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

Sickle Cell Disease Clinical Trial

— MitoQ  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04109820
• Other study ID #: STUDY18120144
• Status: Recruiting
• Phase: N/A
• Start date: March 1, 2020
• Est. completion date: June 30, 2026

Study information

• Verified date: June 2024
• Source: University of Pittsburgh
• Contact: Mikhil N Bamne, PhD
• Phone: (412) 648-6920
• Email: bamnemn2[@]upmc.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: June 30, 2026
• Est. primary completion date: December 30, 2025
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Dietary Supplement:
• MitoQ
Oral; 20mg once a day for 14 days

Locations

Country	Name	City	State
United States	Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Hillman Cancer Center	Pittsburgh	Pennsylvania
United States	Magee Women's Hospital	Pittsburgh	Pennsylvania
United States	UPMC Montefiore	Pittsburgh	Pennsylvania
United States	UPMC Presbyterian	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
University of Pittsburgh

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effect of MitoQ on platelet activation markers in subjects with SCA	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)	Baseline to 14 days
Secondary	Effect of MitoQ on vascular dysfunction in subjects with SCA	Changes in both systolic and diastolic blood pressure will be measured during the study period	Baseline to 14 days
Secondary	Effect of MitoQ on hemolysis in subjects with SCA	Changes in plasma free hemoglobin level (mg/dL) will be measured in blood.	Baseline to 14 days
Secondary	Effect of MitoQ on hemolysis in subjects with SCA	Changes in plasma adenosine diphosphate level (micromole/liter) will be measured in blood.	Baseline to 14 days
Secondary	Effect of MitoQ on hemolysis in subjects with SCA	Changes in serum lactate dehydrogenase level (units/L) will be measured in blood.	Baseline to 14 days
Secondary	Treatment related severe adverse events (SAE)	Overall incidence of treatment emergent severe adverse events (SAE)	Baseline to 14 days