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

Primary Purpose

Myelodysplastic Syndromes, Beta-Thalassemia

Status
Completed
Phase
Phase 4
Locations
Hungary
Study Type
Interventional
Intervention
deferasirox
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

Outcomes

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

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

Full Information

First Posted
November 28, 2007
Last Updated
February 22, 2017
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00564941
Brief Title
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
Official Title
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
December 2007 (undefined)
Primary Completion Date
July 2011 (Actual)
Study Completion Date
July 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes, Beta-Thalassemia
Keywords
Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
309 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
deferasirox
Other Intervention Name(s)
ICL670
Primary Outcome Measure Information:
Title
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)
Secondary Outcome Measure Information:
Title
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Budapest
Country
Hungary
Facility Name
Novartis Investigative Site
City
Debrecen
Country
Hungary
Facility Name
Novartis Investigative Site
City
Pecs
Country
Hungary
Facility Name
Novartis Investigative Site
City
Szeged
Country
Hungary

12. IPD Sharing Statement

Learn more about this trial

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

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