drugset / Trial / NCT04729751
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).
NaSingle-groupOpen-labelTreatment
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 \[PFIC\].
Timeline
- Start
- 2021-09-09
- Primary completion
- 2024-12-17
- Completion
- 2024-12-17
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Maralixibat | Small molecule | 400 ug/kg | Oral |
| Subject | Maralixibat | Small molecule | 600 ug/kg | Oral |