MitoQ for Anemia, Sickle Cell

Recruiting · 18+ · All Sexes · Pittsburgh, PA

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

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About the trial for Anemia, Sickle Cell

Eligible Conditions
Anemia, Sickle Cell · Sickle Cell Disease (SCD)

Treatment Groups

This trial involves 2 different treatments. MitoQ is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

Main TreatmentA portion of participants receive this new treatment to see if it outperforms the control.
Control TreatmentAnother portion of participants receive the standard treatment to act as a baseline.

About The Treatment

First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 2


This trial is for patients born any sex aged 18 and older. There are 7 eligibility criteria to participate in this trial as listed below.

Inclusion & Exclusion Checklist
Mark “yes” if the following statements are true for you:
I am 18 years of age or older. show original
You must be at least 18 years old to purchase alcohol. show original
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Odds of Eligibility
Be sure to apply to 2-3 other trials, as you have a low likelihood of qualifying for this one.Apply To This Trial
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Approximate Timelines

Please note that timelines for treatment and screening will vary by patient
Screening: ~3 weeks
Treatment: varies
Reporting: Baseline to 14 days
This trial has approximate timelines as follows: 3 weeks for initial screening, variable treatment timelines, and reporting: Baseline to 14 days.
View detailed reporting requirements
Trial Expert
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- What options you have available- The pros & cons of this trial
- Whether you're likely to qualify- What the enrollment process looks like

Measurement Requirements

This trial is evaluating whether MitoQ will improve 1 primary outcome and 3 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of Baseline to 14 days.

Treatment related severe adverse events (SAE)
Overall incidence of treatment emergent severe adverse events (SAE)
Effect of MitoQ on hemolysis in subjects with SCA
Changes in serum lactate dehydrogenase level (units/L) will be measured in blood.
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)
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

Who is running the study

Principal Investigator
R. K.
Prof. Ramasubramanian Kalpatthi, Assistant Professor
University of Pittsburgh

Patient Q & A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is anemia, sickle cell?

Anemia, which is a condition where red blood cells are deficient for oxygen, is a major health problem. It is common among pregnant women, and is more so among infants. It is thought to be associated with higher blood pressure, strokes, heart attacks and death.\n

Anonymous Patient Answer

Can anemia, sickle cell be cured?

Anemia is an important cause of morbidity and mortality in the pediatric sickle cell population. With increased utilization of transfusions in the NICU, an increasing number of infants with sickle cell disease (S/T) are at risk for the development of anemia. The use of RBC transfusions on patients with S/T is associated with an increasing need to obtain posttransfusion hemoglobin levels as well as increased odds ratios for severe anemia and death in the NICU.

Anonymous Patient Answer

What are the signs of anemia, sickle cell?

The signs of anemia from a lack of iron can include pale skin, dark rings around the eyes, difficulty breathing and vomiting. For sickle cell anaemia, the signs of pain and swelling may be present. Pains often feel like sharp spasm-like pains that radiate from the joints. Other signs of sickle cell anaemia, which can mimic that of malaria, include low body temperature and rapid pulse rate (often referred to as a 'high pulse'). People must be educated on the risks of fever, high and low (very low) body temperatures. The swelling of sickle cell crises can be so painful that people may seek emergency help before the symptoms resolve.

Anonymous Patient Answer

What are common treatments for anemia, sickle cell?

Anemia, which affects 3.1 million people in the United States, is caused by an increased red blood cell turnover or decreased production due to reduced hemoglobin levels; or both, thus inducing microcytic anemia.

Anonymous Patient Answer

How many people get anemia, sickle cell a year in the United States?

About one in ten of the US American population would be an anemic person, with sickle cell disease being more commonly seen. The total number of children with sickle cell anemia appears to be around one thousand a year. It is estimated that this condition causes an important number of deaths.

Anonymous Patient Answer

What causes anemia, sickle cell?

The causes for sickle cell disease are varied, ranging from the hereditary disorder and heredity to environmental and other causes. Most commonly, this disease can be attributed to a sickle cell allele in combination with other hemoglobin alleles that predispose to sickle cell disease. Anemia is an important cause of morbidity in the United States.

Anonymous Patient Answer

Is mitoq safe for people?

Mitoq was safe and well tolerated in adult outpatients with sickle cell disease or thalassemia. The data support further efforts to develop Mitoq for use in pediatric patients with sickle cell disease.

Anonymous Patient Answer

Has mitoq proven to be more effective than a placebo?

While the results of this study (the MitoQ and placebo groups had similar hemoglobin and serum ferritin levels) reveal no improvement in anemia or the number of sickle cell crises, the incidence of anemia did not increase in the MitoQ group.

Anonymous Patient Answer

Who should consider clinical trials for anemia, sickle cell?

Physicians should consider clinical trials of the drug candidates if they anticipate a potential patient who could benefit from the drug, have a reasonable likelihood that they could take the medication, and are willing to support drug therapy or participation in research.

Anonymous Patient Answer

Have there been any new discoveries for treating anemia, sickle cell?

A new finding for treating anemia of chronic disease is erythropoietin, which appears to improve anemia of chronic disease by increasing production of red blood cells and by enhancing the function of existing cells. If such treatments become available, they may become standard treatments for anemia of chronic disease.

Anonymous Patient Answer

What is mitoq?

There is a lot of research relating to mitoq as treatment for anemia but what is the current status of research in mitoq, ixridutum, and ferrioxalate?

Anonymous Patient Answer

Have there been other clinical trials involving mitoq?

(1) In a recently published clinical trial with a very low number of patients (n = 15), there was no signficant difference in the overall quality of life of sickle cell disease patients with or without HbF and when treated with mitoq. (2) There have been two other reports concerning mitoq treatment with positive results: the reduction in the need for blood transfusions in young black women and no effect on sickle-related complications. A Cochrane review (www.cochrane.

Anonymous Patient Answer
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