Mitochondrial DiseasesPhase 4NCT03351998

Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic Capacity

A Phase 4 study testing Lipitor 20Mg Tablet and Lipitor 80Mg Tablet in people with Mitochondrial Diseases, run by University of Kansas Medical Center at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle health and function, and cardiovascular fitness. Read more about this study

Who is it for?: Ages 35 to 65 yearsHealthy volunteers can take part
Conditions the study is for: Mitochondrial Diseases
What is being tested?: Lipitor 20Mg Tablet and Lipitor 80Mg Tablet
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 68 participants
Where: 2 sitesUnited States
Study dates: 22 Feb 2018to 7 Jul 2023
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 35 to 65 years
All sexes
Healthy volunteers accepted
Adult, Older adult

Who may be able to join

Body mass index (BMI) between 25-43
Weight stable (no more than 5% change in body weight the previous 3 months)
>5% risk for a cardiovascular event in the next 10 years according to the 2013 American College of Cardiology/American Heart Association risk calculator and/or 2 out of 5 metabolic syndrome risk factors (Triglycerides ≥ 150 mg/dL; HDL ≤ 40 mg/dL; Glucose ≥ 100mg/dL; Waist Circumference ≥ 102cm for males, 88cm for females; Blood pressure: ≥ 130mmHg systolic and/or 85mmHg diastolic or being treated for hypertension) and/or LDL-Cholesterol >120 mg/dl.
Stable doses of medications for 90 days
Willing to stop all Nonsteroidal Antiinflammatory Drugs (NSAIDs) and aspirin for 7 days prior to muscle biopsy

Who cannot join

Smoking
Previous use of statins
Use of other medications or supplements that affect lipid profiles or body weight in the last 6 months (e.g., fibric acids, bile acid sequestrants, nicotinic acids, fish oil)
Diagnosis of chronic diseases including CVD, other metabolic diseases (e.g., thyroid), current diagnosis and active treatment of cancer, HIV, or acquired immunodeficiency syndrome.
Diagnosis of type 1 or type 2 diabetes at the time of screening (fasting blood glucose >126mg/dL). If evidence of type 2 diabetes outcome measures is detected during the course of the study (fasting glucose > 126 mg/dl or HbA1c > 6.5%) we will notify the participant to contact their physician.
History of abnormal bleeding problems

Show all 11Show fewer

Currently taking (within the last 10 days) anti-platelet medication (Plavix), Warfarin, and other anti-coagulants (eliquis, pradaxa, and xarelto) medications.
>2 fold upper normal limit (UNL) for alanine aminotransferase (ALT) or creatinine
Women who are pregnant or breastfeeding
Individuals with polymorphisms (SLCO1B1 and GATM) known to be associated with susceptibility for statin induced myopathies (tested at screening)
Currently enrolled in another research study

Trial locations and contacts

2 sites in 1 country.

United States2 sites

East Carolina University
Greenville, North Carolina, 27858
University of Kansas Medical Center
Kansas City, Kansas, 66160

Other trials in United States

About this study

The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle health and function, and cardiovascular fitness.

Conditions: Mitochondrial Diseases
Being tested: Lipitor 20Mg Tablet, Lipitor 80Mg Tablet, Placebo Oral Tablet
Keywords: insulin sensitivity, cardiorespiratory fitness, skeletal muscle mitochondrial function
Medical subject headings: Mitochondrial Diseases, Insulin Resistance

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Lipitor 20Mg TabletDrug
Also called atorvastatin
20 mg/day pills.
Lipitor 80Mg TabletDrug
Also called atorvastatin
80 mg/day pills.
Placebo Oral TabletDrug
Also called Placebo
Matching placebo pill.

Study groups in NCT03351998
Study group	Role	What they receive
Placebo	Placebo comparator	Participants receiving matching placebo oral tablet. Receives: Drug: Placebo Oral Tablet
Low dose statin	Active comparator	Participants will receive Lipitor 20Mg Tablet to take daily. Receives: Drug: Lipitor 20Mg Tablet
High dose statin	Active comparator	Participants will receive Lipitor 80Mg Tablet to take daily. Receives: Drug: Lipitor 80Mg Tablet

What is being measured

The primary outcome is the main question the study is designed to answer.

Difference in Mitochondrial Respiratory Function
Measured: value at 12 months minus value at baseline

Secondary outcomes (1)

Change in VO2 Max
Measured: value at 12 months minus value at baseline

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

22 Feb 20187 Jul 2023

First posted24 Nov 2017
Study start22 Feb 2018
Primary completion7 Jul 2023
Study completion7 Jul 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Mitochondrial DiseasesStudies of the same condition, open and under way.
More trials run by University of Kansas Medical CenterThe 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: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 68 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03351998
Sponsor's protocol ID: STUDY00140789
Other IDs: R01AR071263
Status last verified: Nov 2024
First submitted: 20 Nov 2017
Last updated: 18 Dec 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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All Mitochondrial Diseases trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Impact of Statin Therapy on Muscle Mitochondrial Function and Aerobic Capacity
ClinicalTrials.gov ID: NCT03351998
Acronym / sponsor ID: STUDY00140789
Conditions: Mitochondrial Diseases
Keywords: insulin sensitivity, cardiorespiratory fitness, skeletal muscle mitochondrial function
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 68 participants (actual)
Lead sponsor: University of Kansas Medical CenterUniversity, hospital or other
Collaborators: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Study start: 22 Feb 2018 (actual)
Primary completion: 7 Jul 2023 (actual)
Study completion: 7 Jul 2023 (actual)
First posted: 24 Nov 2017 (actual)
Results: Posted 18 Dec 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle health and function, and cardiovascular fitness.

Study design

Primary purpose: Prevention
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 3

Arm	Type	Receives
Placebo	Placebo comparator	Drug: Placebo Oral TabletParticipants receiving matching placebo oral tablet.
Low dose statin	Active comparator	Drug: Lipitor 20Mg TabletParticipants will receive Lipitor 20Mg Tablet to take daily.
High dose statin	Active comparator	Drug: Lipitor 80Mg TabletParticipants will receive Lipitor 80Mg Tablet to take daily.

Who may qualify

Ages: 35 to 65 years
Sex: All
Healthy volunteers: Accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Body mass index (BMI) between 25-43
Weight stable (no more than 5% change in body weight the previous 3 months)
>5% risk for a cardiovascular event in the next 10 years according to the 2013 American College of Cardiology/American Heart Association risk calculator and/or 2 out of 5 metabolic syndrome risk factors (Triglycerides ≥ 150 mg/dL; HDL ≤ 40 mg/dL; Glucose ≥ 100mg/dL; Waist Circumference ≥ 102cm for males, 88cm for females; Blood pressure: ≥ 130mmHg systolic and/or 85mmHg diastolic or being treated for hypertension) and/or LDL-Cholesterol >120 mg/dl.
Stable doses of medications for 90 days
Willing to stop all Nonsteroidal Antiinflammatory Drugs (NSAIDs) and aspirin for 7 days prior to muscle biopsy

Key exclusion criteria (11 listed)

Smoking
Previous use of statins
Use of other medications or supplements that affect lipid profiles or body weight in the last 6 months (e.g., fibric acids, bile acid sequestrants, nicotinic acids, fish oil)
Diagnosis of chronic diseases including CVD, other metabolic diseases (e.g., thyroid), current diagnosis and active treatment of cancer, HIV, or acquired immunodeficiency syndrome.
Diagnosis of type 1 or type 2 diabetes at the time of screening (fasting blood glucose >126mg/dL). If evidence of type 2 diabetes outcome measures is detected during the course of the study (fasting glucose > 126 mg/dl or HbA1c > 6.5%) we will notify the participant to contact their physician.

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
Lipitor 20Mg TabletAlso: atorvastatin20 mg/day pills.	Drug	Low dose statin
Lipitor 80Mg TabletAlso: atorvastatin80 mg/day pills.	Drug	High dose statin
Placebo Oral TabletAlso: PlaceboMatching placebo pill.	Drug	Placebo

Primary outcome

Difference in Mitochondrial Respiratory Function
Time frame: value at 12 months minus value at baseline

1 secondary outcome

Change in VO2 Max
Time frame: value at 12 months minus value at baseline

Results and safety

Results posted to the registry on 18 Dec 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	23	16
Low Dose Statin	24	19
High Dose Statin	21	17

Primary outcome: Difference in Mitochondrial Respiratory Function

Time frame: value at 12 months minus value at baseline. Mean (Standard Deviation). Unit: pmols/s/mg

Measure	Placebo n = 16	Low Dose Statin n = 17	High Dose Statin n = 16
Value	36 (366)	32 (213)	-3 (180)

Placebo: Mean Difference (Net): 36.02, p = 0.6051, signed rank test
Low Dose Statin: Mean Difference (Net): 31.89, p = 0.9434, signed rank test
High Dose Statin: Mean Difference (Net): -3.39, p = 0.9408, t-test, 2 sided

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Participants were contacted weekly by phone or email and prompted to disclose any adverse events. Additional adverse events could be reported if participant contacted study team outside of the usual weekly check in. Adverse events were monitored until symptoms subsided or adverse event ended or until the end of the 12 month intervention period.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	1 of 23 (4.3%)	15 of 23 (65.2%)	0 of 23
Low Dose Statin	0 of 24 (0%)	17 of 24 (70.8%)	0 of 24
High Dose Statin	0 of 21 (0%)	14 of 21 (66.7%)	0 of 21

Limitations and caveats (from the sponsor): The COVID-19 epidemic started in the middle of the trial forcing us to stop recruitment for four months. Furthermore, the continuation of the pandemic slowed recruitment for the rest of the trial and significantly impacted n size. There was also a national dextrose shortage following a hurricane in Puerto Rico. As a result many of the IVGTT were not conducted.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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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.

John Thyfault, PhD principal investigator

University of Kansas Medical Center

2 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Lipitor 20Mg Tablet and Lipitor 80Mg Tablet in people with Mitochondrial Diseases, run by University of Kansas Medical Center at 2 sites 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 5 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: finished, as of the sponsor's last update on 18 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites 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. Some participants in this study may receive a placebo. 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?

Yes. Results are posted in the registry (first posted 18 Dec 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Difference in Mitochondrial Respiratory Function (time frame: value at 12 months minus value at baseline). 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 NCT03351998.

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 18 Dec 2024.