Phase 2
Completed N=60
Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload
Cardiac Iron Overload
Source: ClinicalTrials.gov NCT01254227 ↗
Enrolled (actual)
60
Serious AEs
28.3%
Results posted
Jun 2021
Primary outcomePrimary: Change in Cardiac Iron Content From Baseline to Month 12 — 1.09 ratio
Summary
This study will evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed be deferasirox monotherapy in patients with severe iron overload due to chronic blood transfusions.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Cardiac Iron Content From Baseline to Month 12 |
1.09 | — |
| SECONDARY Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24 |
12.50; 19.23; 33.33; 47.22 | — |
| SECONDARY Change in Cardiac Iron Content From Baseline to Month 6,18 and 24 |
1.02; 1.17; 1.30 | — |
| SECONDARY Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24 |
66.5; 0.1; -0.2; 0.6; 0.9 | — |
| SECONDARY Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24 |
67.1; -1.2; -1.6; -2.1; -1.4 | — |
| SECONDARY Time to Achieve From Baseline (FAS) of at Least 10% at Month 24 |
722.0 | — |
| SECONDARY Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24 |
4.18; 4.31; 3.93; 3.51; 3.14 | — |
Eligibility Criteria
Inclusion Criteria
- Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy
- Myocardial T2* value that is ≥ 5 and < 10 ms
- Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (MRI)
- Liver Iron Concentration (LIC) ≥ 7 mg Fe /g dw as determined by R2 MRI.
- Lifetime history of at least 50 units of red blood cell transfusions, and must be receiving at least ≥ 8 units/yr of red blood cell transfusions
- Serum ferritin ≥ 1000 ng/mL
Exclusion Criteria
- Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias)
- Patients unable to undergo study assessments including MRI
- Patients with serum creatinine greater than Upper limit of normal ULN)range or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥1.0 mg/mg in a non-first void urine sample at baseline.
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01254227). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.