Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias (FIRST)
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|ClinicalTrials.gov Identifier: NCT02041299|
Recruitment Status : Recruiting
First Posted : January 22, 2014
Last Update Posted : January 14, 2019
|Condition or disease||Intervention/treatment||Phase|
|Iron Overload Sickle Cell Disease Other Anemias||Drug: Deferiprone Drug: Deferoxamine||Phase 4|
Deferiprone (brand name Ferriprox®) is an iron chelator that is approved in the United States and over 60 other countries for the treatment of iron overload in patients with thalassemia, when other treatments are inadequate. This study has been designed to evaluate the efficacy, safety, and tolerability of deferiprone vs. deferoxamine in patients who have SCD or other anemias, and who require chelation because of the extra iron they are taking in through blood transfusions.
About 300 people from North America, South America, Europe, and the Middle East will take part in this study. Participants will be randomized in a 2:1 ratio to receive therapy for 52 weeks with either deferiprone or deferoxamine, another type of iron chelator. Patients who are randomized to the deferiprone group can choose to get the drug as either tablets or liquid, and must take it three times daily. Patients who are randomized to the deferoxamine group will receive it as a subcutaneous infusion that lasts from 8 to 12 hours and is given 5 to 7 days per week. For both drugs, the starting dosage is based on how much extra iron they have taken in through transfusions in the last 3 months and on the severity of iron load, as measured by serum ferritin levels in the blood and by the amount of iron in the liver and the heart. For deferiprone, the starting dosage will be increased each week over the first 3 weeks; and for both drugs, the dosage may be adjusted up or down during the study based on the level of iron overload and on safety considerations.
Patients will need to have their blood count checked every week for the first 26 weeks, then every other week for the remaining 26 weeks; they will also have to give a blood sample for more detailed safety testing every month; and to give a blood sample for the measurement of serum ferritin every 3 months. Every six months, they will undergo an ECG and an MRI scan, and will be asked to complete a quality of life survey.
At the end of the 52 weeks, participants will be invited to enter a 2-year study in which all patients will receive deferiprone, including those who were randomized to receive deferoxamine in the first year.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||300 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||The Efficacy and Safety of Ferriprox® for the Treatment of Transfusional Iron Overload in Patients With Sickle Cell Disease or Other Anemias|
|Study Start Date :||March 2014|
|Estimated Primary Completion Date :||October 2019|
|Estimated Study Completion Date :||October 2019|
Patients randomized to the deferiprone arm will be prescribed either tablets or liquid medication.
Active Comparator: Deferoxamine
Patients randomized to the deferoxamine arm will be prescribed the drug as per the approved US prescribing information.
- Change in liver iron concentration, as measured in mg/g dry weight (dw) using MRI [ Time Frame: Change from baseline to Week 52 ]Without effective iron chelation therapy, transfusion-dependent patients experience a progressive increase in Liver Iron Concentration (LIC). High LIC increases the risk of iron-induced toxicity such as cardiac disease, hepatic fibrosis, diabetes mellitus, and death.
- Change in patient-reported quality of life, as measured by SF-36 or Child Health Questionnaire [ Time Frame: Change from baseline to Week 52 ]Adults patients will complete the SF-36 questionnaire and minors will complete the CHQ, in order to provide a profile of functional health and well-being.
- Change in cardiac MRI T2*, measured in milliseconds (ms) [ Time Frame: Change from baseline to Week 52 ]MRI T2* provides a measure of iron levels in the heart
- Change in serum ferritin, measured in mcg/L [ Time Frame: Change from baseline to Week 52 ]Serum ferritin provides a measure of iron level in the blood
- Occurrence of adverse events [ Time Frame: From first dose (Baseline visit) to last study visit (Week 52 or early termination) ]Number of participants in each group with AEs, by frequency, nature, severity, time to onset, and duration of AEs
- Frequency of serious adverse events (SAEs) [ Time Frame: From first dose (Baseline visit) to last study visit (Week 52 or early termination) ]Number of participants in each group with SAEs
- Hematology assessments [ Time Frame: Assessed at baseline, weekly (till week 26), biweekly (after week 26), monthly and at Weeks 12, 26, 40, and 52 (or early termination) ]Change from baseline at each visit and change from baseline at end of study for hemoglobin, total WBC, ANC, and platelets
- Blood clinical biochemistry assessments [ Time Frame: Assessed at baseline, monthly and Weeks 12, 26, 40, and 52 (or early termination) ]Change from baseline at each visit and change from baseline at end of study for total protein; GGT; lactate dehydrogenase (LDH); sodium, potassium, chloride, glucose (fasting at screening visit only); total, direct and indirect bilirubin; AST; ALT; albumin; blood urea nitrogen; calcium; creatinine; uric acid; alkaline phosphatase; and amylase
- Abnormal and clinically significant findings in 12-lead ECG [ Time Frame: Screening, Week 26, and end of study (Week 52 or early termination) visits ]Change in abnormal or clinically significant ECG parameters from screening to each later time point
- Discontinuations due to AEs [ Time Frame: From first dose (Baseline visit) to last study visit (Week 52 or early termination) ]Number of participants in each group who discontinued from the study due to AEs
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02041299
|Contact: Caroline Fradette, PhDfirstname.lastname@example.org|
|Contact: Fernando Tricta, MDemail@example.com|
|Principal Investigator:||Janet Kwiatkowski, MD||Children's Hospital of Philadelphia|