Trials / Completed
CompletedNCT04729751
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).
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
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 27 (actual)
- Sponsor
- Mirum Pharmaceuticals, Inc. · Industry
- Sex
- All
- Age
- 0 Days – 364 Days
- Healthy volunteers
- Not accepted
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 \[PFIC\].
Detailed description
This is an open label study where all participants will receive maralixibat treatment.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Maralixibat | Maralixibat chloride provided in the form of an oral solution (i.e., 5, 10, 15, and 20 mg/mL) * 400 μg/kg maralixibat chloride is equivalent to 380 µg/kg maralixibat free base * 600 μg/kg maralixibat chloride is equivalent to 570 µg/kg maralixibat free base |
Timeline
- Start date
- 2021-09-09
- Primary completion
- 2024-12-17
- Completion
- 2024-12-17
- First posted
- 2021-01-28
- Last updated
- 2025-02-06
Locations
14 sites across 7 countries: United States, Belgium, Brazil, France, Mexico, Poland, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04729751. Inclusion in this directory is not an endorsement.