Trial · NCT04729751
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
NCT04729751, Phase 2, Completed, Mirum Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS). Trials, record NCT04729751. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04729751
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
- NCT number
- NCT04729751
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 27 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Cholestatic Liver Disease
- Interventions
- Maralixibat
- First posted
- 2021-01-28
- Last updated
- 2026-06-02
- Other registries' IDs
- eudract:2020-004628-40
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Frequency of Treatment-emergent Adverse Events [TEAEs] | From Baseline through to Week 13 | TEAEs = Treatment-emergent Adverse Events |
| Secondary | Change in Fasting Serum Bile Acid (sBA) Levels | From Baseline through to Week 13 | sBA = serum bile acid |
| Secondary | To Evaluate the Effect on Liver Enzymes (ALT) | From Baseline through to Week 13 | ALT= alanine aminotransferase. |
| Secondary | Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin E | From Baseline through to Week 13 | Change from baseline to Week 13 in vitamin E. |
| Secondary | Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for ALGS | At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit | BID=twice daily; QD=once daily. Systemic concentrations of maralixibat in plasma were determined at the following visits: * Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing * Before dosing and \~2.5 hours after the morning dose at Week 6, Week 10, and Week 13 Stable dosing occurred at 400 μg/kg QD for ALGS or at the highest tolerated dose. Note: The values added in section Measured Values are for maralixibat dose of 400 µg/kg QD. |
| Secondary | To Evaluate the Effect on Liver Enzymes (AST) | From Baseline through to Week 13. | AST= aspartate aminotransferase |
| Secondary | To Evaluate the Effect on Bilirubin | From Baseline through to Week 13 | Bilirubin |
| Secondary | Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamin A | From Baseline through to Week 13 | Change from baseline to Week 13 in vitamin A |
| Secondary | Change From Baseline to Week 13 in Lipid-Soluble Vitamins (LSVs) - Vitamins D and K | From Baseline through to Week 13 | Change from baseline to Week 13 in vitamins D and K. |
| Secondary | Maximum Level of Maralixibat Concentration in Plasma From Baseline to Week 13 for PFIC | At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit | BID=twice daily; QD=once daily. Systemic concentrations of maralixibat in plasma were determined at the following visits: * Approximately 2.5 hours after the morning dose at Week 0/Day 1 of dosing * Before dosing and \~2.5 hours after the morning dose at Week 6, Week 10, and Week 13 Stable dosing occurred at 300 μg/kg QD, 300 μg/kg BID and 600 μg/kg BID for PFIC or at the highest tolerated dose. Note: The values added in section Measured Values are for maralixibat dose of 600 µg/kg BID. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT04729751
GET https://conductscience.org/api/trials/v1/NCT04729751/registered