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NCT03905330 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
A Phase 3 study, sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 93
- Enrollment target
NCT03905330: Completed Phase 3 study, sponsored by Mirum Pharmaceuticals.
NCT03905330 is a Phase 3 study that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 93 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03905330, a Phase 3 study, has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 93 participants
- Enrollment target
Study Summary
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
Primary Outcome
The primary efficacy endpoint is the mean change in the average morning ItchRO(Obs) severity score between baseline and Weeks 15-26, using 4-week average morning ItchRO(Obs) severity scores (Mixed Model Repeated Measures). The baseline average morning ItchRO(Obs) severity score is defined as the 4-week average morning ItchRO(Obs) severity score prior to the first dose of the study medication. ItchRo(Obs) Severity Score = Itch Reported Outcome Observer assessment severity score; scale between 0
Interventions
- OTHER Placebo
- DRUG Maralixibat
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 93 participants |
| Start Date | 2019-07-09 |
| Est. Completion | 2022-09-01 |
| Phase | Phase 3 |
What the finished NCT03905330 record still lists
NCT03905330 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 93 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT03905330 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03905330 about?
NCT03905330 is a clinical study titled "A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)". The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
What is the current status of trial NCT03905330?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 93 participants. The study started on 2019-07-09. Estimated completion is 2022-09-01.
What interventions are being tested in trial NCT03905330?
The interventions under investigation include: Placebo (OTHER), Maralixibat (DRUG).
Who is sponsoring clinical trial NCT03905330?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03905330's enrollment target sits among peer trials
93 1722nd of 2000 higher than 278 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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