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Trial record 1 of 1 for:    NCT01868477
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Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.

This study is currently recruiting participants.
Verified February 2017 by Novartis ( Novartis Pharmaceuticals )
Sponsor:
ClinicalTrials.gov Identifier:
NCT01868477
First Posted: June 4, 2013
Last Update Posted: February 15, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
  Purpose

The primary purpose of this trial is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome.

The addition of deferasirox to erythropoietin could lead to a potential synergism with the reduction of reactive oxygen species, through both the NF-kB pathway and the control of free toxic iron. This may create a better environment in the bone marrow for a better response with erythropoietin.

This study is designed to test in a prospective way the combination of deferasirox with erythropoietin in term of their effect on hematopoiesis.


Condition Intervention Phase
Adult Patients With Low Risk Myelodysplastic Syndrome Adult Patients With Int 1-risk Myelodysplastic Syndrome Drug: Erythropoietin alpha Drug: Deferasirox Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Phase II, Randomized, 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 Myelodysplastic Syndrome.

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

Primary Outcome Measures:
  • Change in hemoglobin levels [ Time Frame: Within 12 weeks ]
    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)


Secondary Outcome Measures:
  • Change in hemoglobin, platelets and neutrophil levels [ Time Frame: Within 24 weeks ]
    Proportion of patients achieving a hematological response within 24 weeks of treatment with deferasirox combined with erythropoietin and erythropoietin alone (increase in hemoglobin, improvement of neutropenia and thrombocytopenia) according to modified IWG 2006 criteria

  • Change in hemoglobin levels [ Time Frame: Within 24 weeks ]
    Proportion of patients achieving an erythroid response according to modified IWG 2006 criteria (increase in Hb ≥ 1.5 g/dL) within 24 weeks

  • Time to erythroid response [ Time Frame: up to 6 months ]
    Time to response is defined as the time from date of start of treatment to the date of event defined as the first documented response according to modified IWG 2006 criteria (increase in Hb ≥ 1.5 g/dL)

  • Time to hematologic response [ Time Frame: up to 6 months ]
    Time to response is defined as the time from date of start of treatment to the date of event defined as the first documented response (increase in hemoglobin, improvement of neutropenia and thrombocytopenia) according to modified IWG 2006 criteria

  • Duration of erythroid response [ Time Frame: up to 6 months ]
    Duration of response is defined as the time from onset of the first response to progression/relapse (decrease in Hb ≥ 1.5 g/dL from Hb value at response)

  • Number of adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: up to 6 months ]
    Incidence of adverse events (AEs) overall and by severity, 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 ]
    Change in serum ferritin from baseline to every visit throughout the study in the combination arm

  • Change in platelets and neutrophil levels [ Time Frame: Within 24 weeks ]
    Proportion of patients achieving a hematological improvement within 24 weeks of treatment with deferasirox combined with erythropoietin and erythropoietin alone (improvement of neutropenia and thrombocytopenia)

  • Time to hematologic improvement [ Time Frame: up to 6 months ]
    Time to improvement is defined as the time from date of start of treatment to the date of event defined as the first documented improvement (improvement of neutropenia and thrombocytopenia)


Estimated Enrollment: 60
Actual Study Start Date: January 28, 2014
Estimated Study Completion Date: May 31, 2017
Estimated Primary Completion Date: May 31, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Erythropoietin alpha
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.
Drug: Erythropoietin alpha
Experimental: Deferasirox + Erythropoietin alpha
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.
Drug: Erythropoietin alpha Drug: Deferasirox

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

  • A hemoglobin < 10 g/dL and ≥ 8 g/dL
  • History of transfusions < 10 RBC units and must not be RBC transfusion dependent
  • 300 ng/mL < serum ferritin < 1,500 ng/mL (Values within 10% difference above 1500 ng/ml or 10% difference below 300 ng/ml may be accepted at the investigator's discretion.
  • 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 (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 chronic medical conditions, and intermittent dexamethasone as antiemetics are allowed).
  • B12 and folate deficient patients with and without clinical symptoms (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.

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01868477


Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals +41613241111

  Show 42 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01868477     History of Changes
Other Study ID Numbers: CICL670A2421
First Submitted: May 30, 2013
First Posted: June 4, 2013
Last Update Posted: February 15, 2017
Last Verified: February 2017

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
myelodysplastic syndrome
MDS
myelodysplasia
blood disorder
cytopenias
low blood counts
progressive bone marrow failure

Additional relevant MeSH terms:
Syndrome
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Epoetin Alfa
Deferasirox
Hematinics
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action