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NCT04729751 · ClinicalTrials.gov registry record · Phase 2
A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
A Phase 2 study, sponsored by Mirum Pharmaceuticals.
- Completed
- Registry status
- Phase 2
- Development phase
- 27
- Enrollment target
NCT04729751: Completed Phase 2 study, sponsored by Mirum Pharmaceuticals.
NCT04729751 is a Phase 2 study that has completed, run by Mirum Pharmaceuticals. The registered enrollment target is 27 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (80% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04729751, a Phase 2 study, has completed, sponsored by Mirum Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 27 participants
- Enrollment target
Study Summary
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children \<12 months of age with Alagille Syndrome \[ALGS\] or Progressive Familial Intrahepatic Cholestasis \[PFIC\].
Primary Outcome
TEAEs = Treatment-emergent Adverse Events
Interventions
- DRUG Maralixibat
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 27 participants |
| Start Date | 2021-09-09 |
| Est. Completion | 2024-12-17 |
| Phase | Phase 2 |
What the finished NCT04729751 record still lists
NCT04729751 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 27 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (80% lower).
The record links to 0 conditions, and to 1 intervention - of which Maralixibat is the first listed.
NCT04729751 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04729751 about?
NCT04729751 is a clinical study titled "A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).". This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children \<12 months of age with Alagille Syndrome \[ALGS\] or Progressive Familial Intrahepatic Cholestasis \[PFIC\].
What is the current status of trial NCT04729751?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 27 participants. The study started on 2021-09-09. Estimated completion is 2024-12-17.
What interventions are being tested in trial NCT04729751?
The interventions under investigation include: Maralixibat (DRUG).
Who is sponsoring clinical trial NCT04729751?
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 NCT04729751's enrollment target sits among peer trials
27 1697th of 2000 higher than 293 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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