Trial · NCT04524390
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
NCT04524390, Phase 2, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2025-02-17
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai. Trials, record NCT04524390. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04524390
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
- NCT number
- NCT04524390
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 75 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Biliary Atresia
- Interventions
- Maralixibat, Placebo
- First posted
- 2020-08-24
- Last updated
- 2025-03-19
- Results first posted
- 2025-02-17
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Change in Total Serum Bilirubin Levels | From baseline to Week 26 | |
| Secondary | Mean Change in Total Serum Bile Acids | From baseline to Week 26 | |
| Secondary | Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | From baseline to Week 26 | |
| Secondary | Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | From Baseline to Week 26 | Liver decompensation (hepatic encephalopathy, variceal bleeding, new persistent ascites) |
| Secondary | Proportion of Participants Undergoing Liver Transplantation or Death | From Baseline to Week 26 | |
| Secondary | 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. | From Baseline to Week 26 | Splenomegaly =\> (spleen size \>2 cm below the costal margin palpated on physical examination) |
| Secondary | Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL | From Baseline to Week 26 | |
| Secondary | Proportion of Participants With Mean sBA Levels ≤40 mmol/L | From Baseline to Week 26 |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Change in Total Serum Bilirubin Levels | From baseline to Week 26 | mg/dl | LEAST_SQUARES_MEAN | |
| SECONDARY | Mean Change in Total Serum Bile Acids | From baseline to Week 26 | umol/L | LEAST_SQUARES_MEAN | |
| SECONDARY | Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | From baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | From Baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Proportion of Participants Undergoing Liver Transplantation or Death | From Baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | 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. | From Baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL | From Baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Proportion of Participants With Mean sBA Levels ≤40 mmol/L | From Baseline to Week 26 | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group16
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Change in Total Serum Bilirubin Levels | Double Blind - Maralixibat | -3.5 | Standard Error 0.853 | mg/dl | |
| Mean Change in Total Serum Bilirubin Levels | Double Blind - Placebo | -3.11 | Standard Error 0.947 | mg/dl | |
| Mean Change in Total Serum Bile Acids | Double Blind - Maralixibat | -51.19 | Standard Error 24.436 | umol/L | |
| Mean Change in Total Serum Bile Acids | Double Blind - Placebo | -5.29 | Standard Error 28.440 | umol/L | |
| Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | Double Blind - Maralixibat | 24 | Participants | ||
| Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | Double Blind - Placebo | 20 | Participants | ||
| Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | Double Blind - Maralixibat | 8 | Participants | ||
| Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | Double Blind - Placebo | 7 | Participants | ||
| Proportion of Participants Undergoing Liver Transplantation or Death | Double Blind - Maralixibat | 5 | Participants | ||
| Proportion of Participants Undergoing Liver Transplantation or Death | Double Blind - Placebo | 3 | Participants |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double Blind | STARTED | Double Blind - Maralixibat | 40 | |
| Double Blind | STARTED | Double Blind - Placebo | 35 | |
| Double Blind | STARTED | Open Label - Maralixibat | 0 | |
| Double Blind | COMPLETED | Double Blind - Maralixibat | 28 | |
| Double Blind | COMPLETED | Double Blind - Placebo | 24 | |
| Double Blind | COMPLETED | Open Label - Maralixibat | 0 | |
| Double Blind | NOT COMPLETED | Double Blind - Maralixibat | 12 | |
| Double Blind | NOT COMPLETED | Double Blind - Placebo | 11 | |
| Double Blind | NOT COMPLETED | Open Label - Maralixibat | 0 | |
| Open-Label | STARTED | Double Blind - Maralixibat | 0 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events243
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Alanine aminotransferase increased | Investigations | Double Blind - Maralixibat | 5 | 40 | 5 |
| other | Alanine aminotransferase increased | Investigations | Double Blind - Placebo | 5 | 35 | 5 |
| other | Alanine aminotransferase increased | Investigations | Open Label - Maralixibat | 4 | 52 | 6 |
| other | Blood bilirubin increased | Investigations | Double Blind - Maralixibat | 1 | 40 | 3 |
| other | Blood bilirubin increased | Investigations | Double Blind - Placebo | 2 | 35 | 2 |
| other | Blood bilirubin increased | Investigations | Open Label - Maralixibat | 1 | 52 | 1 |
| other | Hepatic enzyme increased | Investigations | Double Blind - Maralixibat | 3 | 40 | 3 |
| other | Hepatic enzyme increased | Investigations | Double Blind - Placebo | 0 | 35 | 0 |
| other | Hepatic enzyme increased | Investigations | Open Label - Maralixibat | 1 | 52 | 1 |
| other | Spleen palpable | Investigations | Double Blind - Maralixibat | 0 | 40 | 0 |
1 to 10 of 243Source ClinicalTrials.gov
See all 243 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Double Blind - Maralixibat | 0 | 40 |
| Total, all-cause mortality | Double Blind - Placebo | 0 | 35 |
| Total, all-cause mortality | Open Label - Maralixibat | 0 | 52 |
| Total, other adverse events | Double Blind - Maralixibat | 38 | 40 |
| Total, other adverse events | Double Blind - Placebo | 33 | 35 |
| Total, other adverse events | Open Label - Maralixibat | 47 | 52 |
| Total, serious adverse events | Double Blind - Maralixibat | 26 | 40 |
| Total, serious adverse events | Double Blind - Placebo | 25 | 35 |
| Total, serious adverse events | Open Label - Maralixibat | 21 | 52 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons6
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Double Blind - Maralixibat vs Double Blind - Placebo: 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. | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean TSB Levels <2 mg/dL Through Week 26 | Trial comparisons |
Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants Observed to Have a Liver-related Clinical Event Transplantation, Liver Decompensation, Discontinuations Due to Liver Related Events, or Death. | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean TSB Levels ≤1.2 mg/dL | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants With Mean sBA Levels ≤40 mmol/L | Trial comparisons |
| Double Blind - Maralixibat vs Double Blind - Placebo: Proportion of Participants Undergoing Liver Transplantation or Death | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04524390
GET https://conductscience.org/api/trials/v1/NCT04524390/events?page=2