A randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in nontransfusion-dependent thalassemia patients with iron overload (THALASSA) - THALASSA
- Conditions
- Chronic iron overload in patients with non-transfusion-dependent thalassemiaMedDRA version: 9.1 Level: HLT Classification code 10043389 Term: Thalassaemic disorders
- Registration Number
- EUCTR2007-007000-15-GB
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 166
Core:
•Male or female aged = 10 years [= 18 years for sites in Greece] with non-transfusion-dependent thalassemia syndromes, who has not received any transfusion within the previous 6 months prior to entry into the study
• LIC= 5 mg Fe/g dw measured by R2 MRI at screening
• Serum ferritin > 300 ng/mL at screening (two consecutive values at least 14 days apart from each other)
• Written informed consent obtained prior to any screening procedures
Extension:
• Patients who completed the core CICL670A2209 clinical trial
• Written informed consent obtained prior to entry into one year extension study
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
Core:
• Patients whose clinical condition allow phlebotomy
• HbS–variants of thalassemia syndromes
• Anticipated regular transfusion program during the study.
• Any blood transfusion 6 months prior to study start
• Chelation within 1 month prior to study treatment
• Patients unable to undergo study assessments including MRI
• Significant proteinuria
• Creatinine clearance = 60 ml/min on two measurements during visit 1 and visit 2
• Serum creatinine > ULN on two measurements during visit 1 and visit 2
• ALT >5 x ULN at visit 1 and visit 2
• Clinical evidence of active hepatitis B or C
• Known diagnosis of cirrhosis (confirmed by biopsy if available)
• Concomitant therapy with hydroxyurea, erythropoietin, butyrate
• Prior deferasirox treatment
• History of clinically relevant ocular and/or auditory toxicity related to iron chelation therapy
• History of positive HIV serology (ELISA or Western blot)
• Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of the study drug
• Active inflammatory diseases that may interfere with the accurate measurement of serum ferritin
• Pregnancy, breast feeding, patients of reproductive potential not employing an effective method of birth control
• Paticipationn another clinical trial or receiving a systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
• Hstory of non-compliance with medical regimens or patients who are considered potentially unreliable and/or not cooperative, unwilling or unable to comply with the protocol
• Hstory of hypersensitivity to any of the study drug or excipients
• Significant medical condition interfering with the ability to partake in this study
• History of drug or alcohol abuse within the 12 months prior to enrollment
• Body weight < 20 kg
Extension:
Please see protocol Post-text Supplement 2 for details
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method