Clinical trial NCT00303329: Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
Official title: Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Anemia
Hemosiderosis
Keywords: β-thalassemia
rare chronic anemia
iron overload
deferasirox
chronic anemias
transfusional hemosiderosis
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2006-03-16
Enrollment: 184
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
timeFrame: Core study Baseline to the end of the study (up to 60 months)
Secondary outcomes:
measure: The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
timeFrame: Core study Baseline to end of extension study (up to 60 months)
measure: The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
timeFrame: Core study Baseline to end of extension study (up to 60 months)
measure: The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
timeFrame: Core study Baseline to end of extension study (up to 60 months)
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital and Research center at Oakland
state: California
city: Stanford
country: United States
facility: Stanford Hospital
state: California
city: Boston
country: United States
facility: Childres's Hospital Boston
state: Massachusetts
city: New York
country: United States
facility: New York Presbyterian Hospital
state: New York
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Brugge
country: Belgium
facility: Novartis Investigative Site
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Gent
country: Belgium
facility: Novartis Investigative Site
city: La Louvière
country: Belgium
facility: Novartis Investigative Site
city: Leuven
country: Belgium
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
city: Toronto
country: Canada
facility: Novartis Investigative Site
city: Creteil
country: France
facility: Novartis Investigative Site
city: Le Kremlin Bicetre
country: France
facility: Novartis Investigative Site
city: Lille
country: France
facility: Novartis Investigative Site
city: Troyes
country: France
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Hannover
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
city: Bologna
country: Italy
facility: Novartis Investigative Site
city: Brindisi
country: Italy
facility: Novartis Investigative Site
city: Cagliari
country: Italy
facility: Novartis Investigative Site
city: Genova
country: Italy
facility: Novartis Investigative Site
city: Milan
country: Italy
facility: Novartis Investigative Site
city: Pavia
country: Italy
facility: Novartis Investigative Site
city: Rome
country: Italy
facility: Novartis Investigative Site
city: Torino
country: Italy
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: