Trial Outcomes & Findings for Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload (NCT NCT00235391)
NCT ID: NCT00235391
Last Updated: 2011-06-07
Results Overview
Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
1683 participants
Primary outcome timeframe
Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)
Results posted on
2011-06-07
Participant Flow
Participant milestones
| Measure |
2 to < 6 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
6 to < 12 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
12 to < 16 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
16 to < 50 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
50 to < 65 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
≥ 65 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
97
|
200
|
172
|
1164
|
43
|
7
|
|
Overall Study
Deferasirox Treatment
|
97
|
200
|
172
|
1164
|
43
|
7
|
|
Overall Study
COMPLETED
|
85
|
182
|
148
|
944
|
31
|
3
|
|
Overall Study
NOT COMPLETED
|
12
|
18
|
24
|
220
|
12
|
4
|
Reasons for withdrawal
| Measure |
2 to < 6 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
6 to < 12 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
12 to < 16 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
16 to < 50 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
50 to < 65 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
≥ 65 Years
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|---|---|---|---|---|
|
Overall Study
Adverse Event
|
4
|
5
|
10
|
72
|
5
|
2
|
|
Overall Study
Abnormal laboratory value(s)
|
3
|
5
|
4
|
27
|
1
|
2
|
|
Overall Study
Unsatisfactory therapeutic effect
|
0
|
2
|
2
|
17
|
0
|
0
|
|
Overall Study
Condition no longer requires treatment
|
3
|
0
|
0
|
11
|
1
|
0
|
|
Overall Study
Protocol deviation
|
0
|
0
|
1
|
11
|
0
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
3
|
3
|
61
|
5
|
0
|
|
Overall Study
Lost to Follow-up
|
0
|
3
|
3
|
16
|
0
|
0
|
|
Overall Study
Administrative problems
|
0
|
0
|
0
|
1
|
0
|
0
|
|
Overall Study
Death
|
0
|
0
|
1
|
4
|
0
|
0
|
Baseline Characteristics
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Baseline characteristics by cohort
| Measure |
2 to < 6 Years
n=97 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
6 to < 12 Years
n=200 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
12 to < 16 Years
n=172 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
16 to < 50 Years
n=1164 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
50 to < 65 Years
n=43 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
≥ 65 Years
n=7 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
Total
n=1683 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|---|---|
|
Age Continuous
|
3.48 years
STANDARD_DEVIATION 1.13 • n=5 Participants
|
8.52 years
STANDARD_DEVIATION 1.64 • n=7 Participants
|
13.48 years
STANDARD_DEVIATION 1.20 • n=5 Participants
|
28.91 years
STANDARD_DEVIATION 8.06 • n=4 Participants
|
54.58 years
STANDARD_DEVIATION 3.92 • n=21 Participants
|
70.14 years
STANDARD_DEVIATION 3.98 • n=8 Participants
|
24.27 years
STANDARD_DEVIATION 12.63 • n=8 Participants
|
|
Sex: Female, Male
Female
|
44 Participants
n=5 Participants
|
101 Participants
n=7 Participants
|
81 Participants
n=5 Participants
|
633 Participants
n=4 Participants
|
32 Participants
n=21 Participants
|
5 Participants
n=8 Participants
|
896 Participants
n=8 Participants
|
|
Sex: Female, Male
Male
|
53 Participants
n=5 Participants
|
99 Participants
n=7 Participants
|
91 Participants
n=5 Participants
|
531 Participants
n=4 Participants
|
11 Participants
n=21 Participants
|
2 Participants
n=8 Participants
|
787 Participants
n=8 Participants
|
|
Baseline Disease Characteristics
Beta-Thalassemia Major
|
74 participants
n=5 Participants
|
118 participants
n=7 Participants
|
114 participants
n=5 Participants
|
905 participants
n=4 Participants
|
10 participants
n=21 Participants
|
0 participants
n=8 Participants
|
1221 participants
n=8 Participants
|
|
Baseline Disease Characteristics
Beta-Thalassemia Intermedia
|
1 participants
n=5 Participants
|
22 participants
n=7 Participants
|
16 participants
n=5 Participants
|
94 participants
n=4 Participants
|
20 participants
n=21 Participants
|
3 participants
n=8 Participants
|
156 participants
n=8 Participants
|
|
Baseline Disease Characteristics
Sickle Cell Disease
|
8 participants
n=5 Participants
|
41 participants
n=7 Participants
|
31 participants
n=5 Participants
|
93 participants
n=4 Participants
|
2 participants
n=21 Participants
|
1 participants
n=8 Participants
|
176 participants
n=8 Participants
|
|
Baseline Disease Characteristics
Diamond-Blackfan Anemia
|
4 participants
n=5 Participants
|
7 participants
n=7 Participants
|
8 participants
n=5 Participants
|
24 participants
n=4 Participants
|
0 participants
n=21 Participants
|
0 participants
n=8 Participants
|
43 participants
n=8 Participants
|
|
Baseline Disease Characteristics
Other Diseases
|
10 participants
n=5 Participants
|
12 participants
n=7 Participants
|
3 participants
n=5 Participants
|
48 participants
n=4 Participants
|
11 participants
n=21 Participants
|
3 participants
n=8 Participants
|
87 participants
n=8 Participants
|
|
Prior Chelation Drug Therapy
Deferoxamine
|
80 participants
n=5 Participants
|
167 participants
n=7 Participants
|
137 participants
n=5 Participants
|
745 participants
n=4 Participants
|
31 participants
n=21 Participants
|
5 participants
n=8 Participants
|
1165 participants
n=8 Participants
|
|
Prior Chelation Drug Therapy
Deferiprone
|
1 participants
n=5 Participants
|
8 participants
n=7 Participants
|
11 participants
n=5 Participants
|
146 participants
n=4 Participants
|
6 participants
n=21 Participants
|
1 participants
n=8 Participants
|
173 participants
n=8 Participants
|
|
Prior Chelation Drug Therapy
Deferoxamine and Deferiprone
|
2 participants
n=5 Participants
|
16 participants
n=7 Participants
|
21 participants
n=5 Participants
|
269 participants
n=4 Participants
|
6 participants
n=21 Participants
|
0 participants
n=8 Participants
|
314 participants
n=8 Participants
|
|
Prior Chelation Drug Therapy
Other Chelation Drug
|
6 participants
n=5 Participants
|
6 participants
n=7 Participants
|
3 participants
n=5 Participants
|
4 participants
n=4 Participants
|
0 participants
n=21 Participants
|
0 participants
n=8 Participants
|
19 participants
n=8 Participants
|
|
Prior Chelation Drug Therapy
Prior Chelatation Drug Information Missing
|
8 participants
n=5 Participants
|
3 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
0 participants
n=21 Participants
|
1 participants
n=8 Participants
|
12 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Therapy non-compliance
|
57 participants
n=5 Participants
|
125 participants
n=7 Participants
|
106 participants
n=5 Participants
|
659 participants
n=4 Participants
|
25 participants
n=21 Participants
|
2 participants
n=8 Participants
|
974 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Therapy contraindication
|
1 participants
n=5 Participants
|
7 participants
n=7 Participants
|
6 participants
n=5 Participants
|
29 participants
n=4 Participants
|
1 participants
n=21 Participants
|
1 participants
n=8 Participants
|
45 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Therapy unacceptable toxicity
|
11 participants
n=5 Participants
|
29 participants
n=7 Participants
|
22 participants
n=5 Participants
|
131 participants
n=4 Participants
|
6 participants
n=21 Participants
|
1 participants
n=8 Participants
|
200 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Therapy poor response
|
20 participants
n=5 Participants
|
34 participants
n=7 Participants
|
38 participants
n=5 Participants
|
267 participants
n=4 Participants
|
8 participants
n=21 Participants
|
0 participants
n=8 Participants
|
367 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Therapy unacceptable discomfort
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
0 participants
n=5 Participants
|
78 participants
n=4 Participants
|
3 participants
n=21 Participants
|
2 participants
n=8 Participants
|
87 participants
n=8 Participants
|
|
Reason for inadequate prior chelation therapy
Reason for inadequate therapy information missing
|
6 participants
n=5 Participants
|
3 participants
n=7 Participants
|
0 participants
n=5 Participants
|
0 participants
n=4 Participants
|
0 participants
n=21 Participants
|
1 participants
n=8 Participants
|
10 participants
n=8 Participants
|
PRIMARY outcome
Timeframe: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)Population: The safety population, comprising all participants who received at least one dose of deferasirox during the study, was used in the analyses.
Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
Outcome measures
| Measure |
2 to < 6 Years
n=97 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
6 to < 12 Years
n=200 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
12 to < 16 Years
n=172 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
16 to < 50 Years
n=1164 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
50 to < 65 Years
n=43 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
≥ 65 Years
n=7 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|---|---|---|---|---|
|
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
AEs leading dose adjustment/temporary interruption
|
15 Participants
|
31 Participants
|
28 Participants
|
209 Participants
|
11 Participants
|
2 Participants
|
|
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Number of deaths
|
0 Participants
|
0 Participants
|
1 Participants
|
4 Participants
|
0 Participants
|
0 Participants
|
|
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Non-fatal SAEs
|
10 Participants
|
22 Participants
|
27 Participants
|
129 Participants
|
4 Participants
|
2 Participants
|
|
Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
AEs leading to discontinuation
|
4 Participants
|
5 Participants
|
10 Participants
|
75 Participants
|
5 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)Population: Safety population defined as all participants who received at least one dose of study drug. This analysis did not include participants with unknown status at baseline and/or at the end of the study.
The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (\<1000), (1000-\<2500), (2500-\<4000), (4000-\<5500), (5500-\<7000) and (\>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Outcome measures
| Measure |
2 to < 6 Years
n=97 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
6 to < 12 Years
n=200 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
12 to < 16 Years
n=172 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
16 to < 50 Years
n=1164 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
50 to < 65 Years
n=43 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
≥ 65 Years
n=7 Participants
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|---|---|---|---|---|
|
The Change in Serum Ferritin Values From Baseline Through Completion of the Study
Improvement
|
17 Participants
|
27 Participants
|
26 Participants
|
206 Participants
|
4 Participants
|
2 Participants
|
|
The Change in Serum Ferritin Values From Baseline Through Completion of the Study
Worsening
|
16 Participants
|
63 Participants
|
55 Participants
|
341 Participants
|
7 Participants
|
1 Participants
|
|
The Change in Serum Ferritin Values From Baseline Through Completion of the Study
No Change
|
63 Participants
|
106 Participants
|
87 Participants
|
570 Participants
|
31 Participants
|
3 Participants
|
Adverse Events
All Participants
Serious events: 194 serious events
Other events: 85 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Participants
n=1683 participants at risk
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|
|
Blood and lymphatic system disorders
Acute chest syndrome
|
0.12%
2/1683
|
|
Blood and lymphatic system disorders
Anaemia
|
0.12%
2/1683
|
|
Blood and lymphatic system disorders
Anaemia haemolytic autoimmune
|
0.12%
2/1683
|
|
Blood and lymphatic system disorders
Coombs positive haemolytic anaemia
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Haemolytic anaemia
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Hypersplenism
|
0.18%
3/1683
|
|
Blood and lymphatic system disorders
Intravascular haemolysis
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Leukopenia
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.12%
2/1683
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Reticulocytopenia
|
0.06%
1/1683
|
|
Blood and lymphatic system disorders
Splenomegaly
|
0.24%
4/1683
|
|
Cardiac disorders
Arrhythmia
|
0.06%
1/1683
|
|
Cardiac disorders
Atrial fibrillation
|
0.12%
2/1683
|
|
Cardiac disorders
Cardiac failure
|
0.36%
6/1683
|
|
Cardiac disorders
Cardiac failure acute
|
0.06%
1/1683
|
|
Cardiac disorders
Cardiac failure congestive
|
0.12%
2/1683
|
|
Cardiac disorders
Congestive cardiomyopathy
|
0.06%
1/1683
|
|
Cardiac disorders
Myopericarditis
|
0.06%
1/1683
|
|
Cardiac disorders
Pericardial effusion
|
0.06%
1/1683
|
|
Cardiac disorders
Tachycardia
|
0.06%
1/1683
|
|
Cardiac disorders
Ventricular dysfunction
|
0.06%
1/1683
|
|
Congenital, familial and genetic disorders
Hip dysplasia
|
0.06%
1/1683
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia
|
0.12%
2/1683
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
|
1.6%
27/1683
|
|
Congenital, familial and genetic disorders
Thalassaemia
|
0.06%
1/1683
|
|
Congenital, familial and genetic disorders
Thalassaemia beta
|
0.12%
2/1683
|
|
Ear and labyrinth disorders
Deafness unilateral
|
0.06%
1/1683
|
|
Ear and labyrinth disorders
Middle ear effusion
|
0.06%
1/1683
|
|
Eye disorders
Retinopathy
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Abdominal pain
|
0.18%
3/1683
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.12%
2/1683
|
|
Gastrointestinal disorders
Colitis
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Constipation
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Crohn's disease
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Diarrhoea
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Gastritis
|
0.24%
4/1683
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Haemorrhoids
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Pancreatitis
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Peritonitis
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Proctitis
|
0.06%
1/1683
|
|
Gastrointestinal disorders
Vomiting
|
0.06%
1/1683
|
|
General disorders
Catheter thrombosis
|
0.06%
1/1683
|
|
General disorders
Death
|
0.06%
1/1683
|
|
General disorders
Local swelling
|
0.06%
1/1683
|
|
General disorders
Malaise
|
0.18%
3/1683
|
|
General disorders
Non-cardiac chest pain
|
0.30%
5/1683
|
|
General disorders
Pain
|
0.18%
3/1683
|
|
General disorders
Pyrexia
|
0.53%
9/1683
|
|
Hepatobiliary disorders
Biliary colic
|
0.06%
1/1683
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.36%
6/1683
|
|
Hepatobiliary disorders
Hepatitis
|
0.12%
2/1683
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.06%
1/1683
|
|
Hepatobiliary disorders
Liver disorder
|
0.06%
1/1683
|
|
Immune system disorders
Hypersensitivity
|
0.06%
1/1683
|
|
Infections and infestations
Abdominal infection
|
0.06%
1/1683
|
|
Infections and infestations
Acute tonsillitis
|
0.12%
2/1683
|
|
Infections and infestations
Appendicitis
|
0.06%
1/1683
|
|
Infections and infestations
Bacterial infection
|
0.06%
1/1683
|
|
Infections and infestations
Bronchitis
|
0.12%
2/1683
|
|
Infections and infestations
Bronchopneumonia
|
0.06%
1/1683
|
|
Infections and infestations
Catheter related infection
|
0.06%
1/1683
|
|
Infections and infestations
Catheter site infection
|
0.06%
1/1683
|
|
Infections and infestations
Cellulitis
|
0.12%
2/1683
|
|
Infections and infestations
Diarrhoea infectious
|
0.06%
1/1683
|
|
Infections and infestations
Ear infection
|
0.06%
1/1683
|
|
Infections and infestations
Epstein-Barr virus infection
|
0.18%
3/1683
|
|
Infections and infestations
Escherichia bacteraemia
|
0.06%
1/1683
|
|
Infections and infestations
Gastroenteritis
|
0.18%
3/1683
|
|
Infections and infestations
Gastrointestinal infection
|
0.06%
1/1683
|
|
Infections and infestations
Hepatitis C
|
0.06%
1/1683
|
|
Infections and infestations
Influenza
|
0.06%
1/1683
|
|
Infections and infestations
Liver abscess
|
0.06%
1/1683
|
|
Infections and infestations
Necrotising fasciitis
|
0.06%
1/1683
|
|
Infections and infestations
Osteomyelitis
|
0.12%
2/1683
|
|
Infections and infestations
Otitis media acute
|
0.06%
1/1683
|
|
Infections and infestations
Parvovirus infection
|
0.06%
1/1683
|
|
Infections and infestations
Perianal abscess
|
0.06%
1/1683
|
|
Infections and infestations
Pharyngitis
|
0.06%
1/1683
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.06%
1/1683
|
|
Infections and infestations
Pneumonia
|
0.24%
4/1683
|
|
Infections and infestations
Pyelonephritis
|
0.06%
1/1683
|
|
Infections and infestations
Respiratory tract infection
|
0.12%
2/1683
|
|
Infections and infestations
Rhinitis
|
0.06%
1/1683
|
|
Infections and infestations
Sepsis
|
0.06%
1/1683
|
|
Infections and infestations
Tonsillitis
|
0.06%
1/1683
|
|
Infections and infestations
Upper respiratory tract infection
|
0.06%
1/1683
|
|
Infections and infestations
Viral infection
|
0.30%
5/1683
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Comminuted fracture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.12%
2/1683
|
|
Injury, poisoning and procedural complications
Forearm fracture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Haemolytic transfusion reaction
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.12%
2/1683
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Suture rupture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.06%
1/1683
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
0.06%
1/1683
|
|
Investigations
Alanine aminotransferase increased
|
0.12%
2/1683
|
|
Investigations
Blood calcium increased
|
0.06%
1/1683
|
|
Investigations
Blood creatinine increased
|
0.06%
1/1683
|
|
Investigations
Cardiovascular evaluation
|
0.06%
1/1683
|
|
Investigations
Protein urine present
|
0.06%
1/1683
|
|
Investigations
Serum ferritin increased
|
0.06%
1/1683
|
|
Investigations
Transaminases increased
|
0.06%
1/1683
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
0.06%
1/1683
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
0.06%
1/1683
|
|
Metabolism and nutrition disorders
Hypoglycaemic seizure
|
0.06%
1/1683
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.06%
1/1683
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.06%
1/1683
|
|
Musculoskeletal and connective tissue disorders
Fistula
|
0.06%
1/1683
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
0.06%
1/1683
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.06%
1/1683
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign neoplasm of thymus
|
0.06%
1/1683
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic neoplasm malignant recurrent
|
0.06%
1/1683
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Non-small cell lung cancer
|
0.06%
1/1683
|
|
Nervous system disorders
Convulsion
|
0.06%
1/1683
|
|
Nervous system disorders
Headache
|
0.12%
2/1683
|
|
Nervous system disorders
Migraine
|
0.06%
1/1683
|
|
Nervous system disorders
Paraesthesia
|
0.06%
1/1683
|
|
Nervous system disorders
Spinal cord compression
|
0.06%
1/1683
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
0.30%
5/1683
|
|
Psychiatric disorders
Anxiety
|
0.06%
1/1683
|
|
Psychiatric disorders
Depression
|
0.06%
1/1683
|
|
Psychiatric disorders
Suicide attempt
|
0.06%
1/1683
|
|
Renal and urinary disorders
Calculus ureteric
|
0.06%
1/1683
|
|
Renal and urinary disorders
Nephrotic syndrome
|
0.06%
1/1683
|
|
Renal and urinary disorders
Renal colic
|
0.12%
2/1683
|
|
Renal and urinary disorders
Renal failure
|
0.12%
2/1683
|
|
Renal and urinary disorders
Urethral stenosis
|
0.06%
1/1683
|
|
Renal and urinary disorders
Urinary retention
|
0.06%
1/1683
|
|
Reproductive system and breast disorders
Haemorrhagic ovarian cyst
|
0.06%
1/1683
|
|
Reproductive system and breast disorders
Uterine disorder
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.06%
1/1683
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.12%
2/1683
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
0.06%
1/1683
|
|
Skin and subcutaneous tissue disorders
Pigmentation disorder
|
0.06%
1/1683
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.30%
5/1683
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.12%
2/1683
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
0.06%
1/1683
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.06%
1/1683
|
|
Surgical and medical procedures
Uterine dilation and curettage
|
0.06%
1/1683
|
|
Vascular disorders
Deep vein thrombosis
|
0.06%
1/1683
|
|
Vascular disorders
Hypotension
|
0.06%
1/1683
|
Other adverse events
| Measure |
All Participants
n=1683 participants at risk
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
5.1%
85/1683
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
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 (i.e., data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER