Combination study of deferasirox and erythropoietin in patients with low- and int-1-riskmyelodysplastic syndrome.

Phase 1

Conditions: low- and int-1-risk myelodysplastic syndrome
MedDRA version: 16.0Level: LLTClassification code 10028534Term: Myelodysplastic syndrome NOSSystem Organ Class: 100000004864
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2013-000981-12-ES

Lead Sponsor: ovartis Farmacéutica, S.A.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 66

Inclusion Criteria

Patients with low- and Int-1-risk myelodysplastic syndrome
? Documented diagnosis of the following:
? Myelodysplastic syndrome lasting ? 3 months and < 2 years
? Disease must not be secondary to treatment with radiotherapy, chemotherapy,
and/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 < 1,000 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 33
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 33

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 (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 (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

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 deferasirox combined with erythropoietin vs.<br>erythropoietin 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 deferasirox combined with erythropoietin on<br>hematologic improvement within 24 weeks of treatment in patients randomized to<br>combination therapy at baseline defined by hemoglobin, platelets and neutrophil levels<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: baseline at day 1, followed by evaluation at week 2 and after 1,2 and 3 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Proportion of patients achieving a hematological response within 24 weeks of treatment with deferasirox combined with EPO (increase in Hb, improvement of neutropenia and thrombocytopenia) according to modified IWG 2006 criteria.;Timepoint(s) of evaluation of this end point: baseline at day 1, followed by evaluation at week 2 and after 1,2, 3, 4, 5 and 6 months

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