| Trial name or title |
Combination Study of Deferasirox and Erythropoietin in Patients With Low‐ and Int‐1‐risk Myelodysplastic Syndrome |
| Methods |
An open‐label, phase II, randomised, pilot study to assess the effect in term of erythroid improvement of deferasirox combined with erythropoietin compared to erythropoietin alone in patients with low‐ and int‐1‐risk MDS |
| Participants |
Inclusion criteria:
MDS lasting ≥ 3 months and < 2 years Disease must not be secondary to treatment with radiotherapy, chemotherapy, or immunotherapy for malignant or autoimmune diseases
A hemoglobin < 10 g/dL and > 6 g/dL (no RBC transfusions are allowed during study) History of transfusions < 10 RBC units 300 ng/mL < serum ferritin < 1000 ng/mL Endogenous erythropoietin levels < 500 units/L
Exclusion criteria:
Patients with MDS with isolated del(5q) Patients who had received prior EPO treatment or other recombinant growth factors regardless of the outcome (patients who had received prior EPO treatment or other recombinant growth factors for less than 4 weeks and not within 3 months before screening without a documented response are allowed) Patients receiving steroids or immunosuppressive therapy for the improvement of hematological parameters (prophylactic hydrocortisone to prevent transfusion reaction, steroid for adrenal failure and intermittent dexamethasone as antiemetic are allowed). B12 and folate deficient patients (patients could be rescreened after successful therapy of B12 and folate deficiency) Uncontrolled seizures or uncontrolled hypertension
Other protocol‐defined inclusion/exclusion criteria may apply. |
| Interventions |
Group A: Erythropoietin alpha
Group B: Deferasirox + erythropoietin alpha |
| Outcomes |
Primary outcome measure:
Secondary outcome measures:
Change in hemoglobin, platelets and neutrophil levels (time frame: within 24 weeks) Change in hemoglobin levels (time frame: within 24 weeks) Time to erythroid response Time to hematologic improvement Duration of erythroid response Number of adverse events (AEs) and serious adverse events (SAEs) Iron parameters by change in serum ferritin (time frame: baseline, followed by evaluation after 1, 2, 3, 4, 5 and 6 months)
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| Starting date |
Study start date: January 2014
Estimated primary completion date: June 2015 (Final data collection date for primary measure) |
| Contact information |
Novartis Pharmaceuticals |
| Notes |
Planned sample size: 60 ClinicalTrials.gov identifier: NCT01868477 EUCTR identifier: EUCTR2013‐000981‐12‐SE Novartis Study ID Number: CICL670A2421 Registry entry last updated on 10 April 2014 |
