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

Novartis Pharmaceuticals184 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

184

Conditions

Anemia Hemosiderosis

Interventions

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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

Locations (28)

Children's Hospital and Research center at Oakland

Oakland, California, United States

Stanford Hospital

Stanford, California, United States

Childres's Hospital Boston

Boston, Massachusetts, United States

New York Presbyterian Hospital

New York, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Novartis Investigative Site

Bruges, Belgium

Novartis Investigative Site

Brussels, Belgium

Novartis Investigative Site

Ghent, Belgium

Novartis Investigative Site

La Louvière, Belgium

Novartis Investigative Site

Leuven, Belgium

...and 18 more locations

Outcomes

Primary Outcomes

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.

Time frame: Core study Baseline to the end of the study (up to 60 months)

Secondary Outcomes

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.

Time frame: Core study Baseline to end of extension study (up to 60 months)

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.

Time frame: Core study Baseline to end of extension study (up to 60 months)

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.

Time frame: Core study Baseline to end of extension study (up to 60 months)