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NCT Number: NCT04109820

Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia

MitoQ is commercially available as a dietary supplement and it has been tested as a potential drug in other diseases, but it has never been tested in patients with sickle cell disease.

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 organism, improves platelet function in patients with sickle cell disease (SCD).

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States

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About this study

Antioxidant therapies targeted to specific enzymes or compartments may be beneficial in sickle cell anemia (SCA). MitoQ, the most extensively studied mitochondrial-targeted antioxidant, has been shown to be protective against ischemia/reperfusion injury in the heart, endothelial damage due to hypertension and ROS in animal models. MitoQ is commercially available as a dietary supplement to reduce overall oxidative stress and anti-ageing. However, MitoQ has not been tested either as a platelet antagonist or as an endothelial protectant in SCA patients. Investigators propose to conduct a small clinical trial of MitoQ in subjects with SCA to test the hypothesis that MitoQ scavenges platelet mtROS to prevent platelet activation and attenuate vascular dysfunction in SCA.

Investigators will test whether MitoQ decreases basal platelet activation in SCD patients and attenuates vascular dysfunction in subjects with SCA. Investigators will administer MitoQ orally to patients and healthy controls for 14 days. Investigators will obtain platelet count, hemolytic markers, platelet mtROS levels and activation markers, clinic BP measurements before and after MitoQ.

Adult male and female SCA subjects in steady state (n=10) and 5 healthy African-American volunteers will be recruited after obtaining informed consent.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Subjects

  • African American
  • Patients with sickle cell anemia
  • 18 years old or older

Control

  • African American healthy controls
  • 18 years of age or older

Exclusion criteria

  • Pregnancy,
  • Known hypertension,
  • Hemodialysis and active obstructive sleep apnea requiring treatment.
  • Use of anti-platelet medication or have had transfusion in the 4 weeks prior to enrollment.

Treatment and study plan

MitoQ

Dietary Supplement

Oral; 20mg once a day for 14 days

Primary outcomes

  1. Effect of MitoQ on platelet activation markers in subjects with SCA

    Time frame: Baseline to 14 days

    Change in the percentage of platelet activation markers in blood will be measured (p-selectin, activated GpIIb/IIIa expression, platelet mtROS [mitochondrial reactive oxygen species], platelet bioenergetics, mitochondrial Complex V activity)

Secondary outcomes

  1. Effect of MitoQ on vascular dysfunction in subjects with SCA

    Time frame: Baseline to 14 days

    Changes in both systolic and diastolic blood pressure will be measured during the study period

  2. Effect of MitoQ on hemolysis in subjects with SCA

    Time frame: Baseline to 14 days

    Changes in plasma free hemoglobin level (mg/dL) will be measured in blood.

  3. Effect of MitoQ on hemolysis in subjects with SCA

    Time frame: Baseline to 14 days

    Changes in plasma adenosine diphosphate level (micromole/liter) will be measured in blood.

  4. Effect of MitoQ on hemolysis in subjects with SCA

    Time frame: Baseline to 14 days

    Changes in serum lactate dehydrogenase level (units/L) will be measured in blood.

  5. Treatment related severe adverse events (SAE)

    Time frame: Baseline to 14 days

    Overall incidence of treatment emergent severe adverse events (SAE)

Study contacts

Contact information is provided by the study sponsor or research team.

Jude Jonassaint, RN

CONTACT

[email protected]

412-692-2086

Mikhil N Bamne, PhD

CONTACT

[email protected]

(412) 648-6920

Sponsors and collaborators

Lead sponsor

University of Pittsburgh

Other

Registry information

Official study title

Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia

Acronym: MitoQ

Important dates

Study start
2020
Primary completion
2026
Study completion
2027
First posted
Sep 30, 2019
Registry last updated
Jun 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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