iron overload in sickle cell disease or other anemias MedDRA version: 20.0 Level: SOC Classification code 10005329 Term: Blood and lymphatic system disorders System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: Ferriprox 500 mg film-coated tablets
Product Name: Ferriprox
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: DEFERIPRONE
Sponsors
ApoPharma Inc.
Eligibility
Sex/Gender
All
Inclusion 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 Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 170 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1.Thalassemia syndromes; 2.Myelodysplastic syndrome (MDS) or myelofibrosis; 3.Diamond Blackfan anemia; 4.Primary bone marrow failure; 5.Patients with a 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* <10 ms 15.Unable to undergo MRI 16.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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the efficacy of deferiprone vs. deferoxamine in the treatment of iron overload in patients with sickle cell disease or other anemias.; Secondary Objective: To evaluate the effect of deferiprone vs. deferoxamine on the patients’ quality of life; To evaluate the safety and tolerability of deferiprone vs. deferoxamine ;Primary end point(s): The change from baseline to Week 52 in liver iron concentration (measured by MRI).;Timepoint(s) of evaluation of this end point: MRI scans will be performed at the screening/baseline, Week 26, and Week 52 (or early termination) visits | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Change from baseline to Week 52 in patient-reported quality of life (SF-36 or Child Health Questionnaire); 2. Change from baseline to Week 52 in cardiac MRI T2*; 3. Change from baseline to Week 52 in serum ferritin. Safety Endpoints: 1. Frequency, severity and time to onset/duration of adverse events (AEs); 2. Frequency of serious adverse events (SAEs); 3. Discontinuation due to AEs; 4. Hematology assessments; 5. Blood clinical biochemistry assessments; 6. 12-lead ECG. ;Timepoint(s) of evaluation of this end point: The SF-36 Questionnaire will be completed by patients aged 18 years old and above at the baseline, Week 26 and Week 52 (or early termination),Child Health Questionnaire-Parent Form 50 (CHQ-PF50) and the Child Health Questionnaire Child Form 87 (CHQ-CF87)will be completed for patients aged 17 years and under at the baseline, Week 26, and Week 52 (or early termination);MRI scans for the assessment of cardiac MRI T2*: at the screening/baseline, Week 26, and Week 52 (or early termination);Serum ferritin: at baseline and at the Weeks 12, 26, 40 and 52 (or early termination);Hematology assessments:at screening/baseline, weekly until Week 26, and then biweekly until Week 52 (or early termination);Chemistry assessments: at screening/baseline visit and monthly up to Week 52(or early termination). | — |
Countries
Brazil, Canada, Egypt, Saudi Arabia, United Kingdom, United States
Contacts
Public ContactCaroline Fradette
ApoPharma Inc.
Outcome results
None listed