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).
- Conditions
- Alagille SyndromeProgressive Familial Intrahepatic CholestasisCholestatic Liver Disease
- Interventions
- Registration Number
- NCT04729751
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Brief 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 27
- Body weight of ≥2.5 kg
- <12 months of age at the baseline visit (ROW). >31 days and <12 months of age at the baseline visit (US).
- Gestational age ≥36 weeks at birth. For children born with gestational age between 32 and 36 weeks, a postmenstrual age of ≥36 weeks is required.
- Diagnosis of PFIC or ALGS
Exclusion criteria:
- Predicted complete absence of bile salt excretion pump (BSEP) function
- History of surgical disruption of the enterohepatic circulation
- History of liver transplant or imminent need for liver transplant
- Decompensated cirrhosis
- Presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (e.g., inflammatory bowel disease), per investigator discretion
- Presence of other significant liver disease or any other conditions or abnormalities which, in the opinion of the investigator or medical monitor, may compromise the safety of the participant or interfere with the participant's participation in or completion of the study
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Maralixibat Maralixibat Participants will receive up to 600 μg/kg twice daily (PFIC) or up to 400 μg/kg once daily (ALGS) over 13 weeks in the core study and for the duration of the Long Term Extension (LTE) where applicable.
- Primary Outcome Measures
Name Time Method Frequency of treatment-emergent adverse events [TEAEs] From Baseline through to Week 13
- Secondary Outcome Measures
Name Time Method To evaluate the effect on liver enzymes (ALT, AST) and bilirubin From Baseline through to Week 13 To assess the plasma level of maralixibat in infant participants At Baseline, Week 6, Week 10, Week 13 or Early Termination Visit Change in fasting serum bile acid (sBA) levels From Baseline through to Week 13 To evaluate the effect on LSVs From Baseline through to Week 13
Trial Locations
- Locations (14)
Instytut Pomnik-Centrum Zdrowia Dziecka
🇵🇱Warsaw, Poland
King's College Hospital
🇬🇧London, United Kingdom
University of California - San Francisco
🇺🇸San Francisco, California, United States
Medstar Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium
Hôpital Kremlin Bicêtre
🇫🇷Le Kremlin-Bicêtre, France
Hopital Necker
🇫🇷Paris, France
Children Hospital LA
🇺🇸Los Angeles, California, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Sociedade Beneficente de Senhoras - Hospital Sírio-Libanês
🇧🇷São Paulo, Brazil
Consultorio de Joshue David Covarrubias Esquer
🇲🇽Zapopan, Mexico
Ochsner Hospital for Children
🇺🇸New Orleans, Louisiana, United States