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

NCT ID: NCT00171301

Last Updated: 2011-08-31

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

233 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-06-30

Study Completion Date

2008-05-31

Brief Summary

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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

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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.

Conditions

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Beta-thalassemia Major Hemosiderosis Iron Overload Rare Anemia

Keywords

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Transfusional hemosiderosis Beta-thalassemia major Deferasirox iron overload rare anemia iron overload due to transfusion

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferasirox

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

Group Type EXPERIMENTAL

Deferasirox

Intervention Type DRUG

Deferasirox was administered orally once daily. Deferasirox was available as 125 mg, 250 mg, and 500 mg tablets.

Interventions

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Deferasirox

Deferasirox was administered orally once daily. Deferasirox was available as 125 mg, 250 mg, and 500 mg tablets.

Intervention Type DRUG

Other Intervention Names

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Exjade

Eligibility Criteria

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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
Minimum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Cairo, , Egypt

Site Status

Novartis Investigative Site

Beirut, , Lebanon

Site Status

Novartis Investigative Site

Muscat, , Oman

Site Status

Novartis Investigative Site

Riyadh, , Saudi Arabia

Site Status

Novartis Investigative Site

Damascus, , Syria

Site Status

Countries

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Egypt Lebanon Oman Saudi Arabia Syria

Other Identifiers

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CICL670A2402E1

Identifier Type: -

Identifier Source: org_study_id