Liver Adiposity Effects on Pediatric Statin

NCT04903223 | Recruiting Phase 1 FDA Drug

• 1 other identifier: STUDY00001550
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Single center, open-label, prospective investigation to quantify the effects hepatocellular fat has on hepatic statin transport and response in children and adolescents in obese and non-obese children and adolescents 8-21 years of age with normal, wild-type SLCO1B1 c.521TT genotype that are dosed rosuvastatin

Conditions

Cholesterol; Lipidosis

Outcome Measures

Primary Outcomes (2)

• Evaluate effect of Liver Fat Percentage (on MRI) on AUC

  We expect that increased liver adiposity will be associated with higher systemic statin exposure and thus, place those with higher liver adiposity fraction at increased risk for drug toxicity.

  2 years

• Evaluate effect of Liver Fat Percentage (on MRI) on change on plasma mevalonate level

  We expect that increased liver adiposity will be associated with an attenuated mevalonate response to a statin in obese children placing those with increased liver adiposity at risk for treatment failure.

  2 years

Study Arms (2)

Baseline Mevalonate and MRI Imaging

NO INTERVENTION

Mevalonate Change After Rosuvastatin

EXPERIMENTAL

Drug: Rosuvastatin 10mg

Interventions

Rosuvastatin 10mg DRUG

Rosuvastatin will be administered to all participants on Study Day #2

Mevalonate Change After Rosuvastatin

Eligibility Criteria

• Age: 8 Years - 21 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.)

Sponsors & Collaborators

• Children's Mercy Hospital Kansas City: lead

Study Sites (1)

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

RECRUITING

MeSH Terms

Conditions

Lipidoses

Interventions

Rosuvastatin Calcium

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Sulfonamides; Amides; Organic Chemicals; Fluorobenzenes; Hydrocarbons, Fluorinated; Hydrocarbons, Halogenated; Hydrocarbons; Sulfones; Sulfur Compounds; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Central Study Contacts

Jonathan Wagner, DO

CONTACT

816-731-7240; jbwagner@cmh.edu

Jazzie K Holliday

CONTACT

816-302-3500; jkholliday@cmh.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: This is a single center, open-label, prospective, investigation to quantify the effects hepatocellular fat has on hepatic statin transport (SA1) and response (SA2) in children and adolescents.

Study Record Dates

• First Submitted: May 17, 2021
• First Posted: May 26, 2021
• Study Start: April 1, 2021
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: November 26, 2025

Record last verified: 2025-11

Locations

US (1)