Cholestatic Pruritus and Bile Acid–Related Itch Disorders
Mirum Pharmaceuticals Inc. focuses on clinical research in cholestatic pruritus, including itching associated with primary biliary cholangitis and primary sclerosing cholangitis. The trials examine symptom control in liver-related itch conditions and the role of targeted therapies in reducing pruritus severity.
- Primary biliary cholangitis
- Primary sclerosing cholangitis
- Itch severity assessment
- Bile acid modulation
These studies place emphasis on patient-reported itch outcomes and on treatment options for chronic pruritus linked to cholestatic liver disease.
Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis
The sponsor also funds research in rare pediatric cholestatic liver diseases, especially Alagille syndrome and progressive familial intrahepatic cholestasis. The clinical program addresses long-term disease management, symptom burden, and liver-related outcomes in affected children and adolescents.
- Pediatric cholestatic liver disease
- Long-term safety
- Clinical outcomes
- Growth and development
Research in this area includes monitoring of liver function, cholestasis biomarkers, and complications associated with progressive disease.
Long-Term Management of Chronic Liver Disease
Mirum’s trial portfolio includes extended clinical follow-up in chronic liver disease settings, with attention to tolerability, durability of response, and ongoing symptom control in patients treated for cholestatic conditions.
- Liver toxicity monitoring
- Portal hypertension
- Liver fibrosis
- Liver transplant status
These studies reflect interest in disease progression markers, hepatic complications, and long-term therapeutic management across rare liver disorders.
Fragile X Syndrome
In addition to hepatology-focused programs, the sponsor supports research in Fragile X syndrome, a neurodevelopmental disorder. This work evaluates treatment tolerability and safety in male participants affected by the condition.
- Neurodevelopmental disorders
- Safety and tolerability
- Treatment-related adverse events
This area expands the sponsor’s clinical interest beyond liver disease into targeted investigation for inherited neurological conditions.
