Atorvastatin Therapy on Xanthoma in Alagille Syndrome

NCT05488067 | Completed Phase 4

• 1 other identifier: ATO-ALGS-XAN
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Atorvastatin; Xanthoma; Alagille Syndrome

Outcome Measures

Primary Outcomes (1)

• Grade change of xanthoma

  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 Outcomes (2)

• non-HDL-C change

  from enrollment to the 3th/6th month

• Incidence of adverse events

  from enrollment to the 3th/6th month

Study Arms (1)

Atorvastatin+ALGS-Xanthoma

EXPERIMENTAL

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

Interventions

atorvastatin DRUG

Oral atorvastatin treatment for ALGS children with xanthoma

Also known as: Lipitor

Atorvastatin+ALGS-Xanthoma

Eligibility Criteria

• Age: 1 Day - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Location

Related Publications (22)

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MeSH Terms

Conditions

Alagille Syndrome; Xanthomatosis

Interventions

Atorvastatin

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pyrroles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heptanoic Acids; Fatty Acids; Lipids

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2022
• First Posted: August 4, 2022
• Study Start: March 22, 2022
• Primary Completion: August 1, 2024
• Study Completion: August 1, 2024
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)