Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01868477 | Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome. | PHASE2 | COMPLETED | 28 | — | — | Jan 28, 2014 | Apr 5, 2017 | Oct 31, 2018 | 30 | Algeria, Argentina +8 |
Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders
| Arm | Type | Description |
|---|---|---|
| Erythropoietin alpha | EXPERIMENTAL | Patients will receive erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be switched to the combination arm. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. |
| Deferasirox + Erythropoietin alpha | EXPERIMENTAL | Patients will receive deferasirox dispersible tablet (DT) 10 mg/kg/day or deferasirox film-coated tablet (FCT) 7 mg/kg/day in combination with erythropoietin 40,000 units/week. If after 4 weeks erythroid improvement is inadequate, erythropoietin dose will be escalated to 60,000 units/week. If after 12 weeks of treatment, erythroid improvement in inadequate, patients will be discontinued from the study. At any time when erythroid response is achieved, erythropoietin treatment will be stopped until end of study. Patients will continue deferasirox treatment. |
| Name | Type | Description |
|---|---|---|
| Deferasirox DFX, DT | DRUG | provided as dispersible tablets for oral use in 125 and 250, 500 mg |
| Erythropoietin alpha | DRUG | - |
| Deferasirox DFX, FCT | DRUG | provided as film-coated tablet for oral use in 90, 180, 360 mg strengths |
Key Inclusion Criteria: * Patients who had low- and Int-1-risk myelodysplastic syndrome * Documented diagnosis of the following: Myelodysplastic syndrome that lasted ≥ 3 months and \< 3 years Disease must not have been secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy fo...