Cholesterol; LipidosisPhase 1NCT04903223
Liver Adiposity Effects on Pediatric Statin
A Phase 1 study testing Rosuvastatin 10mg in people with Cholesterol; Lipidosis, run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 8 to 21 yearsHealthy volunteers can take part
- Conditions the study is for
- Cholesterol; Lipidosis
- What is being tested?
- Rosuvastatin 10mg
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Apr 2021to 31 Mar 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 8 to 21 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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.)
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's Mercy Hospital
Kansas City, Missouri, 64108
Jonathan B Wagner, DO, 816-234-3000 ext. 59373, jbwagner@cmh.edu
Jonathan B Wagner, DO
About this study
Official title: Effects of Pediatric Liver Adiposity on Statin Disposition and Response
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
- Being tested
- Rosuvastatin 10mg
- Medical subject headings
- Lipidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rosuvastatin 10mgDrug
Rosuvastatin will be administered to all participants on Study Day #2
| Study group | Role | What they receive |
|---|---|---|
| Baseline Mevalonate and MRI Imaging | No intervention | Not described |
| Mevalonate Change After Rosuvastatin | Experimental | Not described Receives: Drug: Rosuvastatin 10mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2021
- Study start1 Apr 2021
- Primary completion30 Oct 2026 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cholesterol; LipidosisStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jon Wagner, Children's Mercy Hospital Kansas City
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04903223
- Sponsor's protocol ID
- STUDY00001550
- Status last verified
- Nov 2025
- First submitted
- 17 May 2021
- Last updated
- 25 Jun 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Pediatric Liver Adiposity on Statin Disposition and Response
- ClinicalTrials.gov ID
- NCT04903223
- Acronym / sponsor ID
- STUDY00001550
- Conditions
- Cholesterol; Lipidosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Study start
- 1 Apr 2021 (actual)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 31 Mar 2027 (estimated)
- First posted
- 26 May 2021 (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.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Baseline Mevalonate and MRI Imaging | No intervention | – |
| Mevalonate Change After Rosuvastatin | Experimental | Drug: Rosuvastatin 10mg |
Who may qualify
- Ages
- 8 to 21 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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.)
Key exclusion criteria (11 listed)
- 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.
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 |
|---|---|---|
| Rosuvastatin 10mgRosuvastatin will be administered to all participants on Study Day #2 | Drug | Mevalonate Change After Rosuvastatin |
Primary outcome
Evaluate effect of Liver Fat Percentage (on MRI) on AUC
Time frame: 2 years
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.
Evaluate effect of Liver Fat Percentage (on MRI) on change on plasma mevalonate level
Time frame: 2 years
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 2026.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Rosuvastatin 10mg in people with Cholesterol; Lipidosis, run by Children's Mercy Hospital Kansas City at 1 site in United States.
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 11 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: looking for participants now, as of the sponsor's last update on 25 Jun 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 United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jonathan Wagner, DO (816-731-7240, jbwagner@cmh.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate effect of Liver Fat Percentage (on MRI) on AUC (time frame: 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 25 Jun 2026.