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 range18 Years
SexALL
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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
✓. 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
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.
✕. 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