Atorvastatin Therapy on Xanthoma in Alagille Syndrome

March 24, 2026 updated by: Children's Hospital of Fudan University

The Safety and Efficacy of Atorvastatin on Xanthoma in Alagille Syndrome

To observe the efficacy and safety of atorvastatin on xanthoma in Alagille syndrome through a prospective study.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Alagille syndrome (ALGS, OMIM 118450) is an important cause of chronic cholestasis in children, and the incidence rate is about 1:30000~1:50000. Most patients with ALGS have hypercholesterolemia. In severe cases, multiple xanthomas can be seen, and some patients are accompanied by severe itching and pain. Disfigured xanthomas affect the normal social interaction of patients, thereby causing physical and mental damage to children. At present, xanthoma caused by hypercholesterolemia can be cured by treating the primary disease, taking lipid-lowering drugs (such as bile acid chelators, ezetimibe, statins, etc.), or lipoprotein apheresis. If it affects the beauty or function, local treatment such as 33% trichloroacetic acid dot coating, carbon dioxide laser, liquid nitrogen freezing or surgical resection is feasible, and even surgical operation( such as portal vena cava anastomosis, liver transplantation). Compared with expensive lipoprotein apheresis and other invasive therapies, taking lipid-lowering drug has the advantages of higher acceptance, lower cost and higher safety. However, at present, there are no guidelines for application of oral lipid-lowering drugs in children under 6 years old with hypercholesterolemia. Therefore, the purpose of this study is to clarify the safety and efficacy of atorvastatin on xanthoma in ALGS , so as to provide reference for the treatment of ALGS patients' xanthomas.

Risk prevention and treatment: The patients began to take atorvastatin from a small dose, followed up closely in the early stage (2-4 weeks) to see if the patients had obvious discomfort such as myalgia, and monitored the changes of Biochemistry (CK, ALT, AST, etc.). If moderate or more serious adverse reactions occurred during the trial or the following laboratory abnormalities occurred (CK exceeded 10 times the upper limit of normal; ALT or AST had been continued to rise, exceeding 2 times the baseline value), atorvastatin was temporarily stopped, and patients should be rechecked within 2 weeks. It is necessary to reevaluate and decide whether to restart atorvastatin treatment.

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 201102
        • Children's Hospital of Fudan University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 day to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Atorvastatin+ALGS-Xanthoma

Drug: atorvastatin Dosage form: tablet Route of administration: oral Duration: 6 months （After 6 months of medication, according to the actual situation of the patient, choose to maintain the original dosage, gradually reduce the dosage or stop the medication）

Administration method:

Initial dose: 1) Categorized by age:① < 1 year old: 1.25mg/d, qd; ② 1-5 years old: 2.5mg/d, qd; ③ 6-9 years old: 5mg/d, qd; ④ ≥ 10 years old: 10mg/d, qd. 2) Categorized by body weight:①5~<10kg: 1.25mg/d, qd; ②10~<20kg: 2.5mg/d, qd; ③20~<30kg: 5mg/d, qd; ④≥30kg: 10mg/d, qd.

Maximum Dose: ①Low myopathy risk SLCO1B1 genotype (521TT): 1 mg/kg/d, not exceeding 40 mg/d; ②Moderate/high myopathy risk SLCO1B1 genotype (521TC/521CC): 0.33 mg/kg/d, not exceeding 10mg/d.

During the follow-up, the medication was adjusted according to the laboratory results until non-HDL-C≤4.2mmol/L（162 mg/dL）, or the patient had moderate or more serious adverse reactions.
Drug: atorvastatin
Oral atorvastatin treatment for ALGS children with xanthoma
Other Names:
  • Lipitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Grade change of xanthoma
Time Frame: from enrollment to the 3th/6th month
The grade change of xanthoma would be assessed at the 3th/6th month after enrollment 【Explanation：Xanthomas were graded as 0 = none, 1 = minimal, 2 =moderate, 3 = disfiguring, and 4 = disabling. Minimal xanthomas signified fewer than 20 scattered individual lesions, moderate represented more than 20 lesions that did not interfere with or limit activities, disfiguring represented large numbers of lesions that by their large numbers or size caused distortion of the face or xtremities, and disabling signified that the xanthomas interfered with function (such as hand use or ability to walk) because of excess size or number.】
from enrollment to the 3th/6th month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of adverse events
Time Frame: from enrollment to the 3th/6th month
It is a binary variable. Incidence Rates for adverse event（including：rhabdomyolysis and myopathy，liver enzymes exceeded twice the baseline value，nasopharyngitis, muscle pain，diarrhea，nausea, fever，urinary tract infection, joint swelling，epistaxis，urticaria，etc.）would be calculated.
from enrollment to the 3th/6th month
non-HDL-C change
Time Frame: from enrollment to the 3th/6th month
The non-HDL-C change would be measured at the 3th/6th month after enrollment 【Explanation: non-HDL-C is defined as the difference between total cholesterol and HDL-C】
from enrollment to the 3th/6th month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Hospital of Fudan University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 22, 2022

Primary Completion (Actual)

August 1, 2024

Study Completion (Actual)

August 1, 2024

Study Registration Dates

First Submitted

August 1, 2022

First Submitted That Met QC Criteria

August 3, 2022

First Posted (Actual)

August 4, 2022

Study Record Updates

Last Update Posted (Actual)

March 27, 2026

Last Update Submitted That Met QC Criteria

March 24, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATO-ALGS-XAN

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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