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

University of Pittsburgh

Status: Enrolling

Conditions: Sickle Cell Disease

Treatments: Dietary Supplement: MitoQ

Study type: Interventional

Funder types: Other

Identifiers

NCT04109820

STUDY18120144

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

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

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

Trial design

Primary purpose: Other

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

Sickle cell patients

Experimental group

Description:

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

Treatment:

Dietary Supplement: MitoQ

Non Sickle cell Control subjects

Active Comparator group

Description:

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

Treatment:

Dietary Supplement: MitoQ