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

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ClinicalTrials.gov Identifier: NCT04109820
Recruitment Status : Recruiting
First Posted : September 30, 2019
Last Update Posted : December 4, 2020
Sponsor:
Information provided by (Responsible Party):
Ramasubramanian Kalpatthi, University of Pittsburgh

Tracking Information
First Submitted Date  ICMJE August 27, 2019
First Posted Date  ICMJE September 30, 2019
Last Update Posted Date December 4, 2020
Actual Study Start Date  ICMJE March 1, 2020
Estimated Primary Completion Date September 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 27, 2019)
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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 27, 2019)
  • 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
  • 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.
  • 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.
  • 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.
  • Treatment related severe adverse events (SAE) [ Time Frame: Baseline to 14 days ]
    Overall incidence of treatment emergent severe adverse events (SAE)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
Official Title  ICMJE Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia
Brief 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).

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 Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Non randomized case control study design.
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Dietary Supplement: MitoQ
Oral; 20mg once a day for 14 days
Study Arms  ICMJE
  • Experimental: Sickle cell patients
    Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days)
    Intervention: Dietary Supplement: MitoQ
  • Active Comparator: Non Sickle cell Control subjects
    Normal control subjects administered oral MitoQ (20mg once a day for 14 days)
    Intervention: Dietary Supplement: MitoQ
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 27, 2019)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 31, 2023
Estimated Primary Completion Date September 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Mikhil N Bamne, PhD (412) 648-6920 bamnemn2@upmc.edu
Contact: Jude Jonassaint, RN 412-692-2086 jonassaintjc@upmc.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04109820
Other Study ID Numbers  ICMJE STUDY18120144
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: 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.
Supporting Materials: Study Protocol
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Data will be available 6 months after the publication. July 2022.
Access Criteria: The IPD and any additional supporting information will be shared, with other investigators/collaborators when requested. The Principal Investigator will review the requests and will provide the instructions to the research site staff to share the IPD with other investigators.
Responsible Party Ramasubramanian Kalpatthi, University of Pittsburgh
Study Sponsor  ICMJE University of Pittsburgh
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Ramasubramanian Kalpatthi, MD University of Pittsburgh
PRS Account University of Pittsburgh
Verification Date December 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP