Sickle Cell DiseaseMitoQNCT04109820
Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
A study testing MitoQ in people with Sickle Cell Disease, run by University of Pittsburgh at 5 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- MitoQ
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 5 sitesUnited States
- Study dates
- 10 Mar 2020to 30 Jun 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Subjects
- African American
- Patients with sickle cell anemia
- 18 years old or older
- Control
- African American healthy controls
- 18 years of age or older
Who cannot join
- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Magee Women's Hospital
Pittsburgh, Pennsylvania, 15213
UPMC Montefiore
Pittsburgh, Pennsylvania, 15213
UPMC Presbyterian
Pittsburgh, Pennsylvania, 15213
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
About this study
Official title: Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) 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).
Read the 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.
- Conditions
- Sickle Cell Disease
- Being tested
- MitoQ
- Keywords
- Basal platelet activation, MitoQ, mitochondrial ROS (Reactive Oxygen Species)
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MitoQDietary supplement
Oral; 20mg once a day for 14 days
| Study group | Role | What they receive |
|---|---|---|
| Sickle cell patients | Experimental | Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days) Receives: Dietary Supplement: MitoQ |
| Non Sickle cell Control subjects | Active comparator | Normal control subjects administered oral MitoQ (20mg once a day for 14 days) Receives: Dietary Supplement: MitoQ |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: Baseline to 14 days
Secondary outcomes (5)
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
Measured: Baseline to 14 days
Effect of MitoQ on hemolysis in subjects with SCA
Changes in plasma free hemoglobin level (mg/dL) will be measured in blood.
Measured: Baseline to 14 days
Effect of MitoQ on hemolysis in subjects with SCA
Changes in plasma adenosine diphosphate level (micromole/liter) will be measured in blood.
Measured: Baseline to 14 days
Effect of MitoQ on hemolysis in subjects with SCA
Changes in serum lactate dehydrogenase level (units/L) will be measured in blood.
Measured: Baseline to 14 days
Treatment related severe adverse events (SAE)
Overall incidence of treatment emergent severe adverse events (SAE)
Measured: Baseline to 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2019
- Study start10 Mar 2020
- Primary completion27 Sep 2022
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Non randomized case control study design.
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ramasubramanian Kalpatthi, University of Pittsburgh
- Investigators
- Ramasubramanian Kalpatthi, MD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04109820
- Sponsor's protocol ID
- STUDY18120144
- Acronym
- MitoQ
- Status last verified
- Aug 2026
- First submitted
- 27 Aug 2019
- Last updated
- 2 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The investigators may share de-identified data with others who are doing similar types of research. All collected individual participant data (IPD), all IPD that underlie results in a publication will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT04109820
- Acronym / sponsor ID
- MitoQ / STUDY18120144
- Conditions
- Sickle Cell Disease
- Keywords
- Basal platelet activation, MitoQ, mitochondrial ROS (Reactive Oxygen Species)
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Study start
- 10 Mar 2020 (actual)
- Primary completion
- 27 Sep 2022 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 30 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Non randomized case control study design.
| Arm | Type | Receives |
|---|---|---|
| Sickle cell patients | Experimental | Dietary Supplement: MitoQSickle Cell subjects administered oral MitoQ (20mg once a day for 14 days) |
| Non Sickle cell Control subjects | Active comparator | Dietary Supplement: MitoQNormal control subjects administered oral MitoQ (20mg once a day for 14 days) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Subjects
- African American
- Patients with sickle cell anemia
- 18 years old or older
- Control
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MitoQOral; 20mg once a day for 14 days | Dietary supplement | Non Sickle cell Control subjects, Sickle cell patients |
Primary outcome
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)
5 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2026.
- Individual participant dataSharing planned: Yes. The investigators may share de-identified data with others who are doing similar types of research. All collected individual participant data (IPD), all IPD that underlie results in a publication will be shared.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ramasubramanian Kalpatthi, MD principal investigator
University of Pittsburgh
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing MitoQ in people with Sickle Cell Disease, run by University of Pittsburgh at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of MitoQ on platelet activation markers in subjects with SCA (time frame: Baseline to 14 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2026.