NCT04109820

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).

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for not_applicable

Timeline
14mo left

Started Mar 2020

Longer than P75 for not_applicable

Geographic Reach
1 country

5 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress84%
Mar 2020Jun 2027

First Submitted

Initial submission to the registry

August 27, 2019

Completed
1 month until next milestone

First Posted

Study publicly available on registry

September 30, 2019

Completed
5 months until next milestone

Study Start

First participant enrolled

March 1, 2020

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2026

Expected
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2027

Last Updated

June 19, 2025

Status Verified

June 1, 2025

Enrollment Period

6.8 years

First QC Date

August 27, 2019

Last Update Submit

June 16, 2025

Conditions

Sickle Cell Disease

Keywords

Basal platelet activationMitoQmitochondrial ROS (Reactive Oxygen Species)

Outcome Measures

Primary Outcomes (1)

  • 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)

    Baseline to 14 days

Secondary Outcomes (5)

  • Effect of MitoQ on vascular dysfunction in subjects with SCA

    Baseline to 14 days

  • Effect of MitoQ on hemolysis in subjects with SCA

    Baseline to 14 days

  • Effect of MitoQ on hemolysis in subjects with SCA

    Baseline to 14 days

  • Effect of MitoQ on hemolysis in subjects with SCA

    Baseline to 14 days

  • Treatment related severe adverse events (SAE)

    Baseline to 14 days

Study Arms (2)

Sickle cell patients

EXPERIMENTAL

Sickle Cell subjects administered oral MitoQ (20mg once a day for 14 days)

Dietary Supplement: MitoQ

Non Sickle cell Control subjects

ACTIVE COMPARATOR

Normal control subjects administered oral MitoQ (20mg once a day for 14 days)

Dietary Supplement: MitoQ

Interventions

MitoQDIETARY_SUPPLEMENT

Oral; 20mg once a day for 14 days

Non Sickle cell Control subjectsSickle cell patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Subjects
  • African American
  • Patients with sickle cell anemia
  • years old or older
  • Control
  • African American healthy controls
  • years of age or older

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

Magee Women's Hospital

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

UPMC Montefiore

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

UPMC Presbyterian

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Hillman Cancer Center

Pittsburgh, Pennsylvania, 15232, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

mitoquinone

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Ramasubramanian Kalpatthi, MD

    University of Pittsburgh

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Mikhil N Bamne, PhD

CONTACT

(412) 648-6920bamnemn2@upmc.edu

Jude Jonassaint, RN

CONTACT

412-692-2086jonassaintjc@upmc.edu

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
PARALLEL
Model Details: Non randomized case control study design.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

August 27, 2019

First Posted

September 30, 2019

Study Start

March 1, 2020

Primary Completion (Estimated)

December 30, 2026

Study Completion (Estimated)

June 30, 2027

Last Updated

June 19, 2025

Record last verified: 2025-06

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, 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.

Locations

US(5)