Effect of Deferiprone on Oxidative-Stress and Iron-Overload in Low Risk Transfusion-Dependent MDS… (NCT02477631) | Clinical Trial Compass
CompletedPhase 2
Effect of Deferiprone on Oxidative-Stress and Iron-Overload in Low Risk Transfusion-Dependent MDS Patients
Israel19 participantsStarted 2016-02
Plain-language summary
The effect oral iron chelator Deferiprone on the Oxidative stress and on Iron Overload status in transfusion dependent, iron-overloaded low risk MDS patients;
Primary Objective:
• To evaluate the effect of Deferiprone on oxidative stress parameter - Reactive oxygen species (ROS).
Secondary Objectives:
* To evaluate the effect of Deferiprone on other oxidative stress parameters
1. Reduced glutathione
2. Membrane lipid peroxidation
3. External phosphatidylserine
* To evaluate the change from baseline to last visit in parameters of iron load.
1. Serum ferritin (despite ongoing RBC transfusions during the study period).
2. LIP
3. LPI
4. serum hepcidin
* To evaluate the change from one month preceding baseline visit to last month on study in transfusion requirements.
* To monitor safety measures:
1. Adverse events (AEs).
2. Number of discontinuations due to AEs
Study design:
Single-arm, open-label, multi-center study in 20 iron-overloaded patients with low risk MDS. All participants will be treated with deferiprone for up to 4 months. Patients will have complete blood count monitored weekly, and will visit the site monthly for assessments of safety and efficacy.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female aged ≥ 18 years
. Have a documented diagnosis of MDS according to WHO 2008 classification (see appendix I), with an International Prognostic Scoring System (IPSS-R) (see Appendix II) of very low, low or intermediate risk.
. Life expectancy of at least 1 year
. Serum ferritin level \> 1000 ng/mL
. Prior receipt of ≥20 RBC units
. Females of childbearing potential must have a negative pregnancy test result month prior to start of dosing, In addition, if applicable, they must:
. Non-sterilized heterosexual males and/or their partners must agree to use an effective method of contraception during the study and for 30 days following their last dose of study medication
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To evaluate the effect of deferiprone on oxidative stress parameter ROS in iron overloaded and blood dependent patients with MDS.
. All patients and/or their authorized legal representatives must provide signed and dated written informed consent prior to the first study intervention, and patients must be able to adhere to study restrictions, appointments, and evaluation schedules
Exclusion criteria
. IPSS-R prognosis of high and very high risk (to avoid the confounding influence of a high blast count)
. Unable or unwilling to undergo a 7-day washout period if currently being treated with deferoxamine or deferasirox
. Evidence of abnormal liver function (serum ALT level \> 5 times upper limit of normal or creatinine level \>2 times upper limit of normal)
. A serious, unstable illness, as judged by the investigator, during the past 3 months before screening, including but not limited to: hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic, or immunologic disease
. Myocardial infarction, cardiac arrest, or cardiac failure within 1 year before screening
. QT interval prolongation on ECG
. Occurrences of severe neutropenia/agranulocytosis (absolute neutrophil count \< 0.5 x 109/L
. History of allergy or sensitivity to deferiprone or related compounds or to other components of the formulation