Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation

Study Overview
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Brief Summary
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Extend evaluation of deferasirox film-coated tablet (FCT) formulation

Detailed Description
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Collection of additional safety and efficacy data with deferasirox film-coated tablet (FCT) in patients who had completed study CICL670F2201 Provide patients who completed 24 weeks of treatment in the core study, CICL670F2201, the possibility to have additional treatment with the deferasirox FCT. Collect additional longer term data on the safety and the tolerability of the deferasirox FCT. Collect efficacy data on the deferasirox FCT in reduction or maintenance of iron burden as measured by serum ferritin level.

Official Title
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Open-label, Multicenter, Single Arm, Phase III Study to Collect Additional Safety and Efficacy Data With Deferasirox Film-coated Tablets in Patients Completing Study CICL670F2201

Conditions
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Chronic Iron Overload

Intervention / Treatment
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* Drug: Deferasirox


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria for subjects: Completed 24-weeks of study treatment as described in the core protocol (CICL670F2201). Were deemed to have tolerated deferasirox treatment by the investigator. Provided written informed consent/assent before any study-specific procedures were performed. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators were to have also obtained assent of patients according to local, regional or national guidelines. Key Exclusion for subjects: The exclusion criteria followed those described for the core protocol CICl670F2201, which were as follows: Creatinine clearance below the contraindication limit in the locally approved prescribing information. Serum creatinine > 1.5 × upper limit of normal range (ULN) at Screening Alanine aminotransferase (ALT)/serum glutamic pyruvic transaminase (SGPT) > 5 × ULN, Significant proteinuria Patients with significant impaired gastrointestinal function or gastrointestinal disease Clinical or laboratory evidence of active Hepatitis B or Hepatitis C Patients with psychiatric or addictive disorders Patients with a known history of HIV seropositivity (Elisa or Western blot). History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there was an evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin. Patients with a history of hypersensitivity to any of the study drug or excipients. Patients with significant medical condition that could interfere with the ability to participate in this study Patients who were participating in another clinical trial or receiving an investigational drug. Patients using prohibited medication, Patients with liver disease with severity of Child-Pugh Class B or C. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they were using effective methods of contraception during dosing of study treatment

Ages Eligible for Study
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Minimum Age: 10 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferasirox<br>Treatment will be administered daily for up to 24 months. For each patient the daily dose is calculated based on the patient's actual body weight. | Drug: Deferasirox<br>* Deferasirox FCT will be provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overview of Number of Participants With Adverse Events | Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study | Baseline up to approximately 25 months | 
| Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 
| Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline | Baseline, 6 and 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study | Baseline, 6 and 12 months | 
| Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study | Baseline, 6 and 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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adult, pediatric,, anemia., Myelodysplastic Syndrome, Thalassemia, Film coated tablet, ICL670, MDS, Chelation, Deferasirox, Iron overload