Effects of liver fat on statin response in children

Effects of Pediatric Liver Adiposity on Statin Disposition and Response

Phase 1 Interventional Children's Mercy Hospital Kansas City · NCT04903223

This study tests how liver fat affects how well the cholesterol medicine rosuvastatin works in children and teens with high LDL cholesterol.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment50 (estimated)
Ages8 Years to 21 Years
SexAll
SponsorChildren's Mercy Hospital Kansas City Academic / other
Locations1 site (Kansas City, Missouri)
Trial IDNCT04903223 on ClinicalTrials.gov

What this trial studies

This interventional study investigates how hepatocellular fat influences the transport and effectiveness of the statin rosuvastatin in children and adolescents aged 8-21 years. It focuses on both obese and non-obese participants with elevated LDL cholesterol levels and a specific genetic profile (SLCO1B1 c.521TT). The study is conducted at a single center and aims to quantify the relationship between liver fat and statin response, providing insights into personalized treatment approaches for pediatric patients. Participants will receive a 10mg dose of rosuvastatin after meeting specific eligibility criteria.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 8-21 years with LDL cholesterol levels above 130mg/dl and the SLCO1B1 c.521TT genotype.

Not a fit: Patients who are pregnant, currently on statin therapy, or have significant underlying health issues may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to more effective cholesterol management strategies in children, particularly those with liver fat issues.

How similar studies have performed: While there is ongoing research in pediatric statin therapy, this specific investigation into liver fat's effects on statin response is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* 8-21 years
* LDL cholesterol \>130mg/dl (\>95% percentile)
* SLCO1B1 c.521TT genotype
* Provide informed permission-assent(\<18 yrs.) or consent (≥18 yrs.)
* Fasting overnight (\~8 hrs.)
* Enrolled in Cardiology Pharmacogenomic Repository

Exclusion Criteria:

* Pregnancy
* Non-fasting
* Non-removable metal in body or MRI unsafe
* Currently on statin therapy and unwilling to wash out of statin therapy for at least 4 weeks prior to Visit 1 and throughout the duration of the study.
* Underlying unrepaired congenital or acquired cardiovascular defects or repaired congenital or acquired cardiovascular defects with hemodynamically significant residual disease.
* History of underlying or laboratory evidence of underlying intestinal, metabolic, autoimmune, renal disease that can alter rosuvastatin disposition\* (absorption, metabolism, distribution, or clearance)
* Pharmacotherapy that interacts with statins (OATP1B1 inducers/inhibitors) \*
* Inability to swallow a tablet
* \>5x the age-specific upper limit of normal for AST, ALT, total and conjugated bilirubin
* Diarrhea in the last 24 hours
* Anything that would exclude a participant from completing an MRI, such as pacemakers, claustrophobia, or body habitus (e.g., weight greater than 350 lbs.)

Where this trial is running

Kansas City, Missouri

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions CholesterolLipidosis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.