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NCT02057718 · ClinicalTrials.gov registry record · Phase 2
Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis
A Phase 2 study of Progressive Familial Intrahepatic Cholestasis (PFIC), sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 2
- Development phase
- 33
- Enrollment target
- 10
- Study locations
NCT02057718: Completed Phase 2 study of Progressive Familial Intrahepatic Cholestasis (PFIC), sponsored by Mirum Pharmaceuticals.
NCT02057718 is a Phase 2 study of Progressive Familial Intrahepatic Cholestasis (PFIC) that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 33 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% lower). The trial reports 10 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02057718, a Phase 2 study of Progressive Familial Intrahepatic Cholestasis (PFIC), has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 33 participants
- Enrollment target
- 10
- Study locations
Study Summary
This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus associated with PFIC.
Conditions Studied
Interventions
- DRUG LUM001 (Maralixibat)
Study Locations (10)
Other
- Hopital Femme Mere Enfant De Lyon - Bron
- The Children's Memorial Health Institute - Warsaw
- Leeds Teaching Hospital NHS Trust - Leeds
- Kings College Hospital - London
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
California
- Children's Hospital Los Angeles - Los Angeles
Colorado
- Children's Hospital Colorado - Aurora
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
West Midlands
- Birmingham Children's Hospital - Birmingham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 33 participants |
| Start Date | 2014-03-01 |
| Est. Completion | 2020-05-08 |
| Phase | Phase 2 |
What the finished NCT02057718 record still lists
NCT02057718 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 33 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (75% 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 LUM001 (Maralixibat) is the first listed.
NCT02057718 lists 10 locations in 6 states (Other, Pennsylvania, California).
Frequently Asked Questions
What is clinical trial NCT02057718 about?
NCT02057718 is a clinical study titled "Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis". This is an open label study in children with Progressive Familial Intrahepatic Cholestasis (PFIC) designed to evaluate the safety and efficacy of LUM001, also known as Maralixibat (MRX). Efficacy will be assessed by evaluating the effect of LUM001 on pruritus and the biochemical markers of pruritus ...
What is the current status of trial NCT02057718?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 33 participants. The study started on 2014-03-01. Estimated completion is 2020-05-08.
What conditions does trial NCT02057718 study?
This clinical trial studies the following conditions: Progressive Familial Intrahepatic Cholestasis (PFIC).
What interventions are being tested in trial NCT02057718?
The interventions under investigation include: LUM001 (Maralixibat) (DRUG).
Who is sponsoring clinical trial NCT02057718?
This trial is sponsored by Mirum Pharmaceuticals, which has 28 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02057718 being conducted?
This trial has 10 study locations across California, Colorado, Ohio, Pennsylvania, West Midlands. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02057718's enrollment target sits among peer trials
33 1553rd of 2000 higher than 438 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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