Trial Outcomes & Findings for Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation (NCT NCT02720536)
NCT ID: NCT02720536
Last Updated: 2020-03-03
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
53 participants
Primary outcome timeframe
Baseline up to approximately 25 months
Results posted on
2020-03-03
Participant Flow
All patients completed study CICL670F2201 (NCT02125877) prior to entry into this study
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
53
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
19
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
|
Overall Study
Primary reason (PR) - subject withdrawal
|
11
|
|
Overall Study
PR - pregnancy
|
2
|
|
Overall Study
PR - unsatisfactory therapeutic effect
|
2
|
|
Overall Study
PR - death
|
1
|
|
Overall Study
PR - adverse event
|
1
|
|
Overall Study
PR - abnormal lab value(s)
|
1
|
|
Overall Study
PR- unwilling to comply with procedures
|
1
|
Baseline Characteristics
Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation
Baseline characteristics by cohort
| Measure |
Deferasirox
n=53 Participants
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.
|
|---|---|
|
Age, Customized
<18
|
3 Participants
n=93 Participants
|
|
Age, Customized
≥18 - <50
|
46 Participants
n=93 Participants
|
|
Age, Customized
≥ 50 - <65
|
1 Participants
n=93 Participants
|
|
Age, Customized
≥ 65
|
3 Participants
n=93 Participants
|
|
Sex: Female, Male
Female
|
35 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
50 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=93 Participants
|
|
Main underlying disease
MDS with very low risk as per the IPSS - R
|
2 participants
n=93 Participants
|
|
Main underlying disease
MDS with low risk as per the IPSS - R
|
1 participants
n=93 Participants
|
|
Main underlying disease
MDS with INT risk as per the IPSS - R
|
1 participants
n=93 Participants
|
|
Main underlying disease
Transfusion-dependent thalassemia
|
49 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: Baseline up to approximately 25 monthsPopulation: Safety analysis set
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
Change from baseline at month 6
|
Month 12
Change from baseline at month 12
|
|---|---|---|---|
|
Overview of Number of Participants With Adverse Events
Treatment related fatal SAEs
|
0 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
AEs leading to discontinuation
|
4 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Treatment related AEs leading to discontinuation
|
2 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
AEs leading to dose adjust/interruption
|
33 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
AEs requiring additional therapy
|
4 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Treatment related AEs
|
20 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Severe adverse events
|
14 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Treatment related severe adverse events
|
2 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Serious adverse events (SAEs)
|
13 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Treatment related SAEs
|
0 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Fatal SAEs
|
1 number of participants
|
—
|
—
|
|
Overview of Number of Participants With Adverse Events
Adverse events (AEs)
|
52 number of participants
|
—
|
—
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=42 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
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
|
3.733 10^12 cells/L
Standard Deviation 0.5016
|
3.746 10^12 cells/L
Standard Deviation 0.5071
|
|
Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12
Post
|
NA 10^12 cells/L
Standard Deviation NA
No post value at baseline
|
3.610 10^12 cells/L
Standard Deviation 0.5153
|
3.607 10^12 cells/L
Standard Deviation 0.5423
|
|
Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12
Change
|
NA 10^12 cells/L
Standard Deviation NA
No post value at baseline
|
-0.124 10^12 cells/L
Standard Deviation 0.3892
|
-0.139 10^12 cells/L
Standard Deviation 0.4330
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=42 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
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
|
9.559 10^9 cells/L
Standard Deviation 5.6650
|
9.100 10^9 cells/L
Standard Deviation 5.4667
|
|
Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12
Post
|
NA 10^9 cells/L
Standard Deviation NA
No post value at baseline
|
9.090 10^9 cells/L
Standard Deviation 4.3712
|
9.007 10^9 cells/L
Standard Deviation 4.4385
|
|
Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12
Change
|
NA 10^9 cells/L
Standard Deviation NA
No post value at baseline
|
-0.469 10^9 cells/L
Standard Deviation 4.0727
|
-0.093 10^9 cells/L
Standard Deviation 4.5792
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=42 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
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
|
336.5 10^9 cells/L
Standard Deviation 195.97
|
339.4 10^9 cells/L
Standard Deviation 196.41
|
|
Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12
Post
|
NA 10^9 cells/L
Standard Deviation NA
No post value at baseline
|
344.2 10^9 cells/L
Standard Deviation 190.43
|
361.9 10^9 cells/L
Standard Deviation 165.91
|
|
Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12
Change
|
NA 10^9 cells/L
Standard Deviation NA
No post value at baseline
|
7.7 10^9 cells/L
Standard Deviation 112.98
|
22.4 10^9 cells/L
Standard Deviation 100.72
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=43 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
Baseline
|
55.1 umol/L
Standard Deviation 16.16
|
54.4 umol/L
Standard Deviation 16.34
|
57.4 umol/L
Standard Deviation 16.91
|
|
Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
Post
|
NA umol/L
Standard Deviation NA
No post value at baseline
|
62.0 umol/L
Standard Deviation 16.17
|
63.5 umol/L
Standard Deviation 16.78
|
|
Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
Change
|
NA umol/L
Standard Deviation NA
No post value at baseline
|
7.6 umol/L
Standard Deviation 9.51
|
6.1 umol/L
Standard Deviation 11.09
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=52 Participants
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
|
Month 6
n=41 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
Baseline
|
131.9 mL/min
Standard Deviation 51.32
|
131.8 mL/min
Standard Deviation 56.98
|
129.6 mL/min
Standard Deviation 50.30
|
|
Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
Post
|
NA mL/min
Standard Deviation NA
No post value at baseline
|
116.0 mL/min
Standard Deviation 48.71
|
119.8 mL/min
Standard Deviation 49.34
|
|
Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
Change
|
NA mL/min
Standard Deviation NA
No post value at baseline
|
-15.8 mL/min
Standard Deviation 35.12
|
-9.8 mL/min
Standard Deviation 32.74
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=44 Participants
Change from baseline at month 6
|
Month 12
n=40 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
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
|
39.4 U/L
Standard Deviation 33.51
|
39.5 U/L
Standard Deviation 33.32
|
|
Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12
Post
|
NA U/L
Standard Deviation NA
No post value at baseline
|
28.9 U/L
Standard Deviation 21.12
|
25.9 U/L
Standard Deviation 19.08
|
|
Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12
Change
|
NA U/L
Standard Deviation NA
No post value at baseline
|
-10.5 U/L
Standard Deviation 29.16
|
-13.6 U/L
Standard Deviation 27.44
|
PRIMARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=43 Participants
Change from baseline at month 6
|
Month 12
n=39 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
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 24.40
|
32.9 U/L
Standard Deviation 26.48
|
33.7 U/L
Standard Deviation 26.67
|
|
Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12
Post
|
NA U/L
Standard Deviation NA
No post value at baseline
|
27.6 U/L
Standard Deviation 18.81
|
25.2 U/L
Standard Deviation 12.78
|
|
Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12
Change
|
NA U/L
Standard Deviation NA
No post value at baseline
|
-5.3 U/L
Standard Deviation 21.03
|
-8.5 U/L
Standard Deviation 19.18
|
SECONDARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=44 Participants
Change from baseline at month 6
|
Month 12
n=36 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
Baseline
|
2523.51 ug/l
Standard Deviation 1746.087
|
2614.12 ug/l
Standard Deviation 1781.287
|
2542.76 ug/l
Standard Deviation 1904.087
|
|
Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
Post
|
NA ug/l
Standard Deviation NA
No values available
|
2228.94 ug/l
Standard Deviation 1910.182
|
1924.49 ug/l
Standard Deviation 1839.818
|
|
Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
Change
|
NA ug/l
Standard Deviation NA
No values available
|
-385.18 ug/l
Standard Deviation 1038.789
|
-618.26 ug/l
Standard Deviation 1054.150
|
SECONDARY outcome
Timeframe: Baseline, 6 and 12 monthsPopulation: 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
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
Outcome measures
| Measure |
Deferasirox
n=53 Participants
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
|
Month 6
n=44 Participants
Change from baseline at month 6
|
Month 12
n=36 Participants
Change from baseline at month 12
|
|---|---|---|---|
|
Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12
|
NA percentage of relative change
Standard Deviation NA
No values available
|
-18.61 percentage of relative change
Standard Deviation 32.969
|
-29.08 percentage of relative change
Standard Deviation 33.056
|
Adverse Events
Deferasirox
Serious events: 13 serious events
Other events: 48 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Deferasirox
n=53 participants at risk
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
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Cardiac disorders
Cardiac failure
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Endocrine disorders
Goitre
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Biliary colic
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Cholecystitis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Cholestasis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Hepatic failure
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Diverticulitis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Lower respiratory tract infection
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Lymph gland infection
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Urosepsis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Injury, poisoning and procedural complications
Femur fracture
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Injury, poisoning and procedural complications
Fracture
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Injury, poisoning and procedural complications
Lumbar vertebral fracture
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Injury, poisoning and procedural complications
Rib fracture
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Injury, poisoning and procedural complications
Ulna fracture
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Ovarian adenoma
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary thyroid cancer
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Product Issues
Device failure
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Psychiatric disorders
Panic attack
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Calculus urinary
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Hydronephrosis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Renal colic
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Ureterolithiasis
|
1.9%
1/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
Other adverse events
| Measure |
Deferasirox
n=53 participants at risk
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
|
|---|---|
|
Cardiac disorders
Palpitations
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain
|
18.9%
10/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Abdominal pain upper
|
20.8%
11/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Constipation
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Diarrhoea
|
26.4%
14/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Dyspepsia
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Gastritis
|
9.4%
5/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Nausea
|
22.6%
12/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Gastrointestinal disorders
Vomiting
|
22.6%
12/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Asthenia
|
18.9%
10/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Influenza like illness
|
9.4%
5/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
General disorders
Pyrexia
|
24.5%
13/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Ear infection
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Gastroenteritis
|
13.2%
7/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Influenza
|
17.0%
9/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Nasopharyngitis
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Pharyngitis
|
13.2%
7/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Respiratory tract infection
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Rhinitis
|
15.1%
8/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Sinusitis
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Tonsillitis
|
9.4%
5/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Upper respiratory tract infection
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Infections and infestations
Urinary tract infection
|
11.3%
6/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Blood creatinine increased
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Investigations
Urine protein/creatinine ratio increased
|
15.1%
8/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
9.4%
5/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
11.3%
6/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
9.4%
5/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Nervous system disorders
Headache
|
26.4%
14/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Dysuria
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Glycosuria
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Proteinuria
|
7.5%
4/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Renal and urinary disorders
Renal colic
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.6%
12/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
17.0%
9/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
|
Vascular disorders
Hypertension
|
5.7%
3/53 • Adverse events were collected from first dose of study treatment until end of study treatment plus 30 days post treatment, up to maximum duration of 25 months
Any sign or symptom that occurs during the study treatment plus the 30 days post treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER