Ignite Creation Date:
2025-12-25 @ 4:17 AM
Ignite Modification Date:
2026-03-02 @ 10:24 PM
Study NCT ID:
NCT04109820
Status:
RECRUITING
Last Update Posted:
2025-06-19
First Post:
2019-08-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C429014', 'term': 'mitoquinone'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'OTHER', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Non randomized case control study design.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 15}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-03-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-06', 'completionDateStruct': {'date': '2027-06-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-06-16', 'studyFirstSubmitDate': '2019-08-27', 'studyFirstSubmitQcDate': '2019-09-27', 'lastUpdatePostDateStruct': {'date': '2025-06-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-09-30', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-12-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Effect of MitoQ on platelet activation markers in subjects with SCA', 'timeFrame': 'Baseline to 14 days', 'description': '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)'}], 'secondaryOutcomes': [{'measure': 'Effect of MitoQ on vascular dysfunction in subjects with SCA', 'timeFrame': 'Baseline to 14 days', 'description': 'Changes in both systolic and diastolic blood pressure will be measured during the study period'}, {'measure': 'Effect of MitoQ on hemolysis in subjects with SCA', 'timeFrame': 'Baseline to 14 days', 'description': 'Changes in plasma free hemoglobin level (mg/dL) will be measured in blood.'}, {'measure': 'Effect of MitoQ on hemolysis in subjects with SCA', 'timeFrame': 'Baseline to 14 days', 'description': 'Changes in plasma adenosine diphosphate level (micromole/liter) will be measured in blood.'}, {'measure': 'Effect of MitoQ on hemolysis in subjects with SCA', 'timeFrame': 'Baseline to 14 days', 'description': 'Changes in serum lactate dehydrogenase level (units/L) will be measured in blood.'}, {'measure': 'Treatment related severe adverse events (SAE)', 'timeFrame': 'Baseline to 14 days', 'description': 'Overall incidence of treatment emergent severe adverse events (SAE)'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Basal platelet activation', 'MitoQ', 'mitochondrial ROS (Reactive Oxygen Species)'], 'conditions': ['Sickle Cell Disease']}, 'descriptionModule': {'briefSummary': '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.\n\nThe 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).', 'detailedDescription': '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.\n\nInvestigators 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.\n\nAdult male and female SCA subjects in steady state (n=10) and 5 healthy African-American volunteers will be recruited after obtaining informed consent.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\nSubjects\n\n* African American\n* Patients with sickle cell anemia\n* 18 years old or older\n\nControl\n\n* African American healthy controls\n* 18 years of age or older\n\nExclusion Criteria:\n\n1. Pregnancy,\n2. Known hypertension,\n3. Hemodialysis and active obstructive sleep apnea requiring treatment.\n4. Use of anti-platelet medication or have had transfusion in the 4 weeks prior to enrollment.'}, 'identificationModule': {'nctId': 'NCT04109820', 'acronym': 'MitoQ', 'briefTitle': 'Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia', 'organization': {'class': 'OTHER', 'fullName': 'University of Pittsburgh'}, 'officialTitle': 'Effect of MitoQ on Platelet Function and Reactive Oxygen Species (ROS) Generation in Patients With Sickle Cell Anemia', 'orgStudyIdInfo': {'id': 'STUDY18120144'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Sickle cell patients', 'description': 'Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days)', 'interventionNames': ['Dietary Supplement: MitoQ']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Non Sickle cell Control subjects', 'description': 'Normal control subjects administered oral MitoQ (20mg once a day for 14 days)', 'interventionNames': ['Dietary Supplement: MitoQ']}], 'interventions': [{'name': 'MitoQ', 'type': 'DIETARY_SUPPLEMENT', 'description': 'Oral; 20mg once a day for 14 days', 'armGroupLabels': ['Non Sickle cell Control subjects', 'Sickle cell patients']}]}, 'contactsLocationsModule': {'locations': [{'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '412-692-2086'}, {'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Magee Women's Hospital", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '412-692-2086'}, {'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'UPMC Montefiore', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '15213', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '(412) 692-2086'}, {'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'UPMC Presbyterian', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '(412) 692-2086'}, {'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Children's Hospital of Pittsburgh", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '15232', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '412-692-2086'}, {'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hillman Cancer Center', 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}], 'centralContacts': [{'name': 'Mikhil N Bamne, PhD', 'role': 'CONTACT', 'email': 'bamnemn2@upmc.edu', 'phone': '(412) 648-6920'}, {'name': 'Jude Jonassaint, RN', 'role': 'CONTACT', 'email': 'jonassaintjc@upmc.edu', 'phone': '412-692-2086'}], 'overallOfficials': [{'name': 'Ramasubramanian Kalpatthi, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Pittsburgh'}]}, 'ipdSharingStatementModule': {'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'timeFrame': 'Data will be available 6 months after the publication. July 2022.', 'ipdSharing': 'YES', '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.', 'accessCriteria': '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.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Pittsburgh', 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Assistant Professor', 'investigatorFullName': 'Ramasubramanian Kalpatthi', 'investigatorAffiliation': 'University of Pittsburgh'}}}}