Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias

Stopped early · Phase 4

Conditions studied: Iron Overload, Sickle Cell Disease, Other Anemias

In brief

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.

Key facts

Study ID

NCT02041299

Run by

ApoPharma

People needed

230

Starts

2014-04-17

Expected to finish

2019-06-18

Last updated by the study team

2021-08-10

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female ≥ 2 years of age;
• 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);
• Baseline LIC >7 mg/g dw (measured by MRI);
• Patients who have received no less than 20 transfusions of RBCs;
• 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

You may not qualify if…

• Thalassemia syndromes;
• Myelodysplastic syndrome (MDS) or myelofibrosis;
• Diamond Blackfan anemia;
• Primary bone marrow failure;
• Baseline LIC >30 mg/g dw (measured by MRI);
• Unable or unwilling to undergo a 7 day washout period if currently being treated with deferiprone or deferoxamine or deferasirox;
• Previous discontinuation of treatment with deferiprone or deferoxamine due to adverse events;
• History or presence of hypersensitivity or idiosyncratic reaction to deferiprone or deferoxamine;
• Treated with hydroxyurea within 30 days;
• History of malignancy;
• 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);
• 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;
• Clinically significant abnormal 12-lead ECG findings;
• Cardiac MRI T2* <10ms;
• Myocardial infarction, cardiac arrest or cardiac failure within 1 year before screening/baseline visit;
• Unable to undergo MRI
• 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

Where it is running

• Children's Hospital Oakland — Oakland, California, United States
• University of Illinois at Chicago — Chicago, Illinois, United States
• Children's Hospital — New Orleans, Louisiana, United States
• University of Michigan Comprehensive Cancer Center — Ann Arbor, Michigan, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• The Children's Hospital of Philadephia — Philadelphia, Pennsylvania, United States
• Thomas Jefferson University — Philadelphia, Pennsylvania, United States
• Medical University of South Carolina — Charleston, South Carolina, United States
• Centro Infantil Boldrini — Campinas, Brazil
• Hospital de Clínicas de Porto Alegre-HCPA, — Rio Branco, Brazil
• Instituto Estadual de Hematologia Arthur Siqueira Cavalcanti - HEMORIO — Rio de Janeiro, Brazil
• Casa de Saúde Santa Marcelina — São Paulo, Brazil
• Universidade Federal de São Paulo — São Paulo, Brazil
• Hospital for Sick Kids — Toronto, Ontario, Canada
• Mansoura University Children's Hospital — Al Mansurah, Egypt
• Alexandria University — Alexandria, Egypt
• Zagazig University — Alexandria, Egypt
• Ains Shams University — Cairo, Egypt
• Cairo University — Cairo, Egypt
• Pediatric Hospital of Cairo University — Cairo, Egypt
• King Abdulaziz University Hospital — Jeddah, Western Region, Saudi Arabia
• Asser Central Hospital — Abhā, Saudi Arabia
• King Khalid University Hospital — Riyadh, Saudi Arabia
• National Center for Bone Marrow Transplantation — Tunis, Bad Saadoun, Tunisia
• Farhat Hached Hospital, Hematology Department — Sousse, Tunisia

Full record on ClinicalTrials.gov

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