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. Read our disclaimer for details. Identifier: NCT01058369
Recruitment Status : Terminated (Insufficient patient recruitment (only 2 patients))
First Posted : January 28, 2010
Last Update Posted : June 1, 2015
Novartis Pharmaceuticals
Information provided by (Responsible Party):
University of Erlangen-Nürnberg Medical School

Brief Summary:
Study outline: Deferasirox (Exjade®) is regularly used in severe iron overload in order to avoid organ damage of liver, heart and other organs. It has been proposed, that iron overload may not only impose damage to other organs but also to the bone marrow and thus worsen hematopoietic insufficiency in patients with MDS. Patients presenting with low or INT-1 risk MDS with only mild iron overload will be treated with deferasirox in this study. It will be analyzed if hematological improvement can be observed during this treatment.

Condition or disease Intervention/treatment Phase
Myelodysplastic Syndromes Drug: Deferasirox (Novartis Pharma) Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Early Treatment With Deferasirox (Exjade®) in Low Risk MDS - a Prospective Multicentre Single-arm Single-stage Phase II Study -
Study Start Date : April 2010
Actual Primary Completion Date : January 2013
Actual Study Completion Date : January 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Deferasirox

Arm Intervention/treatment
Experimental: Deferasirox Drug: Deferasirox (Novartis Pharma)
Treatment period 102 weeks. Starting dose 10mg/kg/day. Up to 30/mg/kg according to dose adjustment table as specified in the protocol
Other Name: Exjade(R)

Primary Outcome Measures :
  1. Fraction of patients with hematologic improvement according to modified IWG criteria (reduction of transfusions and/or increase in Hb, improvement of neutropenia and thrombocytopenia) [ Time Frame: within two years ]

Secondary Outcome Measures :
  1. Evaluate the safety and tolerability profile of deferasirox in MDS patients [ Time Frame: within two years ]
  2. Effectiveness of iron depletion [ Time Frame: within two years ]
  3. Correlation between hematological improvement and effectiveness of iron depletion [ Time Frame: two years ]
  4. Development of bone marrow morphology [ Time Frame: two years ]
  5. Correlation between hematological improvement and pretreatment parameters. Extension of this analysis to MDS patients on deferasirox within the licensed indication (more severe iron overload) [ Time Frame: two years ]
  6. overall survival [ Time Frame: within two years ]
  7. AML-free survival [ Time Frame: within two years ]

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

Inclusion Criteria:

  • MDS of subtype RA, RARS, RCMD, RCMD-RS (i.e. lower risk)
  • RAEB I allowed, if clinically stable for > 3 months
  • 5q-minus syndrome allowed, if lenalidomide unsuccessful or unavailable at the time of inclusion
  • IPSS score < intermediate-1
  • transfusion dependent or Hb < 10,5 g/dl
  • History of less than 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs), except for transfusions for acute bleeding
  • Serum ferritin > 300 µg/l and < 1500 μg/l. This level should have been verified at least at two occasions within 3 months. Samples must be obtained in the absence of concomitant severe infection
  • no indication for EPO (due to high endogenous EPO levels) or EPO without benefit in the past
  • no indication and/or no plans for cytostatic drugs
  • no previous exposure to cytostatic drugs, thalidomide, lenalidomide, G-CSF or EPO or exposure to any of these drugs has been terminated since > 8 weeks (4 weeks for G-CSF).
  • no indication and/or no plans for stem cell transplantation
  • stable or worsening cytopenia during the past 8 weeks. If in doubt, extend screening period to >= 8 weeks
  • Patients of either gender and age > 18 years
  • Life expectancy > 12 months
  • Females of childbearing potential must use double-barrier contraception (for example orale contraception and condom).
  • Mental ability of the patient to understand explications concerning the study and to understand and follow instructions of the investigating physician
  • Written informed consent by the patient

Exclusion Criteria:

  • Treatment with deferasirox or other chelation therapy for periods > 4 weeks before study start
  • Patients with intolerance to Deferasirox
  • Patients with a concomitant second malignant disease, possibly interfering with life expectancy
  • Patients with mean levels of alanine aminotransferase (ALT) > 5x ULN
  • Patients with uncontrolled systemic hypertension
  • Patients with serum creatinine > 1.5x the upper limit of normal (ULN) or a creatinine clearance < 60 ml/min according to the MDRD formula (Levey 2005)
  • History of nephrotic syndrome
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
  • Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
  • Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
  • Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
  • history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
  • history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
  • history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
  • history of urinary obstruction or difficulty in voiding
  • History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
  • History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by laboratory assays conducted during the screening period
  • Patients with active uncontrolled infectious disease
  • Pregnancy or breast feeding
  • QT > 470 msec on screening ECG
  • Patients with a history of Torsades de Pointes

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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 identifier (NCT number): NCT01058369

Medizinische Klinik 5, Universitätsklinikum Erlangen
Erlangen, Bavaria, Germany, 91054
Sponsors and Collaborators
University of Erlangen-Nürnberg Medical School
Novartis Pharmaceuticals
Study Chair: Stefan Krause, Prof. Dr. Medizinische Klinik 5, Universitätsklinikum Erlangen

Responsible Party: University of Erlangen-Nürnberg Medical School Identifier: NCT01058369     History of Changes
Other Study ID Numbers: CICL670ADE06T
First Posted: January 28, 2010    Key Record Dates
Last Update Posted: June 1, 2015
Last Verified: May 2015

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action