A new study testing the safety and efficacy of atorvastatin in treating xanthomas in patients with Alagille syndrome (ALGS) is recruiting participants.

The prospective phase 4 study aims to clarify the safety and efficacy of atorvastatin to provide a reference for the treatment of xanthomas in children with the disease.

The study will recruit 10 patients, aged 1 day to 17 years, who meet the diagnostic criteria for ALGS. Those who have had liver transplants are not eligible.

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Participants will be given oral atorvastatin tablets for 3 months to 1 year. The dosage will be adjusted according to age. Those who are under the age of 1 year will receive 0.25 mg/kg per day, those aged 1 to 5 years will receive 2.5 mg per day, those aged 6 to 9 years will receive 5 mg per day, and those aged 10 years and above will receive 10 mg per day.

Read more about the classic criteria for the diagnosis of Alagille syndrome

The primary outcome measure will be the grade change of xanthoma from enrollment to months 3 and 6. Secondary outcome measures will be the change in serum concentration of low-density lipoprotein cholesterol and the incidence of adverse events such as rhabdomyolysis and myopathy, high liver enzyme levels, nasopharyngitis, muscle pain, diarrhea, nausea, fever, urinary tract infection, joint swelling, epistaxis, and urticaria.

The trial started on March 22, 2022, and is expected to be completed on March 22, 2025.

Xanthomas are common in patients with ALGS, especially in more severe cases. These can be disfiguring and cause serious physical and mental damage. 

Atorvastatin, marketed under the brand name Lipitor®, among others, is a statin used to lower the levels of low-density lipoprotein cholesterol. Lipid-lowering drugs can help treat xanthomas, however, there are currently no guidelines for their use in children with hypercholesterolemia under the age of 6 years.


Atorvastatin therapy on xanthoma in Alagille syndrome. ClinicalTrials.gov. August 4, 2022. Accessed August 10, 2022.