Trial · NCT03353454
A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT03353454, Phase 3, Withdrawn, Mirum Pharmaceuticals, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC). Trials, record NCT03353454. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03353454
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
- NCT number
- NCT03353454
- Status
- Withdrawn
- Phases
- Phase 3
- Enrollment
- 0 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- Interventions
- Maralixibat, Placebo
- First posted
- 2017-11-27
- Last updated
- 2019-03-18
- Other registries' IDs
- eudract:2017-003138-99
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment Response as Measured by the Observer Itch Reported Outcome (ItchRO[Obs]) | Baseline up to Week 26 | Compare the percentage of participants on active treatment versus (vs.) placebo who meet response criteria which is defined as improvement in before midday (AM) Observer Itch Reported Outcome (ItchRO\[Obs\]) severity decrease from baseline demonstrated on at least 2 of the last 3 study visits. |
| Secondary | Treatment Response as Measured by the Observer Itch Reported Outcome (ItchRO[Obs]) and Serum Bile Acids (sBA) | Baseline up to Week 26 | Compare the percentage of participants on active treatment versus (vs.) placebo who meet response criteria which is defined as improvement in average before midday (AM) Observer Itch Reported Outcome (ItchRO\[Obs\]) severity decrease from baseline and normalization or reduction from baseline sBA demonstrated on at least 2 of the last 3 study visits. |
| Secondary | Normalization or Reduction From Baseline in Serum Bile Acids (sBA) | Baseline up to Week 26 | Compare the percentage of participants on active treatment vs. placebo with normalization or significant reduction from baseline in sBA. |
| Secondary | Change Over Time in Daily Average Itch Reported Outcome (ItchRO[Obs]) Score | Baseline up to Week 26 | Change over time in daily average ItchRO scores will be reported. |
| Secondary | Change Over Time in Before Midday (AM) Itch Reported Outcome (ItchRO[Obs]) Score | Baseline up to Week 26 | Change over time in AM ItchRO scores will be reported. |
| Secondary | Change Over Time in After Midday (PM) Itch Reported Outcome (ItchRO[Obs]) Score | Baseline up to Week 26 | Change over time in PM ItchRO scores will be reported. |
| Secondary | Disappearance of Pruritus as Measured by Observer Itch Reported Outcome (ItchRO[Obs]) | Baseline up to Week 26 | Compare the percentage of participants on active treatment vs. placebo of participants who experience disappearance of pruritus as measured by ItchRO(Obs). |
| Secondary | Improvement in Height | Baseline up to Week 26 | Number of participants on active treatment vs. placebo with a height z-score change from baseline \>0. |
| Secondary | Improvement in Weight | Baseline up to Week 26 | Number of participants on active treatment vs. placebo with a weight z-score change from baseline \>0. |
| Secondary | Change From Baseline in Nutritional Status as Measured by Mid-arm Circumference | Baseline, Week 26 | Compare the change in nutritional status as measured by mid-arm circumference in participants on active treatment vs. placebo. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes0
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/NCT03353454
GET https://conductscience.org/api/trials/v1/NCT03353454/registered