Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) (ESCALATOR)
This study has been completed.
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00171301
First received: September 12, 2005
Last updated: August 28, 2011
Last verified: August 2011
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Purpose
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available. To evaluate the long-term safety and efficacy of deferasirox by measuring treatment success, change in liver iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by incidence of adverse events (AEs)and clinically significant lab parameters.
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-thalassemia Major Hemosiderosis Iron Overload Rare Anemia |
Drug: 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: | 1-year Extension to CICL670A2402 an Open-label, Multi-center Trial of the Efficacy and Safety of Long-term Treatment With Deferasirox (10 to 20 mg/kg/Day) in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2- Year Duration) |
Resource links provided by NLM:
Genetics Home Reference related topics:
beta thalassemia
Drug Information available for:
Deferasirox
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age [ Time Frame: From Core Study Baseline, to Extension End of Study, Up to 3 Years ] [ Designated as safety issue: No ]Success was defined as the percentage of participants with decreased liver iron content (LIC) at the end of extension study compared to core baseline (BL) LIC. Success Criteria: For participants with Baseline LIC from 1 - <7 mg Fe/g dw, success was achieved if LIC level maintained at 1 - <7 mg Fe/g dw. For participants with Baseline LIC ≥7 - <10 mg Fe/g dw, success was achieved if LIC dropped to between 1 and < 7 mg Fe/g dw. For participants with Baseline LIC ≥10 mg Fe/g dw, success was achieved if LIC dropped by at least 3 mg Fe/g dw. LIC was measured by biopsy or magnetic resonance imaging.
- Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ] [ Designated as safety issue: No ]
Liver MRI or Liver Biopsy was performed at the core study baseline (BL) and then 1 year and 2 years in the core study, baseline of the extension study and time of discontinuation from the extension visit (end of study). Liver iron content (LIC) is reported in milligram Iron per gram dry weight (mg Fe/g dw).
Absolute change in LIC from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-<7 mg Fe/g dw and ≥7 mg Fe/g dw.
Secondary Outcome Measures:
- Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ] [ Designated as safety issue: No ]Serum Levels were assessed at core study baseline (BL), 1 year, 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study) in monthly intervals. Serum Ferritin is reported in micrograms per Liter (µg/L).
- Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC) [ Time Frame: From Baseline of Core Study to End of Extension Study, up to 3 years ] [ Designated as safety issue: No ]Serum Levels were assessed at core study baseline (BL) and then 1 year and 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study). Serum Ferritin is reported in micrograms per Liter. Absolute change in Serum Ferritin from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-<7 mg Fe/g dw and ≥7 mg Fe/g dw.
| Enrollment: | 233 |
| Study Start Date: | June 2005 |
| Study Completion Date: | May 2008 |
| Primary Completion Date: | May 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Deferasirox
Deferasirox was given orally once daily (10 to 20 mg/kg) to participants 2 years and older based on participant's body weight.
|
Drug: Deferasirox
Deferasirox was administered orally once daily. Deferasirox was available as 125 mg, 250 mg, and 500 mg tablets.
Other Name: Exjade
|
Detailed Description:
Iron accumulation is an inevitable consequence of chronic blood transfusions and results in serious complications in the absence of chelation treatment to remove excess iron. Deferasirox (Exjade, ICL670) is an oral chelator with high iron-binding potency and selectivity. This extension study aimed at collecting efficacy and safety data during 2 years of treatment with deferasirox in the extension study or until deferasirox became commercially available in the countries where the centers were located, whichever came first. The population comprised of β-thalassemia patients with transfusional hemosiderosis who could not be satisfactorily treated with deferoxamine or deferiprone.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients completing the planned 12-month core study (NCT00171171)
- Female patients who have reached menarche and who are sexually active must use an effective method of contraception, 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.
- Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the core study protocol, or the planned extension protocol
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00171301
Locations
| Egypt | |
| Novartis Investigative Site | |
| Cairo, Egypt | |
| Lebanon | |
| Novartis Investigative Site | |
| Beirut, Lebanon | |
| Oman | |
| Novartis Investigative Site | |
| Muscat, Oman | |
| Saudi Arabia | |
| Novartis Investigative Site | |
| Riyadh, Saudi Arabia | |
| Syrian Arab Republic | |
| Novartis Investigative Site | |
| Damascus, Syrian Arab Republic | |
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: | NCT00171301 History of Changes |
| Other Study ID Numbers: | CICL670A2402E1 |
| Study First Received: | September 12, 2005 |
| Results First Received: | January 12, 2011 |
| Last Updated: | August 28, 2011 |
| Health Authority: | Lebanon: Institutional Review Board Egypt: The Egyptian Society for Medical Ethics Oman: The Research and Ethics Committee Saudia Arabia: Clinical Research Committee Syria: Institutional Review Board |
Keywords provided by Novartis:
|
Transfusional hemosiderosis Beta-thalassemia major Deferasirox |
iron overload rare anemia iron overload due to transfusion |
Additional relevant MeSH terms:
|
Anemia Beta-Thalassemia Hemosiderosis Thalassemia Iron Overload Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies |
Genetic Diseases, Inborn Iron Metabolism Disorders Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 22, 2013