Atorvastatin Therapy on Xanthoma in Alagille Syndrome

Launched by CHILDREN'S HOSPITAL OF FUDAN UNIVERSITY · Aug 3, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the effects of a medication called atorvastatin on a skin condition known as xanthoma in patients with Alagille Syndrome, a genetic disorder that affects the liver and other parts of the body. The goal is to see if atorvastatin can help reduce xanthomas and improve overall health. The trial is currently looking for participants, including children and young adults aged 0 to 17 years, who have been diagnosed with Alagille Syndrome and have xanthomas that are not improving with other treatments. Participants must also have high levels of cholesterol before starting the atorvastatin treatment.

Eligible participants can expect to be monitored closely throughout the study to assess how well the medication works and whether it is safe for them. It’s important to note that individuals who have had a liver transplant, have certain serious health conditions, or are allergic to atorvastatin cannot participate. This trial offers a chance to explore a new treatment option for managing xanthomas in Alagille Syndrome, which may lead to better care for young patients facing this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Meet the ALGS diagnostic criteria;
• • Xanthoma of skin;
• • Before treatment with atorvastatin,non-HDL-C≥5.76mmol/L（223 mg/dL）;
• • Informed consent;
• • Age 0-17 years old, male or female;
• • Taking bile acid chelator (colenemide) has no obvious effect or intolerance.
• Exclusion Criteria:
• • Liver transplantation has been performed;
• • In the recovery period of cholestasis, xanthoma is obviously subsiding;
• • Patients with serious systemic diseases and unstable vital signs;
• • Progressive active liver injury, such as continuous increase of transaminase;
• • Serious myopathy;
• • Known to be allergic to any component of atorvastatin;
• • The weight is less than 5kg.