Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.

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Key information

Conditions

Liver Iron Overload Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia beta-Thalassemia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Novartis Investigative Site, Cagliari, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
Patients currently participating in the food-effect sub-study, according to amendment 3.
Ability to provide written informed consent prior to participation in this non-comparative extension study.
Female patients 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.
Body weight of at least 35 kg.

Exclusion criteria

Pregnant or breastfeeding patients.
History of non-compliance to medical regimens and patients who are considered potentially unreliable.
Proteinuria > 300 mg/L second void morning urine.
Patients with serum creatinine above the upper limit normal.

Other protocol-defined inclusion/exclusion criteria may apply.

Treatment and study plan

Deferasirox

Drug

10 mg/kg or 30 mg/kg orally daily

Primary outcomes

The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox

Time frame: Baseline to 7 Years

The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.

Secondary outcomes

Relative Change in Serum Ferritin From Baseline to 3.5 Years

Time frame: Baseline to 3.5 years

The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Day Deferoxamine in Patients With Transfusion-dependent Iron Overload

Important dates

Study start: 2003
Primary completion: 2008
Study completion: 2008
First posted: Dec 16, 2009
Registry last updated: Aug 11, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed