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Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00481143
First received: May 31, 2007
Last updated: November 2, 2011
Last verified: November 2011
  Purpose

The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions.

This trial is not recruiting patients in the United States.


Condition Intervention Phase
Myelodysplastic Syndromes
Transfusion Dependent Iron Overload
Drug: ICL670/Deferasirox
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 63
Study Start Date: May 2007
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: deferasirox Drug: ICL670/Deferasirox
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload
  • Patients of either gender and age ≥ 18 years
  • History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
  • Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
  • Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Exclusion Criteria:

  • Non-transfusion related iron overload
  • Treatment with deferasirox (ICL670) before study start
  • Patients with a concomitant malignant disease
  • Patients with out of range lab values
  • History of nephrotic syndrome
  • Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from 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.
  • Patients with active uncontrolled infectious disease
  • Pregnancy or breast feeding

Other protocol-defined inclusion/exclusion criteria may apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00481143

Locations
Germany
Novartis Investigative Site
Berlin, Germany, 12200
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmeceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00481143     History of Changes
Other Study ID Numbers: CICL670ADE03
Study First Received: May 31, 2007
Last Updated: November 2, 2011
Health Authority: Germany: Federal Institute for Drugs and Medical Devices

Keywords provided by Novartis:
Myelodysplastic Syndrome
MDS
iron overload
chelation
chelators
ICL670
Deferasirox
serum ferritin
LIC
transfusional hemosiderosis
Refractory Anemia
RA
Refractory Anemia with Ringed Sideroblasts
RARS
blood transfusions
Mydelodysplastic Syndrome(s)

Additional relevant MeSH terms:
Iron Overload
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Disease
Hematologic Diseases
Iron Metabolism Disorders
Metabolic Diseases
Neoplasms
Pathologic Processes
Precancerous Conditions
Deferasirox
Chelating Agents
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Sequestering Agents

ClinicalTrials.gov processed this record on November 27, 2014