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NCT04185363 · ClinicalTrials.gov registry record · Phase 3
An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
A Phase 3 study of Progressive Familial Intrahepatic Cholestasis (PFIC), sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 90
- Enrollment target
- 20
- Study locations
NCT04185363: Completed Phase 3 study of Progressive Familial Intrahepatic Cholestasis (PFIC), sponsored by Mirum Pharmaceuticals.
NCT04185363 is a Phase 3 study of Progressive Familial Intrahepatic Cholestasis (PFIC) that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 90 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (88% lower). The trial reports 20 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04185363, a Phase 3 study of Progressive Familial Intrahepatic Cholestasis (PFIC), has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 90 participants
- Enrollment target
- 20
- Study locations
Study Summary
The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
Primary Outcome
The Score on the ItchRo scale is a score on a 5-point scale from 0 (no itch) to 4 (very severe itch)
Conditions Studied
Interventions
- DRUG Maralixibat
Study Locations (20)
Other
- Hospital Italiano de Buenos Aires - Buenos Aires
- Medizinische Universität Wien, Universitätsklinik für Kinder- und Jugendheilkunde - Vienna
- Cliniques Universitaires Saint-Luc - Brussels
- Sociedade Beneficente de Senhoras Hospital Sírio-Libanês - São Paulo
- Fundacion Cardioinfantil - Bogotá
- Groupement Hospitalier Est Hopital, Femme Mère Enfant de Lyon - Lyon
- CHU de Toulouse - Hôpital des Enfants - Toulouse
- Medizinische Hochschule - Hanover
- Azienda Ospedaliera Papa Giovanni XXIII - Unita di Pediatria - Bergamo
Texas
- University of Texas Southwestern Medical Center - Dallas
- University of Texas, Health Science Center San Antonio - San Antonio
California
- Children's Hospital Los Angeles - Los Angeles
District of Columbia
- Medstar Georgetown University Hospital - Washington D.C.
Florida
- Advent Health - Orlando
New York
- Children's Hospital at Montefiore - The Bronx
Ohio
- Cincinnati Children's Hospital - Cincinnati
Pennsylvania
- Children Hospital of Pittsburgh - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 90 participants |
| Start Date | 2020-01-08 |
| Est. Completion | 2025-04-23 |
| Phase | Phase 3 |
What the finished NCT04185363 record still lists
NCT04185363 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 90 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 1 condition, with Progressive Familial Intrahepatic Cholestasis (PFIC) appearing as the primary indexed condition, and to 1 intervention - of which Maralixibat is the first listed.
NCT04185363 names 20 study sites across 11 states, led by Other, Texas, California.
Frequently Asked Questions
What is clinical trial NCT04185363 about?
NCT04185363 is a clinical study titled "An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)". The primary objective of this open label extension study is to evaluate the long-term safety and tolerability of maralixibat.
What is the current status of trial NCT04185363?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 90 participants. The study started on 2020-01-08. Estimated completion is 2025-04-23.
What conditions does trial NCT04185363 study?
This clinical trial studies the following conditions: Progressive Familial Intrahepatic Cholestasis (PFIC).
What interventions are being tested in trial NCT04185363?
The interventions under investigation include: Maralixibat (DRUG).
Who is sponsoring clinical trial NCT04185363?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04185363 being conducted?
This trial has 20 study locations across California, District of Columbia, Florida, New York, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT04185363's enrollment target sits among peer trials
90 1724th of 2000 higher than 263 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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