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Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Primary Purpose

Anemia, Hemosiderosis

Status
Completed
Phase
Phase 2
Locations
International
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 Anemia focused on measuring β-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosis

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients completed the planned 12-month core study Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation Exclusion Criteria: Pregnant or breast feeding patients Other protocol-defined inclusion/exclusion criteria may apply

Sites / Locations

  • Children's Hospital and Research center at Oakland
  • Stanford Hospital
  • Childres's Hospital Boston
  • New York Presbyterian Hospital
  • Children's Hospital of Philadelphia
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • 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

Deferasirox daily oral dose between 5-40 mg/kg/day

Outcomes

Primary Outcome Measures

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.

Secondary Outcome Measures

The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.

Full Information

First Posted
October 14, 2005
Last Updated
April 15, 2011
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00303329
Brief Title
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
Official Title
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
April 2011
Overall Recruitment Status
Completed
Study Start Date
March 2004 (undefined)
Primary Completion Date
October 2008 (Actual)
Study Completion Date
October 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years. The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Hemosiderosis
Keywords
β-thalassemia, rare chronic anemia, iron overload, deferasirox, chronic anemias, transfusional hemosiderosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
184 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Arm Description
Deferasirox daily oral dose between 5-40 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Other Intervention Name(s)
ICL670
Intervention Description
Deferasirox available as 125 mg, 250 mg or 500 mg tablets
Primary Outcome Measure Information:
Title
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Description
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
Time Frame
Core study Baseline to the end of the study (up to 60 months)
Secondary Outcome Measure Information:
Title
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
Description
Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.
Time Frame
Core study Baseline to end of extension study (up to 60 months)
Title
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
Description
Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.
Time Frame
Core study Baseline to end of extension study (up to 60 months)
Title
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Description
Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.
Time Frame
Core study Baseline to end of extension study (up to 60 months)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients completed the planned 12-month core study Female patients who have reached menarche and who are sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation Exclusion Criteria: Pregnant or breast feeding patients 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
Children's Hospital and Research center at Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609-1809
Country
United States
Facility Name
Stanford Hospital
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Childres's Hospital Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
New York Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104-4399
Country
United States
Facility Name
Novartis Investigative Site
City
Brugge
Country
Belgium
Facility Name
Novartis Investigative Site
City
Bruxelles
Country
Belgium
Facility Name
Novartis Investigative Site
City
Gent
Country
Belgium
Facility Name
Novartis Investigative Site
City
La Louvière
Country
Belgium
Facility Name
Novartis Investigative Site
City
Leuven
Country
Belgium
Facility Name
Novartis Investigative Site
City
Montreal
Country
Canada
Facility Name
Novartis Investigative Site
City
Toronto
Country
Canada
Facility Name
Novartis Investigative Site
City
Creteil
Country
France
Facility Name
Novartis Investigative Site
City
Le Kremlin Bicetre
Country
France
Facility Name
Novartis Investigative Site
City
Lille
Country
France
Facility Name
Novartis Investigative Site
City
Troyes
Country
France
Facility Name
Novartis Investigative Site
City
Duesseldorf
Country
Germany
Facility Name
Novartis Investigative Site
City
Hannover
Country
Germany
Facility Name
Novartis Investigative Site
City
Ulm
Country
Germany
Facility Name
Novartis Investigative Site
City
Bologna
Country
Italy
Facility Name
Novartis Investigative Site
City
Brindisi
Country
Italy
Facility Name
Novartis Investigative Site
City
Cagliari
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
Country
Italy
Facility Name
Novartis Investigative Site
City
Milan
Country
Italy
Facility Name
Novartis Investigative Site
City
Pavia
Country
Italy
Facility Name
Novartis Investigative Site
City
Rome
Country
Italy
Facility Name
Novartis Investigative Site
City
Torino
Country
Italy
Facility Name
Novartis Investigative Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

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