Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias
Information source: ApoPharma
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Iron Overload; Sickle Cell Disease; Other Anemias
Intervention: Deferiprone (Drug); Deferoxamine (Drug)
Phase: Phase 4
Status: Recruiting
Sponsored by: ApoPharma Official(s) and/or principal investigator(s): Janet Kwiatkowski, MD, Principal Investigator, Affiliation: Children's Hospital of Philadelphia
Overall contact: Caroline Fradette, PhD, Phone: 416-401-7543, Email: cfradett@apopharma.com
Summary
This research is being done so that we can look at the safety and efficacy of deferiprone
in people with sickle cell disease or other anemias. Deferiprone is a drug that removes
iron from the body. We will be comparing deferiprone with deferoxamine, another drug that
removes iron from the body.
Clinical Details
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 design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Change in liver iron concentration, as measured in mg/g dry weight (dw) using MRI
Secondary outcome: Change in patient-reported quality of life, as measured by SF-36 or Child Health QuestionnaireChange in cardiac MRI T2*, measured in milliseconds (ms) Change in serum ferritin, measured in mcg/L Occurrence of adverse events Frequency of serious adverse events (SAEs) Hematology assessments Blood clinical biochemistry assessments Abnormal and clinically significant findings in 12-lead ECG Discontinuations due to AEs
Detailed description:
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; 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.
Eligibility
Minimum age: 2 Years.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
1. Male or female ≥ 2 years of age;
2. Have sickle cell disease (confirmed by Hb electrophoresis or more specific tests) or
other conditions with iron overload from repeated blood transfusions (see exclusion
criteria for exceptions);
3. Baseline LIC >7 mg/g dw (measured by MRI);
4. Patients who have received no less than 20 transfusions of RBCs;
5. Patients who have received at least 1 transfusion per year in the last 2 years and
who are expected to have a continuing requirement (based on Investigator's judgement)
during the duration of the trial
Exclusion Criteria:
1. Thalassemia syndromes;
2. Myelodysplastic syndrome (MDS) or myelofibrosis;
3. Diamond Blackfan anemia;
4. Primary bone marrow failure;
5. Baseline LIC >30 mg/g dw (measured by MRI);
6. Unable or unwilling to undergo a 7 day washout period if currently being treated with
deferiprone or deferoxamine or deferasirox;
7. Previous discontinuation of treatment with deferiprone or deferoxamine due to adverse
events;
8. History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or
deferoxamine;
9. Treated with hydroxyurea within 30 days;
10. History of malignancy;
11. Evidence of abnormal liver function (serum ALT level(s) > 5 times upper limit of
normal at screening or creatinine levels >2 times upper limit of normal at
screening);
12. A serious, unstable illness, as judged by the Investigator, during the past 3 months
before screening/baseline visit including but not limited to: hepatic, renal,
gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or
immunologic disease;
13. Clinically significant abnormal 12-lead ECG findings;
14. Cardiac MRI T2* <10ms;
15. Myocardial infarction, cardiac arrest or cardiac failure within 1 year before
screening/baseline visit;
16. Unable to undergo MRI
17. Presence of metallic objects such as artificial joints, inner ear (cochlear)
implants, brain aneurysm clips, pacemakers, and metallic foreign bodies in the eye or
other body areas that would prevent use of MRI imaging
Locations and Contacts
Caroline Fradette, PhD, Phone: 416-401-7543, Email: cfradett@apopharma.com
Centro Infantil Boldrini, Campinas, Brazil; Recruiting Monica Verissimo, Principal Investigator
Hospital de ClÃnicas de Porto Alegre, Porto Alegre, Brazil; Recruiting Christina Bittar, Principal Investigator
Instituto Estadual de Hematologia, Rio de Janeiro, Brazil; Recruiting Clarisse Lobo, Principal Investigator
Casa de Saúde Santa Marcelina, São Paulo, Brazil; Recruiting Sandra Calegare, Principal Investigator
Universidade Federal de São Paulo, São Paulo, Brazil; Recruiting Maria Stella Figueiredo, Principal Investigator
Zagazig University, Alexandria, Egypt; Recruiting Mohammed Badr, Principal Investigator
Zagazig University, Alexandria, Egypt; Recruiting Mohammed Elsafy, Principal Investigator
Ains Shams University, Cairo, Egypt; Recruiting Mohsen Saleh Elalfy, Principal Investigator
Cairo University, Cairo, Egypt; Recruiting Mona Hamdy, Principal Investigator
Pediatric Hospital of Cairo University, Cairo, Egypt; Recruiting Amal El Beshlawy, Principal Investigator
Mansoura University Children's Hospital, Mansoura, Egypt; Recruiting Ahmed Mansour, Principal Investigator
Asser Central Hospital, Abha, Saudi Arabia; Not yet recruiting Abdulrahman Alshehri, Principal Investigator
King Abdulaziz University Hospital, Jeddah, Saudi Arabia; Recruiting Mohammed Hassan Qari, Principal Investigator
King Khalid University Hospital, Riyadh, Saudi Arabia; Recruiting Abdulkareem Almomen, Principal Investigator
Barts and The London, London, United Kingdom; Recruiting Banu Kaya, Principal Investigator
Evelina Children's Hospital, London, United Kingdom; Recruiting Baba Inusa, Principal Investigator
Imperial College Healthcare NHS Trust, London, United Kingdom; Recruiting Subarna Chakravorty, Principal Investigator Mark Layton, Sub-Investigator
St. Mary's Hospital and Hammersmith Hospital, London, United Kingdom; Recruiting Sadaqat Luqmani, Principal Investigator
Children's Hospital Oakland, Oakland, California, United States; Recruiting Elliott Vichinsky, Principal Investigator
University of Illinois at Chicago, Chicago, Illinois 60612, United States; Recruiting Victor Gordeuk, Principal Investigator
Children's Hospital, New Orleans, Louisiana 70118, United States; Recruiting Renee Gardner, Principal Investigator
University of Michigan Comprehensive Cancer Center, Ann Arbor, Michigan, United States; Recruiting Andrew Campbell, Principal Investigator
Children's Hospital of Michigan, Detroit, Michigan 48201, United States; Recruiting Sharada Sarnaik, Principal Investigator
Steven and Alexandra Cohen Children's Medical Center of New York, New Hyde Park, New York, United States; Withdrawn
Hospital for Sick Kids, Toronto, Ontario, Canada; Recruiting Suzan Williams, Principal Investigator
The Children's Hospital of Philadephia, Philadelphia, Pennsylvania, United States; Recruiting Janet Kwiatkowski, Principal Investigator
Thomas Jefferson University, Philadelphia, Pennsylvania, United States; Terminated
Medical University of South Carolina, Charleston, South Carolina, United States; Recruiting Julie Kantor, Principal Investigator
Additional Information
Starting date: March 2014
Last updated: August 17, 2015
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