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

Phase 1

• Conditions: low- and int-1-risk myelodysplastic syndrome; MedDRA version: 20.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2013-000981-12-GB
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-07-23
• Study Completion: 2017-03-22
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 23

Inclusion Criteria

? Patients with low- and Int-1-risk myelodysplastic syndrome
? Documented diagnosis of the following:
a) Myelodysplastic syndrome lasting = 3 months and < 3 years
b) Disease must not be secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
? Hemoglobin < 10 g/dL and > 6 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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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 (stable steroid treatment for adrenal failure or other chronic medical conditions, and intermittent dexamethasone as antiemetic 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

Other exclusion criteria as per full protocol may apply

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;<br> Secondary Objective: Key secondary objective:<br> To assess the effect of treatment with DFX + EPO and EPO alone on hematological response within 24 weeks of treatment.<br> <br> Other secondary objectives as per protocol may apply.<br> ;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
Timepoint(s) of evaluation of this end point: within 24 weeks;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.