndrome. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

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 Primary · 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.

Group	Value	95% CI
Randomized Withdrawal Period: MRX	-21.73	± 43.125
Randomized Withdrawal Period: Placebo	95.55	± 30.488

Change From Baseline to Week 18 in Fasting sBA Levels Secondary · Baseline to Week 18

This secondary efficacy endpoint is the mean change from baseline to Week 18 in fasting sBA levels

Group	Value	95% CI
Open-label Period: MRX Baseline	283.43	± 210.569
Open-label Period: MRX Week 18	192.50	± 161.278

Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Obs) Secondary · Baseline to Week 18

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).

Group	Value	95% CI
Open-label Period: MRX Baseline	2.909	± 0.5480
Open-label Period: MRX Week 18	1.203	± 0.8446

Change From Baseline to Week 18 in Pruritus as Measured by ItchRO (Pt) Secondary · Baseline to Week 18

This secondary efficacy endpoint is the change from baseline to Week 18 in pruritus as measured by ItchRO(Pt) weekly average morning score

Group	Value	95% CI
Open-label Period: MRX Baseline	2.903	± 0.6616
Open-label Period: MRX Week 18	0.831	± 0.8122

Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Obs) Secondary · Week 18 to Week 22

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).

Group	Value	95% CI
Randomized Withdrawal Period: MRX	0.217	± 0.2345
Randomized Withdrawal Period: Placebo	1.700	± 0.2031

Change From Week 18 to Week 22 in Pruritus as Measured by ItchRO(Pt) Secondary · Week 18 to Week 22

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).

Group	Value	95% CI
Randomized Withdrawal Period: MRX	-0.149	± 0.3719
Randomized Withdrawal Period: Placebo	1.839	± 0.2771

Change From Baseline to Week 18 in Alkaline Phosphatase Secondary · Baseline to Week 18

This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALP

Group	Value	95% CI
Open-label Period: MRX Baseline	601.3	± 274.77
Open-label Period: MRX Week 18	580.8	± 215.50

Change From Week 18 to Week 22 in Alkaline Phosphatase Secondary · Week 18 to Week 22

This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in ALP

Group	Value	95% CI
Randomized Withdrawal Period: MRX	2.8	± 22.55
Randomized Withdrawal Period: Placebo	-7.2	± 20.31

Change From Baseline to Week 18 in Alanine Aminotransferase Secondary · Baseline to Week 18

This secondary efficacy endpoint is the mean change from baseline to Week 18 in ALT

Group	Value	95% CI
Open-label Period: MRX Baseline	181.0	± 108.56
Open-label Period: MRX Week 18	177.4	± 92.08

Change From Week 18 to Week 22 in Alanine Aminotransferase Secondary · Week 18 to Week 22

This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in alanine aminotransferase (ALT)

Group	Value	95% CI
Randomized Withdrawal Period: MRX	34.5	± 14.04
Randomized Withdrawal Period: Placebo	19.4	± 12.56

Change From Baseline to Week 18 in Total Bilirubin Secondary · Baseline to Week 18

This secondary efficacy endpoint is the mean change from baseline to Week 18 in total bilirubin

Group	Value	95% CI
Open-label Period: MRX Baseline	6.09	± 5.781
Open-label Period: MRX Week 18	5.12	± 5.337

Change From Week 18 to Week 22 in Total Bilirubin Secondary · Week 18 to Week 22

This secondary efficacy endpoint is the mean change from Week 18 to Week 22 in total bilirubin

Group	Value	95% CI
Randomized Withdrawal Period: MRX	0.32	± 0.265
Randomized Withdrawal Period: Placebo	0.46	± 0.238

Adverse events — posted to ClinicalTrials.gov

Time frame: Baseline to study completion (median of 2 years). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Open-label Period: Maralixibat (LUM001)

Serious: 4/31 (13%)

Deaths: 0/31

Randomized Withdrawal Period: MRX

Serious: 1/13 (8%)

Deaths: 0/13

Randomized Withdrawal Period: Placebo

Serious: 1/16 (6%)

Deaths: 0/16

After Randomized Withdrawal Period: Maralixibat

Serious: 5/29 (17%)

Deaths: 0/29

Long-term Extension Period: Maralixibat

Serious: 6/23 (26%)

Deaths: 0/23

Safety Population

Serious: 13/31 (42%)

Deaths: 0/31

Serious adverse events (27 terms)

Reaction	System	Open-label Period: Maralix…	Randomized Withdrawal Peri…	Randomized Withdrawal Peri…	After Randomized Withdrawa…	Long-term Extension Period…	Safety Population
Pyrexia	General disorders	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Shock haemorrhagic	Vascular disorders	—	—	—	—	—	—
Extradural haematoma	Injury, poisoning and procedural complications	—	—	—	—	—	—
Forearm fracture	Injury, poisoning and procedural complications	—	—	—	—	—	—
Multiple injuries	Injury, poisoning and procedural complications	—	—	—	—	—	—
Splenic rupture	Injury, poisoning and procedural complications	—	—	—	—	—	—
Subdural haemorrhage	Injury, poisoning and procedural complications	—	—	—	—	—	—
Toxicity to various agents	Injury, poisoning and procedural complications	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—
Marrow hyperplasia	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—
Cardiac dysfunction	Cardiac disorders	—	—	—	—	—	—
Aplasia pure red cell	Blood and lymphatic system disorders	—	—	—	—	—	—
Seizure	Nervous system disorders	—	—	—	—	—	—
Chest pain	General disorders	—	—	—	—	—	—
Influenza like illness	General disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Acute kidney injury	Renal and urinary disorders	—	—	—	—	—	—
Campylobacter gastroenteritis	Infections and infestations	—	—	—	—	—	—
Epstein-Barr virus infection	Infections and infestations	—	—	—	—	—	—
Gastroenteritis	Infections and infestations	—	—	—	—	—	—
Rotavirus infection	Infections and infestations	—	—	—	—	—	—
Tonsillitis	Infections and infestations	—	—	—	—	—	—

Other adverse events (144 terms — click to expand)

Reaction	System	Open-label Period: Maralix…	Randomized Withdrawal Peri…	Randomized Withdrawal Peri…	After Randomized Withdrawa…	Long-term Extension Period…	Safety Population
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Nasopharyngitis	Infections and infestations	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—	—	—
Ear infection	Infections and infestations	—	—	—	—	—	—
Viral infection	Infections and infestations	—	—	—	—	—	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Gastroenteritis	Infections and infestations	—	—	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—	—	—
Influenza	Infections and infestations	—	—	—	—	—	—
Contusion	Injury, poisoning and procedural complications	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Otitis media	Infections and infestations	—	—	—	—	—	—
Pharyngitis	Infections and infestations	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—
Influenza like illness	General disorders	—	—	—	—	—	—
Head injury	Injury, poisoning and procedural complications	—	—	—	—	—	—
Muscle strain	Injury, poisoning and procedural complications	—	—	—	—	—	—
Nasal injury	Injury, poisoning and procedural complications	—	—	—	—	—	—
Skin abrasion	Injury, poisoning and procedural complications	—	—	—	—	—	—
Skin laceration	Injury, poisoning and procedural complications	—	—	—	—	—	—
Ear pain	Ear and labyrinth disorders	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Faeces pale	Gastrointestinal disorders	—	—	—	—	—	—
Gastritis	Gastrointestinal disorders	—	—	—	—	—	—
Bronchitis	Infections and infestations	—	—	—	—	—	—
Rhinitis	Infections and infestations	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—	—	—
Lip injury	Injury, poisoning and procedural complications	—	—	—	—	—	—
Procedural pain	Injury, poisoning and procedural complications	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—
Phimosis	Congenital, familial and genetic disorders	—	—	—	—	—	—

Most-reported serious reactions: Pyrexia, Hypertension, Shock haemorrhagic, Extradural haematoma, Forearm fracture, Multiple injuries, Splenic rupture, Subdural haemorrhage.

Data from ClinicalTrials.gov NCT02160782 adverse events section.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Bile acid metabolism and signaling in health and disease: molecular mechanisms and therapeutic targets.
Fleishman JS, Kumar S. · · 2024 · cited 267× · PMID 38664391 · DOI 10.1038/s41392-024-01811-6
Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study.
Gonzales E, Hardikar W, Stormon M, Baker A, et al · · 2021 · cited 114× · PMID 34755627 · DOI 10.1016/s0140-6736(21)01256-3
Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome.
Shneider BL, Spino C, Kamath BM, Magee JC, et al · · 2018 · cited 70× · PMID 30288474 · DOI 10.1002/hep4.1244
The molecular mechanisms of cardiac development and related diseases.
Li Y, Du J, Deng S, Liu B, et al · · 2024 · cited 53× · PMID 39715759 · DOI 10.1038/s41392-024-02069-8
Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis.
Kamath BM, Stein P, Houwen RHJ, Verkade HJ. · · 2020 · cited 53× · PMID 32492754 · DOI 10.1111/liv.14553
Pediatric Cholestatic Liver Disease: Review of Bile Acid Metabolism and Discussion of Current and Emerging Therapies.
Kriegermeier A, Green R. · · 2020 · cited 40× · PMID 32432119 · DOI 10.3389/fmed.2020.00149
Bile acid-mediated signaling in cholestatic liver diseases.
Zeng J, Fan J, Zhou H. · · 2023 · cited 39× · PMID 37120573 · DOI 10.1186/s13578-023-01035-1
Maralixibat: First Approval.
Shirley M. · · 2022 · cited 38× · PMID 34813049 · DOI 10.1007/s40265-021-01649-0

Verify or expand the search:

Other trials of LUM001 (Maralixibat)

Trials testing the same drug.

NCT02117713 — An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic L · Phase 2 · completed
NCT02057718 — Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver · Phase 2 · completed
NCT02047318 — An Extension Study to Evaluate the Long-Term Safety and Durability of Effect of LUM001 in the Treatment of Cholestatic L · Phase 2 · completed

Other recruiting trials for Alagille Syndrome

Currently open trials in the same condition.

NCT07290257 — Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome i · Phase 4 · recruiting
NCT06193928 — Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) · recruiting
NCT05035030 — Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome · Phase 3 · recruiting

Other Mirum Pharmaceuticals, Inc. trials

Trials by the same sponsor.

NCT07454837 — Phase 2b/3 Study to Evaluate Switching to Brelovitug for the Treatment of CHD in Participants Receiving Bulevirtide · Phase 2, PHASE3 · recruiting
NCT07290257 — Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome i · Phase 4 · recruiting
NCT07200908 — A Trial Evaluating Brelovitug (BJT-778) vs Bulevirtide for the Treatment of Chronic Hepatitis Delta Infection (AZURE-2) · Phase 3 · recruiting
NCT06193928 — Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US) · recruiting
NCT04729751 — A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases In · Phase 2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02160782 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Mirum Pharmaceuticals, Inc.
Last refreshed: 14 July 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02160782.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing