BSEP Function Rescue During Childhood Inhereditary Cholestatic Diseases

• Status: Not yet recruiting
• Trial end date: 2023-12-30
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of the study is to improve the prognosis of inhereditary cholestasis caused by ABCB11 gene mutations by using BSEP function rescue drugs

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Fudan University
• Treatments: 4-phenylbutyric acid

Criteria

Inclusion Criteria:

• with signed informed consent form from the guardian, and the patient if applicable.

• aged from 2 month to 18 years old.

• with cholestatic disease caused by ABCB11 biallelic mutation.

• Long-term residence in China.

Exclusion Criteria:

• Currently receiving or previously received experimental drugs.

• The child is already in the stage of liver failure, or in unstable state that are not suitable for drug treatment according to the researcher's judgment: serious complications such as bleeding tendency and skin rash.

• accompany with other chronic liver disease (viral hepatitis B and C, autoimmune hepatitis, wilson disease, cystic fibrosis, primary biliary cirrhosis, biliary atresia, sclerosing cholangitis, bile acid synthesis defects, and infections, cholestasis caused by space-occupying and other reasons).

• Suffered from congenital TORCHES infection, including toxoplasma gondii, rubella virus, cytomegalovirus, herpes simplex virus, EB virus, syphilis, HIV, etc.

• With any other major medical conditions that may affect drug absorption, metabolism, or excretion based on the researcher's judgment.

• Known or suspected hypersensitivity to any experimental drugs or their indigents.

• Patients with alcohol or drug dependence.

• In receiving any investigational drugs or within 60 days before enrollment.