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

• Register: EUCTR
• Last refreshed on: 4 September 2017
• Main ID: EUCTR2016-004322-42-Outside-EU/EEA
• Date of registration: 31/08/2017
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)”.
• Scientific title: Efficacy and safety of long-term treatment with ICL670 in beta-thalassemia patients with transfusional hemosiderosis
• Target sample size: 250
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-004322-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Egypt, Lebanon, Oman, Saudi Arabia, Syria

Contacts

• Name: Clinical trial Information Desk
• Affiliation: Novartis pharma AG

• Name: Clinical trial Information Desk
• Affiliation: Novartis pharma AG

Key inclusion & exclusion criteria

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 162
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 69
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Tranfusional hemosiderosis

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: Exjade
Product Name: Exjade
Product Code: ICL670
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: DEFERASIROX
CAS Number: 201530-41-8
Current Sponsor code: CICL670A2402E1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. From Core Study Baseline, to Extension End of Study, Up to 3 Years
2. From Baseline of Core Study to End of Extension Study, up to 3 years

Secondary Objective: 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.

Main Objective: 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.

Primary end point(s): 1.Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age
2. Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. From Baseline of Core Study to End of Extension Study, up to 3 years
2. From Baseline of Core Study to End of Extension Study, up to 3 years

Secondary end point(s): 1. Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study
2. Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC)

Secondary ID(s)

CICL670A2402E1

Source(s) of Monetary Support

Novartis Pharma Serivces AG