Combination study of deferasirox and erythropoietin in patients with low- and int-1-riskmyelodysplastic syndrome.
- Conditions
- MedDRA version: 19.1Level: LLTClassification code 10028534Term: Myelodysplastic syndrome NOSSystem Organ Class: 100000004864Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]low- and int-1-risk myelodysplastic syndrome
- Registration Number
- EUCTR2013-000981-12-SE
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 60
Patients with low- and Int-1-risk myelodysplastic syndrome
? Documented diagnosis of the following:
? Myelodysplastic syndrome lasting = 3 months and < 3 years
? Disease must not be secondary to treatment with radiotherapy, chemotherapy,
and/or immunotherapy for malignant or autoimmune diseases
? Hemoglobin < 10 g/dL and = 8 g/dL (Values within 5% difference above 10g/dL or 5% difference below 8 g/dL may be accepted at discretion of the investigator if the patient represents the investigational population. However a notification to the study team is required)
? History of < 10 RBC transfusions in total and must not be RBC transfusion dependent
? 300 ng/mL < serum ferritin < 1,500 ng/mL
? Endogenous erythropoietin levels < 500 units/L
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 40
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20
? Patients with MDS with isolated del(5q)
? Patients who had received prior EPO treatment or other recombinant growth factors
regardless of the outcome (Patient 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 (stable steroid treatment for adrenal failure or other chronic medical conditions, and intermittent dexamethasone as antimetic are allowed).
? B12 and folate deficient patients with or without clinical symptoms (patients could be rescreened after successful therapy of B12 and folate deficiency)
? Uncontrolled seizures or uncontrolled hypertension
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the effect of treatment with DFX+EPO vs. EPO alone on erythropoiesis after 12 weeks of treatment defined by hemoglobin levels;Secondary Objective: Key secondary objective:<br>To assess the effect of treatment with DFX + EPO and EPO alone on<br>hematological response within 24 weeks of treatment<br><br>Other secondary objectives as per protocol may apply.;Primary end point(s): Difference in proportion of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria (increase in Hb = 1.5 g/dL);Timepoint(s) of evaluation of this end point: within 12 weeks
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Proportion of patients achieving a hematological response within 24 weeks of treatment with DFX + EPO and EPO alone (increase in Hb, improvement of neutropenia and thrombocytopenia) according to modified IWG 2006 criteria.;Timepoint(s) of evaluation of this end point: within 24 weeks