MitoQ for Sickle Cell Disease

(MitoQ Trial)

Recruiting at 4 trial locations

Overseen By Mikhil N Bamne, PhD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: University of Pittsburgh

Must not be taking: Anti-platelet

Disqualifiers: Pregnancy, Hypertension, Hemodialysis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 2 weeks

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if MitoQ, an antioxidant supplement, can improve platelet function in individuals with sickle cell disease (SCD). SCD causes red blood cells to become misshapen, leading to various health issues. Participants will take MitoQ daily for 14 days to assess its effects. African Americans with sickle cell anemia who are not on anti-platelet medications or undergoing transfusion may qualify. Healthy African American individuals can also participate as a control group. As an unphased trial, this study provides a unique opportunity to explore how MitoQ might benefit those with sickle cell disease.

Will I have to stop taking my current medications?

The trial requires that you stop taking anti-platelet medications at least 4 weeks before joining. If you're on such medications, you'll need to stop them to participate.

Is there any evidence suggesting that MitoQ is likely to be safe for humans?

Research has shown that MitoQ is generally safe for use. In earlier studies, even at high doses, MitoQ did not cause kidney damage compared to a placebo (a pill with no active ingredient). This indicates that MitoQ is safe for the kidneys, which supports its overall safety profile. MitoQ is also available as a dietary supplement, meaning it has already passed some safety checks. However, it has not been tested in individuals with sickle cell disease. Therefore, while current information is reassuring, this trial is crucial to confirm its safety specifically for people with sickle cell disease. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike the standard treatments for sickle cell disease, which often focus on managing pain and preventing complications, MitoQ is unique because it works at a cellular level to combat oxidative stress. MitoQ is an antioxidant that specifically targets mitochondria, the powerhouses of our cells, and may help protect cells from damage caused by sickle cell-related oxidative stress. Researchers are excited about MitoQ because it offers a new way of addressing the root causes of cell damage in sickle cell disease, potentially improving patients' quality of life and reducing disease symptoms.

What evidence suggests that MitoQ might be an effective treatment for sickle cell disease?

Research has shown that MitoQ, a strong antioxidant, might help with sickle cell disease by improving platelet function. Studies have found that MitoQ can lower the amount of harmful oxygen-related molecules in cells. This is important because sickle cell disease involves oxidative stress, which harms cells. MitoQ has also proven to be 24% more effective than CoQ10 at reducing certain harmful substances in mitochondria, the parts of cells that produce energy. Additionally, it helps restore balance in cell function, which could benefit people with sickle cell disease. In this trial, sickle cell patients will receive MitoQ to evaluate its effectiveness in managing the disease. These promising effects suggest MitoQ could effectively manage sickle cell disease. ^{2 4 5 6 7}

Who Is on the Research Team?

Ramasubramanian Kalpatthi, MD

Principal Investigator

University of Pittsburgh

Are You a Good Fit for This Trial?

This trial is for African American adults over 18 with sickle cell anemia. Healthy African American adults can also participate as controls. People cannot join if they are pregnant, have known high blood pressure, use anti-platelet medication, had a transfusion within the last month, require hemodialysis or treatment for obstructive sleep apnea.

Inclusion Criteria

None. (This criterion is not acceptable in a clinical trial as it is discriminatory.)

The study is not looking for African American healthy individuals.

This term by itself is not a clear exclusion criteria. Please provide more context or information to help me clarify it for you.

Exclusion Criteria

I have not used anti-platelet medication or had a transfusion in the last 4 weeks.

You are pregnant.

I have been diagnosed with high blood pressure.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral MitoQ (20mg once a day) for 14 days to assess its effect on platelet function and vascular dysfunction in SCA patients

2 weeks

Baseline and end of treatment visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of adverse events and changes in blood pressure and hemolytic markers

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• MitoQ

Trial Overview The study is testing MitoQ, an antioxidant supplement not yet studied in sickle cell disease patients. It aims to see if MitoQ can improve how platelets function and reduce harmful oxygen-related molecules in those with sickle cell disease.

How Is the Trial Designed?

Group I: Sickle cell patients Experimental Treatment 1 Intervention

Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days)

Group II: Non Sickle cell Control subjects Active Control 1 Intervention

Normal control subjects administered oral MitoQ (20mg once a day for 14 days)

Who Is Running the Clinical Trial?

University of Pittsburgh

Lead Sponsor

Trials

1,820

Recruited

16,360,000+

Published Research Related to This Trial

MitoQ, a targeted antioxidant, effectively reduces oxidative DNA damage in T lymphocytes from older adults when exposed to oxidative stress, indicating its potential to improve immune function with age.

Higher concentrations of mitoQ (0.5 and 1.0 μM) significantly decreased endogenous DNA damage, suggesting that it may help combat age-related decline in immune cell function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An investigation of the effects of MitoQ on human peripheral mononuclear cells. Marthandan, S., Murphy, MP., Billett, E., et al.[2021]

Mitoquinone (MitoQ(10)) is effectively absorbed in the intestines, with 18-41% of the administered dose accumulating intracellularly, indicating its potential as a treatment for neurodegenerative diseases.

The absorption of MitoQ(10) can be significantly enhanced by the presence of serum proteins like bovine serum albumin, suggesting that its bioavailability may improve in real-life conditions despite some limitations from metabolism and transport proteins.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transport and metabolism of MitoQ10, a mitochondria-targeted antioxidant, in Caco-2 cell monolayers. Li, Y., Fawcett, JP., Zhang, H., et al.[2018]

MitoQ, a mitochondria-targeted antioxidant, significantly reduced inflammation and oxidative stress in a mouse model of colitis, indicating its potential as a treatment for inflammatory bowel disease.

The study found that MitoQ decreased levels of harmful inflammatory cytokines IL-1 beta and IL-18, and suppressed the activation of the NLRP3 inflammasome, which plays a key role in inflammation, suggesting a mechanism for its therapeutic effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mitochondria-targeted antioxidant MitoQ ameliorates experimental mouse colitis by suppressing NLRP3 inflammasome-mediated inflammatory cytokines. Dashdorj, A., Jyothi, KR., Lim, S., et al.[2021]

A sensitive and accurate liquid chromatography/tandem mass spectrometry (LC/MS/MS) assay was developed to measure mitoquinone levels and its metabolites in rat plasma, demonstrating a linear calibration curve and a low limit of quantitation (0.5 ng/mL).

The pharmacokinetic study identified four metabolites of MitoQ10 in rat plasma, suggesting that MitoQ10 is effectively processed in the body, which is important for understanding its potential therapeutic effects in neurodegenerative diseases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Quantitation and metabolism of mitoquinone, a mitochondria-targeted antioxidant, in rat by liquid chromatography/tandem mass spectrometry. Li, Y., Zhang, H., Fawcett, JP., et al.[2018]

In a study using a humanized mouse model of chronic HIV infection, treatment with the mitochondrial antioxidant MitoQ alongside antiretroviral therapy for 90 days improved mitochondrial function, as indicated by higher ratios of mitochondrial to nuclear DNA in end organs.

This research provides promising evidence that MitoQ could be a potential treatment for mitochondrial dysfunction associated with HIV, which currently has no existing therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mitoquinone Mesylate and Mitochondrial DNA in End Organs in Humanized Mouse Model of Chronic Treated Human Immunodeficiency Virus Infection. Song, S., Satta, S., Sharma, MB., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04109820

Effect of MitoQ on Platelet Function and Reactive Oxygen ... Antioxidant therapies targeted to specific enzymes or compartments may be beneficial in sickle cell anemia (SCA). MitoQ, the most extensively studied ...

2. mitoq.com mitoq.com/pages/research?srsltid=AfmBOopLAHnNc65RzouWZXXIqzdWn7X5UZVGvc44ftMlMNbtnTXcs-yc

mitoquinol mesylate research studies & clinical trials After six weeks of supplementation, MitoQ was found to be 24% more effective than CoQ10 at reducing hydrogen peroxide levels in the mitochondria during states ...

3. withpower.com withpower.com/trial/mitoq-for-sickle-cell-disease-e427e

MitoQ for Sickle Cell Disease It aims to see if MitoQ can improve how platelets function and reduce harmful oxygen-related molecules in those with sickle cell disease.

4. sciencedirect.com sciencedirect.com/science/article/pii/S2667137922000091

Pharmacological significance of MitoQ in ameliorating ... MitoQ restored impaired mitophagosome-lysosome fusion and lysosomal function, as well as mitochondrial dynamics balance, resulting in the elimination of ...

5. trial.medpath.com trial.medpath.com/clinical-trial/a8a51cae9d7ee150/nct04109820-effect-mitoq-platelet-function-sickle-cell-anemia

Effect of MitoQ on Platelet Function and Reactive Oxygen ... MitoQ, the most extensively studied mitochondrial-targeted antioxidant, has been shown to be protective against ischemia/reperfusion injury in the heart, ...

6. mitoq.com mitoq.com/pages/research?srsltid=AfmBOoo8UJV0cZATJTpRu9NyLotVU33F-n0VIpa2pBoFB247kyGMCh03

mitoquinol mesylate research studies & clinical trials Results found that acute, high-dose MitoQ supplementation did not result in high concentrations of kidney injury biomarkers compared to placebo samples.

7. trialfinder.panfoundation.org trialfinder.panfoundation.org/en-US/trial/listing/239425

Effect of MitoQ on Platelet Function and Reactive Oxygen ... The goal of this research is to study if MitoQ, a molecule that works as an antioxidant by removing potentially damaging agents in a living ...