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

Phase 2

Completed

• Conditions: Anemia; Hemosiderosis
• Interventions: Drug: Deferasirox

• Registration Number: NCT00303329
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-03
• Study First Submitted: 2005-10-14
• Study First Submitted QC: 2006-03-15
• Study First Posted: 2006-03-16
• Primary Completion: 2008-10
• Study Completion: 2008-10
• Results First Submitted: 2010-12-15
• Status Verified: 2011-04
• Results First Submitted QC: 2011-04-15
• Last Update Submitted: 2011-04-15
• Results First Posted: 2011-05-09
• Last Update Posted: 2011-05-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 184

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths	Core study Baseline to the end of the study (up to 60 months)	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

Name	Time	Method
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study	Core study Baseline to end of extension study (up to 60 months)	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	Core study Baseline to end of extension study (up to 60 months)	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	Core study Baseline to end of extension study (up to 60 months)	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.

Trial Locations

Locations (6)

Childres's Hospital Boston; 🇺🇸 Boston, Massachusetts, United States

Stanford Hospital; 🇺🇸 Stanford, California, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Children's Hospital and Research center at Oakland; 🇺🇸 Oakland, California, United States

Novartis Investigative Site; 🇬🇧 London, United Kingdom

New York Presbyterian Hospital; 🇺🇸 New York, New York, United States