Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: MitoQ.
- Who it may be relevant to
- Registry conditions: Sickle Cell Disease. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia
Overview
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).
Detailed 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.
Interventions
- Dietary supplement MitoQ
Oral; 20mg once a day for 14 days
Primary outcome measures
- Effect of MitoQ on platelet activation markers in subjects with SCA [Time frame: Baseline to 14 days]
Secondary outcome measures (5)
- Effect of MitoQ on vascular dysfunction in subjects with SCA [Time frame: Baseline to 14 days]
- Effect of MitoQ on hemolysis in subjects with SCA [Time frame: Baseline to 14 days]
- Effect of MitoQ on hemolysis in subjects with SCA [Time frame: Baseline to 14 days]
- Effect of MitoQ on hemolysis in subjects with SCA [Time frame: Baseline to 14 days]
- Treatment related severe adverse events (SAE) [Time frame: Baseline to 14 days]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- Non-randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Other
Study locations
United States · 5 centers
- Magee Women's Hospital — Pittsburgh
- UPMC Montefiore — Pittsburgh
- UPMC Presbyterian — Pittsburgh
- Children's Hospital of Pittsburgh — Pittsburgh
- Hillman Cancer Center — Pittsburgh
Identifiers
NCT: NCT04109820 · STUDY18120144