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).
Public ClinicalTrials.gov record NCT04729751. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Open-Label, Phase 2 Study to Evaluate the Safety and Tolerability of Maralixibat in the Treatment of Infants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis and Alagille Syndrome
Study identification
- NCT ID
- NCT04729751
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Enrollment
- 27 participants
Conditions and interventions
Conditions
Interventions
- Maralixibat Drug
Drug
Eligibility (public fields only)
- Age range
- 0 Days to 364 Days
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 8, 2021
- Primary completion
- Dec 16, 2024
- Completion
- Dec 16, 2024
- Last update posted
- Feb 5, 2025
2021 – 2024
United States locations
- U.S. sites
- 7
- U.S. states
- 6
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children Hospital LA | Los Angeles | California | 90027 | — |
| University of California - San Francisco | San Francisco | California | 94158 | — |
| Medstar Georgetown University Hospital | Washington D.C. | District of Columbia | 20007 | — |
| Ochsner Hospital for Children | New Orleans | Louisiana | 70121 | — |
| Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 7 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04729751, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 5, 2025 · Synced May 19, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04729751 live on ClinicalTrials.gov.