Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00564941
First received: November 28, 2007
Last updated: September 8, 2011
Last verified: September 2011
  Purpose

This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload


Condition Intervention Phase
Myelodysplastic Syndromes
Beta-Thalassemia
Drug: deferasirox
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload [ Time Frame: monthly during the therapy and at the end of the treatment (aftr 9 months therapy) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events [ Time Frame: during the treatment (9 months) ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 309
Study Start Date: December 2007
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox Drug: deferasirox
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria:

  • In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion
  • Serum ferritin> 1800 µg/L
  • Age: 18-80 years
  • men and women
  • Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells
  • Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason
  • Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2
  • written informed consent

Exclusion criteria:

  • beta thalassaemia minor,
  • haemosiderosis caused by other than chronic transfusional iron overload,
  • patients with impaired renal function (Creatinin clearance< 60 ml/ min),
  • pregnancy,
  • lactation,
  • patient of childbearing potential unwilling to use contraceptive precautions
  • known hypersensitivity to deferasirox or any ingredients,
  • impaired hepatic function (SGOT,SGPT 5x above UNL).
  • Patients severely ill due to underlying disease progression or other severe concomitant disease.
  • Patients with poor prognosis of karyotype
  • patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition
  • History of nephrotic syndrome
  • Significant proteinuria
  • Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
  • Patients with positive test to HIV

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: NCT00564941

Locations
Hungary
Novartis Investigative Site
Budapest, Hungary
Novartis Investigative Site
Debrecen, Hungary
Novartis Investigative Site
Pecs, Hungary
Novartis Investigative Site
Szeged, Hungary
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00564941     History of Changes
Other Study ID Numbers: CICL670AHU02
Study First Received: November 28, 2007
Last Updated: September 8, 2011
Health Authority: Hungary: National Institute of Pharmacy

Keywords provided by Novartis:
Myelodysplastic Syndromes
beta-Thalassemia
Iron Overload
Iron Chelating Agents
deferasirox

Additional relevant MeSH terms:
Beta-Thalassemia
Iron Overload
Myelodysplastic Syndromes
Preleukemia
Syndrome
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Bone Marrow Diseases
Disease
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
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 October 20, 2014