Alagille SyndromePhase 4NCT05488067
Atorvastatin Therapy on Xanthoma in Alagille Syndrome
A Phase 4 study testing atorvastatin in people with Alagille Syndrome and Atorvastatin, run by Children's Hospital of Fudan University at 1 site in China.
Trial summary
To observe the efficacy and safety of atorvastatin on xanthoma in Alagille syndrome through a prospective study. Read more about this study
- Who is it for?
- Ages 1 day to 17 years
- Conditions the study is for
- Alagille Syndrome, Atorvastatin, Xanthoma
- What is being tested?
- atorvastatin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteChina
- Study dates
- 22 Mar 2022to 1 Aug 2024
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, 201102
About this study
Official title: 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.
Read the 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.
- Conditions
- Alagille Syndrome, Atorvastatin, Xanthoma
- Being tested
- atorvastatin
- Medical subject headings
- Alagille Syndrome, Xanthomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
atorvastatinDrug
Also called Lipitor
Oral atorvastatin treatment for ALGS children with xanthoma
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: atorvastatin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.】
Measured: from enrollment to the 3th/6th month
Secondary outcomes (2)
non-HDL-C change
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】
Measured: from enrollment to the 3th/6th month
Incidence of adverse events
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.
Measured: from enrollment to the 3th/6th month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2022
- Study start22 Mar 2022
- Primary completion1 Aug 2024
- Study completion1 Aug 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (22)Papers the sponsor has linked to this study.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05488067
- Sponsor's protocol ID
- ATO-ALGS-XAN
- Status last verified
- Mar 2026
- First submitted
- 1 Aug 2022
- Last updated
- 27 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Gilbert MA, Bauer RC, Rajagopalan R, Grochowski CM, Chao G, McEldrew D, Nassur JA, Rand EB, Krock BL, Kamath BM, Krantz ID, Piccoli DA, Loomes KM, Spinner NB. Alagille syndrome mutation update: Comprehensive overview of JAG1 and NOTCH2 mutation frequencies and insight into missense variant classification. Hum Mutat. 2019 Dec;40(12):2197-2220. doi: 10.1002/humu.23879. Epub 2019 Aug 26.
PubMed 31343788Ben Ameur S, Chabchoub I, Telmoudi J, Belfitouri Y, Rebah O, Lacaille F, Aloulou H, Mehrzi A, Hachicha M. Management of cholestatic pruritus in children with Alagille syndrome: Case report and literature review. Arch Pediatr. 2016 Dec;23(12):1247-1250. doi: 10.1016/j.arcped.2016.09.004. Epub 2016 Oct 27.
PubMed 28492167Martinsen MH, Klausen IC, Tybjaerg-Hansen A, Hedegaard BS. Autosomal recessive hypercholesterolemia in a kindred of Syrian ancestry. J Clin Lipidol. 2020 Jul-Aug;14(4):419-424. doi: 10.1016/j.jacl.2020.06.002. Epub 2020 Jun 8.
PubMed 32636080Lin M, Dai H, Zhao S. Long-term atorvastatin-ezetimibe-probucol triple therapy for homozygous familial hypercholesterolaemia from early childhood. Cardiol Young. 2016 Jan;26(1):197-201. doi: 10.1017/S1047951115000591. Epub 2015 Apr 24.
PubMed 25907359Sreedharan AV, Pek SLT, Tan TH, Tavintharan S, Yap F. Successful pharmacological management of a child with compound heterozygous familial hypercholesterolemia and review of the recent literature. J Clin Lipidol. 2020 Sep-Oct;14(5):639-645. doi: 10.1016/j.jacl.2020.07.006. Epub 2020 Jul 15.
PubMed 32800790Luirink IK, Hutten BA, Greber-Platzer S, Kolovou GD, Dann EJ, de Ferranti SD, Taylan C, Bruckert E, Saheb S, Oh J, Driemeyer J, Farnier M, Pape L, Schmitt CP, Novoa FJ, Maeser M, Masana L, Shahrani A, Wiegman A, Groothoff JW. Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: Data from an international registry. Atherosclerosis. 2020 Apr;299:24-31. doi: 10.1016/j.atherosclerosis.2020.01.031. Epub 2020 Feb 18.
PubMed 32199148Shurberg JL, Resnick RH, Koff RS, Ros E, Baum RA, Pallotta JA. Serum lipids, insulin, and glucagon after portacaval shunt in cirrhosis. Gastroenterology. 1977 Feb;72(2):301-4.
PubMed 830579Kakaei F, Nikeghbalian S, Kazemi K, Salahi H, Bahador A, Dehghani SM, Dehghani M, Nejatollahi SM, Shamsaeefar A, Khosravi MB, Malek-Hosseini SA. Liver transplantation for homozygous familial hypercholesterolemia: two case reports. Transplant Proc. 2009 Sep;41(7):2939-41. doi: 10.1016/j.transproceed.2009.07.028.
PubMed 19765481Larrosa-Haro A, Saenz-Rivera C, Gonzalez-Ortiz M, Coello-Ramirez P, Vazquez-Camacho G. Lack of cholesterol-lowering effect of graded doses of cholestyramine in children with Alagille syndrome: a pilot study. J Pediatr Gastroenterol Nutr. 2003 Jan;36(1):50-3. doi: 10.1097/00005176-200301000-00011.
PubMed 12499996Sheflin-Findling S, Arnon R, Lee S, Chu J, Henderling F, Kerkar N, Iyer K. Partial internal biliary diversion for Alagille syndrome: case report and review of the literature. J Pediatr Surg. 2012 Jul;47(7):1453-6. doi: 10.1016/j.jpedsurg.2012.04.008.
PubMed 22813814Quek SC, Aw M, Quak SH, Prabhakaran K, Tan KC. Liver transplantation in a child with severe hypercholesterolaemia in Alagille syndrome. Ann Acad Med Singap. 2001 Jan;30(1):44-7.
PubMed 11242624Nakajima H, Tsuma Y, Fukuhara S, Kodo K. A Case of Infantile Alagille Syndrome With Severe Dyslipidemia: New Insight into Lipid Metabolism and Therapeutics. J Endocr Soc. 2022 Jan 18;6(3):bvac005. doi: 10.1210/jendso/bvac005. eCollection 2022 Mar 1.
PubMed 35155971Tapia Ceballos L, Picazo Angelin B, Ruiz Garcia C. [Use of statins in children]. An Pediatr (Barc). 2008 Apr;68(4):385-92. doi: 10.1157/13117712. Spanish.
PubMed 18394386Melvin AJ, Montepiedra G, Aaron L, Meyer WA 3rd, Spiegel HM, Borkowsky W, Abzug MJ, Best BM, Crain MJ, Borum PR, Graham B, Anthony P, Shin K, Siberry GK; P1063 Study Team. Safety and Efficacy of Atorvastatin in Human Immunodeficiency Virus-infected Children, Adolescents and Young Adults With Hyperlipidemia. Pediatr Infect Dis J. 2017 Jan;36(1):53-60. doi: 10.1097/INF.0000000000001352.
PubMed 27749649Hari P, Khandelwal P, Satpathy A, Hari S, Thergaonkar R, Lakshmy R, Sinha A, Bagga A. Effect of atorvastatin on dyslipidemia and carotid intima-media thickness in children with refractory nephrotic syndrome: a randomized controlled trial. Pediatr Nephrol. 2018 Dec;33(12):2299-2309. doi: 10.1007/s00467-018-4036-x. Epub 2018 Aug 8.
PubMed 30091061
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Safety and Efficacy of Atorvastatin on Xanthoma in Alagille Syndrome
- ClinicalTrials.gov ID
- NCT05488067
- Acronym / sponsor ID
- ATO-ALGS-XAN
- Conditions
- Alagille Syndrome, Atorvastatin, Xanthoma
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 22 Mar 2022 (actual)
- Primary completion
- 1 Aug 2024 (actual)
- Study completion
- 1 Aug 2024 (actual)
- First posted
- 4 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To observe the efficacy and safety of atorvastatin on xanthoma in Alagille syndrome through a prospective study.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Atorvastatin+ALGS-Xanthoma | Experimental | Drug: atorvastatinDrug: 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. |
Who may qualify
- Ages
- 1 day to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (7 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| atorvastatinAlso: LipitorOral atorvastatin treatment for ALGS children with xanthoma | Drug | Atorvastatin+ALGS-Xanthoma |
Primary outcome
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.】
2 secondary outcomes
non-HDL-C change
Time frame: from enrollment to the 3th/6th month
Incidence of adverse events
Time frame: from enrollment to the 3th/6th month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2026.
- Background: PubMed 31343788Gilbert MA, Bauer RC, Rajagopalan R, Grochowski CM, Chao G, McEldrew D, Nassur JA, Rand EB, Krock BL, Kamath BM, Krantz ID, Piccoli DA, Loomes KM, Spinner NB. Alagille syndrome mutation update: Comprehensive overview of JAG1 and NOTCH2 mutation frequencies and insight into missense variant classification. Hum Mutat. 2019 Dec;40(12):2197-2220. doi: 10.1002/humu.23879. Epub 2019 Aug 26.
- Background: PubMed 28492167Ben Ameur S, Chabchoub I, Telmoudi J, Belfitouri Y, Rebah O, Lacaille F, Aloulou H, Mehrzi A, Hachicha M. Management of cholestatic pruritus in children with Alagille syndrome: Case report and literature review. Arch Pediatr. 2016 Dec;23(12):1247-1250. doi: 10.1016/j.arcped.2016.09.004. Epub 2016 Oct 27.
- Background: PubMed 32636080Martinsen MH, Klausen IC, Tybjaerg-Hansen A, Hedegaard BS. Autosomal recessive hypercholesterolemia in a kindred of Syrian ancestry. J Clin Lipidol. 2020 Jul-Aug;14(4):419-424. doi: 10.1016/j.jacl.2020.06.002. Epub 2020 Jun 8.
- Background: PubMed 25907359Lin M, Dai H, Zhao S. Long-term atorvastatin-ezetimibe-probucol triple therapy for homozygous familial hypercholesterolaemia from early childhood. Cardiol Young. 2016 Jan;26(1):197-201. doi: 10.1017/S1047951115000591. Epub 2015 Apr 24.
- Background: PubMed 32800790Sreedharan AV, Pek SLT, Tan TH, Tavintharan S, Yap F. Successful pharmacological management of a child with compound heterozygous familial hypercholesterolemia and review of the recent literature. J Clin Lipidol. 2020 Sep-Oct;14(5):639-645. doi: 10.1016/j.jacl.2020.07.006. Epub 2020 Jul 15.
- Background: PubMed 32199148Luirink IK, Hutten BA, Greber-Platzer S, Kolovou GD, Dann EJ, de Ferranti SD, Taylan C, Bruckert E, Saheb S, Oh J, Driemeyer J, Farnier M, Pape L, Schmitt CP, Novoa FJ, Maeser M, Masana L, Shahrani A, Wiegman A, Groothoff JW. Practice of lipoprotein apheresis and short-term efficacy in children with homozygous familial hypercholesterolemia: Data from an international registry. Atherosclerosis. 2020 Apr;299:24-31. doi: 10.1016/j.atherosclerosis.2020.01.031. Epub 2020 Feb 18.
- Background: PubMed 830579Shurberg JL, Resnick RH, Koff RS, Ros E, Baum RA, Pallotta JA. Serum lipids, insulin, and glucagon after portacaval shunt in cirrhosis. Gastroenterology. 1977 Feb;72(2):301-4.
- Background: PubMed 19765481Kakaei F, Nikeghbalian S, Kazemi K, Salahi H, Bahador A, Dehghani SM, Dehghani M, Nejatollahi SM, Shamsaeefar A, Khosravi MB, Malek-Hosseini SA. Liver transplantation for homozygous familial hypercholesterolemia: two case reports. Transplant Proc. 2009 Sep;41(7):2939-41. doi: 10.1016/j.transproceed.2009.07.028.
- Background: PubMed 12499996Larrosa-Haro A, Saenz-Rivera C, Gonzalez-Ortiz M, Coello-Ramirez P, Vazquez-Camacho G. Lack of cholesterol-lowering effect of graded doses of cholestyramine in children with Alagille syndrome: a pilot study. J Pediatr Gastroenterol Nutr. 2003 Jan;36(1):50-3. doi: 10.1097/00005176-200301000-00011.
- Background: PubMed 22813814Sheflin-Findling S, Arnon R, Lee S, Chu J, Henderling F, Kerkar N, Iyer K. Partial internal biliary diversion for Alagille syndrome: case report and review of the literature. J Pediatr Surg. 2012 Jul;47(7):1453-6. doi: 10.1016/j.jpedsurg.2012.04.008.
- Background: PubMed 11242624Quek SC, Aw M, Quak SH, Prabhakaran K, Tan KC. Liver transplantation in a child with severe hypercholesterolaemia in Alagille syndrome. Ann Acad Med Singap. 2001 Jan;30(1):44-7.
- Background: PubMed 35155971Nakajima H, Tsuma Y, Fukuhara S, Kodo K. A Case of Infantile Alagille Syndrome With Severe Dyslipidemia: New Insight into Lipid Metabolism and Therapeutics. J Endocr Soc. 2022 Jan 18;6(3):bvac005. doi: 10.1210/jendso/bvac005. eCollection 2022 Mar 1.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing atorvastatin in people with Alagille Syndrome and Atorvastatin, run by Children's Hospital of Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Grade change of xanthoma (time frame: from enrollment to the 3th/6th month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05488067, which also lists 22 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2026.