Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04109820
Recruitment Status : Not yet recruiting
First Posted : September 30, 2019
Last Update Posted : September 30, 2019
Sponsor:
Information provided by (Responsible Party):
Ramasubramanian Kalpatthi, University of Pittsburgh

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).


Condition or disease Intervention/treatment Phase
Sickle Cell Disease Dietary Supplement: MitoQ Not Applicable

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Non randomized case control study design.
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia
Estimated Study Start Date : October 1, 2019
Estimated Primary Completion Date : September 30, 2020
Estimated Study Completion Date : December 31, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Sickle cell patients
Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days)
Dietary Supplement: MitoQ
Oral; 20mg once a day for 14 days

Active Comparator: Non Sickle cell Control subjects
Normal control subjects administered oral MitoQ (20mg once a day for 14 days)
Dietary Supplement: MitoQ
Oral; 20mg once a day for 14 days




Primary Outcome Measures :
  1. 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)


Secondary Outcome Measures :
  1. 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

  2. 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.

  3. 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.

  4. 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.

  5. Treatment related severe adverse events (SAE) [ Time Frame: Baseline to 14 days ]
    Overall incidence of treatment emergent severe adverse events (SAE)



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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:

  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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04109820


Contacts
Layout table for location contacts
Contact: Mikhil N Bamne, PhD (412) 648-6920 bamnemn2@upmc.edu
Contact: Jude Jonassaint, RN 412-692-2086 jonassaintjc@upmc.edu

Locations
Layout table for location information
United States, Pennsylvania
Magee Women's Hospital
Pittsburgh, Pennsylvania, United States, 15213
Contact: Mikhil N Bamne, PhD    412-648-6920    bamnemn2@upmc.edu   
Contact: Jude Jonassaint, RN    412-692-2086    jonassaintjc@upmc.edu   
Principal Investigator: Ramasubramanian Kalpatthi, MD         
UPMC Montefiore
Pittsburgh, Pennsylvania, United States, 15213
Contact: Mikhil N Bamne, PhD    412-648-6920    bamnemn2@upmc.edu   
Contact: Jude Jonassaint, RN    412-692-2086    jonassaintjc@upmc.edu   
Principal Investigator: Ramasubramanian Kalpatthi, MD         
UPMC Presbyterian
Pittsburgh, Pennsylvania, United States, 15213
Contact: Mikhil N Bamne, PhD    412-648-6920    bamnemn2@upmc.edu   
Contact: Jude Jonassaint, RN    (412) 692-2086    jonassaintjc@upmc.edu   
Principal Investigator: Ramasubramanian Kalpatthi, MD         
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Contact: Mikhil N Bamne, PhD    412-648-6920    bamnemn2@upmc.edu   
Contact: Jude Jonassaint, RN    (412) 692-2086    jonassaintjc@upmc.edu   
Principal Investigator: Ramasubramanian Kalpatthi, MD         
Hillman Cancer Center
Pittsburgh, Pennsylvania, United States, 15232
Contact: Mikhil N Bamne, PhD    412-648-6920    bamnemn2@upmc.edu   
Contact: Jude Jonassaint, RN    412-692-2086    jonassaintjc@upmc.edu   
Principal Investigator: Ramasubramanian Kalpatthi, MD         
Sponsors and Collaborators
University of Pittsburgh
Investigators
Layout table for investigator information
Principal Investigator: Ramasubramanian Kalpatthi, MD University of Pittsburgh

Layout table for additonal information
Responsible Party: Ramasubramanian Kalpatthi, Assistant Professor, University of Pittsburgh
ClinicalTrials.gov Identifier: NCT04109820    
Other Study ID Numbers: STUDY18120144
First Posted: September 30, 2019    Key Record Dates
Last Update Posted: September 30, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
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
Statistical Analysis Plan (SAP)
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.

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ramasubramanian Kalpatthi, University of Pittsburgh:
Basal platelet activation
MitoQ
mitochondrial ROS (Reactive Oxygen Species)
Additional relevant MeSH terms:
Layout table for MeSH terms
Anemia, Sickle Cell
Anemia
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn