Chronic Iron Overload
Conditions
Keywords
adult, pediatric,, anemia., Myelodysplastic Syndrome, Thalassemia, Film coated tablet, ICL670, MDS, Chelation, Deferasirox, Iron overload
Brief summary
Extend evaluation of deferasirox film-coated tablet (FCT) formulation
Detailed description
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.
Interventions
DRUGDeferasirox
Deferasirox FCT will be provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Overview of Number of Participants With Adverse Events | Baseline up to approximately 25 months | 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 |
| Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Baseline, 6 and 12 months | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | Baseline, 6 and 12 months | 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 |
| Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Baseline, 6 and 12 months | 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 |
Countries
Austria, Greece, Italy
Participant flow
Pre-assignment details
All patients completed study CICL670F2201 (NCT02125877) prior to entry into this study
Participants by arm
| Arm | Count |
|---|---|
| Deferasirox 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. | 53 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | PR - abnormal lab value(s) | 1 |
| Overall Study | PR - adverse event | 1 |
| Overall Study | PR - death | 1 |
| Overall Study | Primary reason (PR) - subject withdrawal | 11 |
| Overall Study | PR - pregnancy | 2 |
| Overall Study | PR - unsatisfactory therapeutic effect | 2 |
| Overall Study | PR- unwilling to comply with procedures | 1 |
Baseline characteristics
| Characteristic | Deferasirox |
|---|---|
| Age, Customized <18 | 3 Participants |
| Age, Customized ≥18 - <50 | 46 Participants |
| Age, Customized ≥ 50 - <65 | 1 Participants |
| Age, Customized ≥ 65 | 3 Participants |
| Main underlying disease MDS with INT risk as per the IPSS - R | 1 participants |
| Main underlying disease MDS with low risk as per the IPSS - R | 1 participants |
| Main underlying disease MDS with very low risk as per the IPSS - R | 2 participants |
| Main underlying disease Transfusion-dependent thalassemia | 49 participants |
| Race/Ethnicity, Customized Asian | 1 Participants |
| Race/Ethnicity, Customized Caucasian | 50 Participants |
| Race/Ethnicity, Customized Other | 2 Participants |
| Sex: Female, Male Female | 35 Participants |
| Sex: Female, Male Male | 18 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 53 |
| other Total, other adverse events | 48 / 53 |
| serious Total, serious adverse events | 13 / 53 |
Outcome results
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Post | NA U/L | — |
| Deferasirox | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Baseline | 37.4 U/L | Standard Deviation 31.47 |
| Deferasirox | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Change | NA U/L | — |
| Month 6 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Post | 28.9 U/L | Standard Deviation 21.12 |
| Month 6 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Baseline | 39.4 U/L | Standard Deviation 33.51 |
| Month 6 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Change | -10.5 U/L | Standard Deviation 29.16 |
| Month 12 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Baseline | 39.5 U/L | Standard Deviation 33.32 |
| Month 12 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Change | -13.6 U/L | Standard Deviation 27.44 |
| Month 12 | Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12 | Post | 25.9 U/L | Standard Deviation 19.08 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Post | NA U/L | — |
| Deferasirox | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Baseline | 30.7 U/L | Standard Deviation 24.4 |
| Deferasirox | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Change | NA U/L | — |
| Month 6 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Post | 27.6 U/L | Standard Deviation 18.81 |
| Month 6 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Baseline | 32.9 U/L | Standard Deviation 26.48 |
| Month 6 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Change | -5.3 U/L | Standard Deviation 21.03 |
| Month 12 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Baseline | 33.7 U/L | Standard Deviation 26.67 |
| Month 12 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Change | -8.5 U/L | Standard Deviation 19.18 |
| Month 12 | Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12 | Post | 25.2 U/L | Standard Deviation 12.78 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Change | NA mL/min | — |
| Deferasirox | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Post | NA mL/min | — |
| Deferasirox | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Baseline | 131.9 mL/min | Standard Deviation 51.32 |
| Month 6 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Change | -15.8 mL/min | Standard Deviation 35.12 |
| Month 6 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Baseline | 131.8 mL/min | Standard Deviation 56.98 |
| Month 6 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Post | 116.0 mL/min | Standard Deviation 48.71 |
| Month 12 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Post | 119.8 mL/min | Standard Deviation 49.34 |
| Month 12 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Baseline | 129.6 mL/min | Standard Deviation 50.3 |
| Month 12 | Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12 | Change | -9.8 mL/min | Standard Deviation 32.74 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Post | NA 10^9 cells/L | — |
| Deferasirox | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 330.1 10^9 cells/L | Standard Deviation 188.81 |
| Deferasirox | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Change | NA 10^9 cells/L | — |
| Month 6 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Post | 344.2 10^9 cells/L | Standard Deviation 190.43 |
| Month 6 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 336.5 10^9 cells/L | Standard Deviation 195.97 |
| Month 6 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Change | 7.7 10^9 cells/L | Standard Deviation 112.98 |
| Month 12 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 339.4 10^9 cells/L | Standard Deviation 196.41 |
| Month 12 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Change | 22.4 10^9 cells/L | Standard Deviation 100.72 |
| Month 12 | Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12 | Post | 361.9 10^9 cells/L | Standard Deviation 165.91 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Post | NA 10^12 cells/L | — |
| Deferasirox | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Baseline | 3.753 10^12 cells/L | Standard Deviation 0.4909 |
| Deferasirox | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Change | NA 10^12 cells/L | — |
| Month 6 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Post | 3.610 10^12 cells/L | Standard Deviation 0.5153 |
| Month 6 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Baseline | 3.733 10^12 cells/L | Standard Deviation 0.5016 |
| Month 6 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Change | -0.124 10^12 cells/L | Standard Deviation 0.3892 |
| Month 12 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Baseline | 3.746 10^12 cells/L | Standard Deviation 0.5071 |
| Month 12 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Change | -0.139 10^12 cells/L | Standard Deviation 0.433 |
| Month 12 | Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12 | Post | 3.607 10^12 cells/L | Standard Deviation 0.5423 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Post | NA umol/L | — |
| Deferasirox | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Baseline | 55.1 umol/L | Standard Deviation 16.16 |
| Deferasirox | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Change | NA umol/L | — |
| Month 6 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Post | 62.0 umol/L | Standard Deviation 16.17 |
| Month 6 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Baseline | 54.4 umol/L | Standard Deviation 16.34 |
| Month 6 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Change | 7.6 umol/L | Standard Deviation 9.51 |
| Month 12 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Baseline | 57.4 umol/L | Standard Deviation 16.91 |
| Month 12 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Change | 6.1 umol/L | Standard Deviation 11.09 |
| Month 12 | Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12 | Post | 63.5 umol/L | Standard Deviation 16.78 |
Primary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Post | NA 10^9 cells/L | — |
| Deferasirox | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 9.336 10^9 cells/L | Standard Deviation 5.1383 |
| Deferasirox | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Change | NA 10^9 cells/L | — |
| Month 6 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Post | 9.090 10^9 cells/L | Standard Deviation 4.3712 |
| Month 6 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 9.559 10^9 cells/L | Standard Deviation 5.665 |
| Month 6 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Change | -0.469 10^9 cells/L | Standard Deviation 4.0727 |
| Month 12 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Baseline | 9.100 10^9 cells/L | Standard Deviation 5.4667 |
| Month 12 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Change | -0.093 10^9 cells/L | Standard Deviation 4.5792 |
| Month 12 | Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12 | Post | 9.007 10^9 cells/L | Standard Deviation 4.4385 |
Primary
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
Time frame: Baseline up to approximately 25 months
Population: Safety analysis set
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox | Overview of Number of Participants With Adverse Events | Adverse events (AEs) | 52 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Treatment related AEs | 20 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Severe adverse events | 14 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Treatment related severe adverse events | 2 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Serious adverse events (SAEs) | 13 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Treatment related SAEs | 0 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Fatal SAEs | 1 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Treatment related fatal SAEs | 0 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | AEs leading to discontinuation | 4 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | Treatment related AEs leading to discontinuation | 2 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | AEs leading to dose adjust/interruption | 33 number of participants |
| Deferasirox | Overview of Number of Participants With Adverse Events | AEs requiring additional therapy | 4 number of participants |
Secondary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Change | NA ug/l | — |
| Deferasirox | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Post | NA ug/l | — |
| Deferasirox | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Baseline | 2523.51 ug/l | Standard Deviation 1746.087 |
| Month 6 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Change | -385.18 ug/l | Standard Deviation 1038.789 |
| Month 6 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Baseline | 2614.12 ug/l | Standard Deviation 1781.287 |
| Month 6 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Post | 2228.94 ug/l | Standard Deviation 1910.182 |
| Month 12 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Post | 1924.49 ug/l | Standard Deviation 1839.818 |
| Month 12 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Baseline | 2542.76 ug/l | Standard Deviation 1904.087 |
| Month 12 | Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12 | Change | -618.26 ug/l | Standard Deviation 1054.15 |
Secondary
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
Time frame: Baseline, 6 and 12 months
Population: Safety analysis set - at each timepoint the subjects from the safety analysis set who have a value of the lab parameter of interest at both baseline and the timepoint of interest
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Deferasirox | Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | NA percentage of relative change | — |
| Month 6 | Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | -18.61 percentage of relative change | Standard Deviation 32.969 |
| Month 12 | Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12 | -29.08 percentage of relative change | Standard Deviation 33.056 |