The Safety and Efficacy of Atorvastatin on Xanthoma in Alagille Syndrome
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 4
SUMMARY
To observe the efficacy and safety of atorvastatin on xanthoma in Alagille syndrome through a prospective study.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1 day
Maximum Age: 17
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
China
Children's Hospital of Fudan University
RECRUITING
Shanghai
Contact Information
Primary
Jianshe Wang, doctor
jshwang@shmu.edu.cn
18017590987
Backup
Yiqiong Zhang, master
21111240032@m.fudan.edu.cn
13199536035
Time Frame
Start Date: 2022-03-22
Estimated Completion Date: 2025-03-22
Participants
Target number of participants: 10
Treatments
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 \< 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. The maximum dose is 40mg/d and not more than 1mg/kg/d. During the follow-up, the medication was adjusted according to the laboratory results until non-HDL-C≤4.2mmol/L（162 mg/dL）, xanthoma disappear, or the patient had moderate or more serious adverse reactions.
Related Therapeutic Areas
Sponsors
Leads: Children's Hospital of Fudan University