Mean Change in Total Serum Bilirubin Levels
Primary
· From baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | -3.5 | ± 0.853 |
| Double Blind - Placebo | -3.11 | ± 0.947 |
Last reviewed · How we verify
NCT04524390: EMBARK
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
Completed
Phase 2
Results posted
Last updated 19 March 2025
What this trial tests
Phase 2 trial testing Maralixibat in Biliary Atresia in 75 participants. Completed in 7 February 2024.
Timeline
8 July 2021
Primary endpoint
7 November 2023
7 November 2023
7 February 2024
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 | 75 |
| Start date | 8 July 2021 |
| Primary completion | 7 November 2023 |
| Estimated completion | 7 February 2024 |
| Sites | 22 locations across Taiwan, United Kingdom, Germany, Poland, Vietnam, China, Singapore, United States |
Drugs / interventions tested
- Maralixibat — full drug profile →
- Placebo
Conditions studied
- Biliary Atresia — all drugs for Biliary Atresia →
Sponsor
Mirum Pharmaceuticals, Inc. — full company profile →
Who can join
Adults 21 Days to 111 Days, any sex, with Biliary Atresia. 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.
Mean Change in Total Serum Bile Acids
Secondary
· From baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | -51.19 | ± 24.436 |
| Double Blind - Placebo | -5.29 | ± 28.440 |
Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26
Secondary
· From baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 24 | |
| Double Blind - Placebo | 20 |
Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death.
Secondary
· From Baseline to Week 26
Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites)
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 8 | |
| Double Blind - Placebo | 7 |
Proportion of Participants Undergoing Liver Transplantation or Death
Secondary
· From Baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 5 | |
| Double Blind - Placebo | 3 |
Proportion of Participants Observed to Develop Clinically Evident Portal Hypertension Defined as Splenomegaly and Thrombocytopenia (Platelet Count <150 x 109/L) or Clinically Evident Ascites or Endoscopic Evidence of Esophageal or Gastric Varices.
Secondary
· From Baseline to Week 26
Splenomegaly =\> (spleen size \>2 cm below the costal margin palpated on physical examination)
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 3 | |
| Double Blind - Placebo | 4 |
Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL
Secondary
· From Baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 23 | |
| Double Blind - Placebo | 18 |
Proportion of Participants With Mean sBA Levels ≤40 mmol/L
Secondary
· From Baseline to Week 26
| Group | Value | 95% CI |
|---|---|---|
| Double Blind - Maralixibat | 10 | |
| Double Blind - Placebo | 7 |
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline to EOT. In this study the longest time frame was 102.4 weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Double Blind - Maralixibat
Serious: 26/40 (65%)
Deaths: 0/40
Double Blind - Placebo
Serious: 25/35 (71%)
Deaths: 0/35
Open Label - Maralixibat
Serious: 21/52 (40%)
Deaths: 0/52
Serious adverse events (42 terms)
| Reaction | System | Double Blind - Maralixibat | Double Blind - Placebo | Open Label - Maralixibat |
|---|---|---|---|---|
| Cholangitis | Hepatobiliary disorders | — | — | — |
| Pneumonia | Infections and infestations | — | — | — |
| COVID-19 | Infections and infestations | — | — | — |
| Hepatic enzyme increased | Investigations | — | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — | — |
| Adenovirus infection | Infections and infestations | — | — | — |
| Bronchiolitis | Infections and infestations | — | — | — |
| Gastroenteritis | Infections and infestations | — | — | — |
| Parainfluenzae virus infection | Infections and infestations | — | — | — |
| Sepsis | Infections and infestations | — | — | — |
| Alanine aminotransferase increased | Investigations | — | — | — |
| Accidental exposure to product by child | Injury, poisoning and procedural complications | — | — | — |
| Procedural complication | Injury, poisoning and procedural complications | — | — | — |
| Coagulopathy | Blood and lymphatic system disorders | — | — | — |
| Pyrexia | General disorders | — | — | — |
| Systemic inflammatory response syndrome | General disorders | — | — | — |
| Drug hypersensitivity | Immune system disorders | — | — | — |
| Faeces discoloured | Gastrointestinal disorders | — | — | — |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | — | — | — |
| Ileus | Gastrointestinal disorders | — | — | — |
| Mouth ulceration | Gastrointestinal disorders | — | — | — |
| Stomatitis | Gastrointestinal disorders | — | — | — |
| Cholangitis acute | Hepatobiliary disorders | — | — | — |
| Drug-induced liver injury | Hepatobiliary disorders | — | — | — |
| Hyperbilirubinaemia | Hepatobiliary disorders | — | — | — |
Other adverse events (39 terms — click to expand)
| Reaction | System | Double Blind - Maralixibat | Double Blind - Placebo | Open Label - Maralixibat |
|---|---|---|---|---|
| Pyrexia | General disorders | — | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — | — |
| Vitamin D deficiency | Metabolism and nutrition disorders | — | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Vitamin A deficiency | Metabolism and nutrition disorders | — | — | — |
| Vomiting | Gastrointestinal disorders | — | — | — |
| Cholangitis | Hepatobiliary disorders | — | — | — |
| Eczema | Skin and subcutaneous tissue disorders | — | — | — |
| Pneumonia | Infections and infestations | — | — | — |
| Alanine aminotransferase increased | Investigations | — | — | — |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | — | — | — |
| COVID-19 | Infections and infestations | — | — | — |
| Zinc deficiency | Metabolism and nutrition disorders | — | — | — |
| Bronchitis | Infections and infestations | — | — | — |
| Viral upper respiratory tract infection | Infections and infestations | — | — | — |
| Vitamin E deficiency | Metabolism and nutrition disorders | — | — | — |
| Hepatic enzyme increased | Investigations | — | — | — |
| Anaemia | Blood and lymphatic system disorders | — | — | — |
| Coagulopathy | Blood and lymphatic system disorders | — | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — | — |
| Urticaria | Skin and subcutaneous tissue disorders | — | — | — |
| Nasopharyngitis | Infections and infestations | — | — | — |
| Otitis media | Infections and infestations | — | — | — |
| Gastroenteritis | Infections and infestations | — | — | — |
| Influenza | Infections and infestations | — | — | — |
| Blood bilirubin increased | Investigations | — | — | — |
| Spleen palpable | Investigations | — | — | — |
| Vitamin A decreased | Investigations | — | — | — |
| Scratch | Injury, poisoning and procedural complications | — | — | — |
| Splenomegaly | Blood and lymphatic system disorders | — | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — | — |
| Haematochezia | Gastrointestinal disorders | — | — | — |
| Hepatomegaly | Hepatobiliary disorders | — | — | — |
| Pruritus | Skin and subcutaneous tissue disorders | — | — | — |
| Pharyngitis | Infections and infestations | — | — | — |
| Respiratory tract infection | Infections and infestations | — | — | — |
| Cytomegalovirus infection | Infections and infestations | — | — | — |
| Parainfluenzae virus infection | Infections and infestations | — | — | — |
Most-reported serious reactions: Cholangitis, Pneumonia, COVID-19, Hepatic enzyme increased, Diarrhoea, Adenovirus infection, Bronchiolitis, Gastroenteritis.
Data from ClinicalTrials.gov NCT04524390 adverse events section.
Sponsor's own description
A study to evaluate the efficacy and safety of maralixibat in infants with Biliary Atresia (BA) after Hepatoportoenterostomy (HPE, also known as the Kasai procedure).
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Membrane transporters in drug development and as determinants of precision medicine.
Galetin A, Brouwer KLR, Tweedie D, Yoshida K, et al · · 2024 · cited 169× · PMID 38267543 · DOI 10.1038/s41573-023-00877-1 -
Biliary atresia.
Tam PKH, Wells RG, Tang CSM, Lui VCH, et al · · 2024 · cited 90× · PMID 38992031 · DOI 10.1038/s41572-024-00533-x -
Biliary Atresia - emerging diagnostic and therapy opportunities.
Lendahl U, Lui VCH, Chung PHY, Tam PKH. · · 2021 · cited 83× · PMID 34781099 · DOI 10.1016/j.ebiom.2021.103689 -
Maralixibat: First Approval.
Shirley M. · · 2022 · cited 38× · PMID 34813049 · DOI 10.1007/s40265-021-01649-0 -
Serum bile acids as a prognostic biomarker in biliary atresia following Kasai portoenterostomy.
Harpavat S, Hawthorne K, Setchell KDR, Rivas MN, et al · · 2023 · cited 29× · PMID 36131538 · DOI 10.1002/hep.32800 -
Current and emerging adjuvant therapies in biliary atresia.
Fligor SC, Hirsch TI, Tsikis ST, Adeola A, et al · · 2022 · cited 11× · PMID 36313875 · DOI 10.3389/fped.2022.1007813 -
Recent advances in the management of pediatric cholestatic liver diseases.
Mysore KR, Cheng K, Suri LA, Fawaz R, et al · · 2025 · cited 5× · PMID 39840645 · DOI 10.1002/jpn3.12462 -
Biliary fibrosis is an important but neglected pathological feature in hepatobiliary disorders: from molecular mechanisms to clinical implications.
Zhao J, Yue P, Mi N, Li M, et al · · 2024 · cited 5× · PMID 39135601 · DOI 10.1515/mr-2024-0029
Verify or expand the search:
- PubMed search for NCT04524390
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
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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 NCT04524390 (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: 19 March 2025
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/NCT04524390.
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