Using atorvastatin to treat xanthomas in children with Alagille syndrome
The Safety and Efficacy of Atorvastatin on Xanthoma in Alagille Syndrome
This study is testing if atorvastatin can help reduce skin bumps called xanthomas in children with Alagille syndrome who have high cholesterol.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 1 Day to 17 Years |
| Sex | All |
| Sponsor | Children's Hospital of Fudan University Academic / other |
| Locations | 1 site (Shanghai, Shanghai) |
| Trial ID | NCT05488067 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the safety and efficacy of atorvastatin in treating xanthomas associated with Alagille syndrome, a condition that can lead to chronic cholestasis in children. The trial will involve children aged 0-17 who have been diagnosed with Alagille syndrome and exhibit skin xanthomas due to high non-HDL cholesterol levels. Participants will receive atorvastatin, a lipid-lowering medication, to determine its effectiveness in reducing xanthomas and improving the quality of life for these patients. The study seeks to provide valuable insights into the management of hypercholesterolemia in young patients with this rare syndrome.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 0-17 years diagnosed with Alagille syndrome who have skin xanthomas and elevated non-HDL cholesterol levels.
Not a fit: Patients who have undergone liver transplantation or those with serious systemic diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the appearance of xanthomas and enhance the quality of life for children with Alagille syndrome.
How similar studies have performed: While there is limited research specifically on atorvastatin for xanthomas in Alagille syndrome, the use of lipid-lowering drugs in similar conditions has shown promise.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Shanghai, Shanghai
- Children's Hospital of Fudan University — Shanghai, Shanghai, China (Recruiting)
Study contacts
- Study coordinator: Jianshe Wang, doctor
- Email: jshwang@shmu.edu.cn
- Phone: 18017590987
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.