Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)

Phase 4

Completed

• Conditions: Beta-thalassemia Major; Hemosiderosis; Iron Overload; Rare Anemia
• Interventions: Drug: Deferasirox

• Registration Number: NCT00171301
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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.

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.

Study Timeline

• Study Start: 2005-06
• Study First Submitted: 2005-09-12
• Study First Submitted QC: 2005-09-14
• Study First Posted: 2005-09-15
• Primary Completion: 2008-05
• Study Completion: 2008-05
• Results First Submitted: 2011-01-12
• Results First Submitted QC: 2011-06-30
• Results First Posted: 2011-07-29
• Status Verified: 2011-08
• Last Update Submitted: 2011-08-28
• Last Update Posted: 2011-08-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 233

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	Deferasirox was given orally once daily (10 to 20 mg/kg) to participants 2 years and older based on participant's body weight.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age	From Core Study Baseline, to Extension End of Study, Up to 3 Years	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	From Baseline of Core Study to End of Extension Study, up to 3 years	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

Name	Time	Method
Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study	From Baseline of Core Study to End of Extension Study, up to 3 years	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)	From Baseline of Core Study to End of Extension Study, up to 3 years	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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇸🇾 Damascus, Syrian Arab Republic