Trial · NCT04185363
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT04185363, Phase 3, Completed, Mirum Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC). Trials, record NCT04185363. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04185363
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
- NCT number
- NCT04185363
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 84 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- Interventions
- Maralixibat
- First posted
- 2019-12-04
- Last updated
- 2026-06-23
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 From Baseline Over Time in the Average Morning ItchRO(Obs) Severity Score | From Baseline to Weeks 75-78 | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) |
| Secondary | Maintenance of ItchRO(Obs) Response (Weeks 15 - 26) | Week 15 - 26 | Maintenance of treatment effect is defined as the proportion of participants in the MRX-MRX treatment group (MRX-502 and MRX-503 data for subjects on MRX in the MRX-502 study) who obtain an ItchRO (Obs) response from Week 15 to Week 26 in Study MRX-503 in the primary cohort and PFIC cohort. |
| Secondary | Proportion of ItchRO(Obs) Responders Over Time | Baseline to EOT | Proportion of ItchRO responders over time at each study visit using the 4-week study period prior to the visit as per ItchRO(Obs) responder definition. |
| Secondary | Mean Change From Baseline Over Time in the Average Morning ItchRO(Obs) Frequency Score | From Baseline to Weeks 75-78 | The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch) |
| Secondary | Mean Change From Baseline Over Time in Total Serum Bile Acid (sBA) Levels | Baseline to week 70 | Mean change from baseline over time in total serum bile acid (sBA) levels |
| Secondary | Proportion of Subjects Who Experience an sBA Control Over Time From Week 18 to Week 26 | From Week 18 to Week 26 | Proportion of subjects who experience an sBA control over time from Week 18 to Week 26 |
| Secondary | Change From Baseline in Height Z-score | From baseline to Week 70 | Height-for-age Z-score measures a participant's height relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate height above the reference population mean, and negative Z-scores indicate height below the reference population mean. Higher Z-scores generally indicate greater linear growth relative to the reference population. Change from baseline was calculated as post-baseline height-for-age Z-score minus baseline height-for-age Z-score. |
| Secondary | Change From Baseline in Weight Z-score | From Baseline to Week 70 | Weight-for-age Z-score measures a participant's weight relative to a reference population of children of the same age and sex. Z-scores were derived using World Health Organization growth charts for participants less than 24 months of age and Centers for Disease Control and Prevention growth charts for participants 24 months of age or older. A Z-score of 0 represents the population mean for age and sex. Positive Z-scores indicate weight above the reference population mean, and negative Z-scores indicate weight below the reference population mean. Higher Z-scores generally indicate greater body weight relative to the reference population. Change from baseline was calculated as post-baseline weight-for-age Z-score minus baseline weight-for-age Z-score. |
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/NCT04185363
GET https://conductscience.org/api/trials/v1/NCT04185363/registered