Trial Outcomes & Findings for Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients (NCT NCT00303329)
NCT ID: NCT00303329
Last Updated: 2011-05-09
Results Overview
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
184 participants
Primary outcome timeframe
Core study Baseline to the end of the study (up to 60 months)
Results posted on
2011-05-09
Participant Flow
Participant milestones
| Measure |
β-thalassemia Patients
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
Overall Study
STARTED
|
85
|
99
|
|
Overall Study
COMPLETED
|
50
|
37
|
|
Overall Study
NOT COMPLETED
|
35
|
62
|
Reasons for withdrawal
| Measure |
β-thalassemia Patients
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
Overall Study
Adverse Event
|
7
|
14
|
|
Overall Study
Abnormal Laboratory Values
|
0
|
1
|
|
Overall Study
Unsatisfactory therapeutic effect
|
9
|
2
|
|
Overall Study
Condition no longer requires study drug
|
0
|
8
|
|
Overall Study
Protocol Violation
|
3
|
0
|
|
Overall Study
Withdrawal by Subject
|
9
|
15
|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
|
Overall Study
Administrative problems
|
1
|
0
|
|
Overall Study
Death
|
2
|
12
|
|
Overall Study
Stopped at end of core
|
3
|
9
|
|
Overall Study
Stopped at end of extension 1
|
1
|
0
|
Baseline Characteristics
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
Baseline characteristics by cohort
| Measure |
β-thalassemia Patients
n=85 Participants
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
n=99 Participants
Deferasirox (5-40 mg/kg/day)
|
Total
n=184 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Region of Enrollment
Germany
|
2 participants
n=5 Participants
|
23 participants
n=7 Participants
|
25 participants
n=5 Participants
|
|
Age Continuous
|
24.7 years
STANDARD_DEVIATION 10.03 • n=5 Participants
|
43.7 years
STANDARD_DEVIATION 26.13 • n=7 Participants
|
35.0 years
STANDARD_DEVIATION 22.4 • n=5 Participants
|
|
Age, Customized
<6 years
|
2 participants
n=5 Participants
|
9 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Age, Customized
Between 6 and 11 years
|
5 participants
n=5 Participants
|
6 participants
n=7 Participants
|
11 participants
n=5 Participants
|
|
Age, Customized
Between 12 and 15 years
|
8 participants
n=5 Participants
|
5 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Age, Customized
Between 16 and 49 years
|
69 participants
n=5 Participants
|
30 participants
n=7 Participants
|
99 participants
n=5 Participants
|
|
Age, Customized
Between 50 and 64 years
|
1 participants
n=5 Participants
|
19 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Age, Customized
>=65 years
|
0 participants
n=5 Participants
|
30 participants
n=7 Participants
|
30 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
43 Participants
n=5 Participants
|
48 Participants
n=7 Participants
|
91 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
42 Participants
n=5 Participants
|
51 Participants
n=7 Participants
|
93 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
21 participants
n=7 Participants
|
36 participants
n=5 Participants
|
|
Region of Enrollment
France
|
9 participants
n=5 Participants
|
11 participants
n=7 Participants
|
20 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
12 participants
n=5 Participants
|
6 participants
n=7 Participants
|
18 participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
4 participants
n=5 Participants
|
12 participants
n=7 Participants
|
16 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
12 participants
n=5 Participants
|
0 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
31 participants
n=5 Participants
|
26 participants
n=7 Participants
|
57 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Core study Baseline to the end of the study (up to 60 months)Population: The safety analysis set comprised all participants who received at least one dose of deferasirox during either the core or extension studies.
Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.
Outcome measures
| Measure |
β-thalassemia Patients
n=85 Participants
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
n=99 Participants
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Adverse Events
|
85 Participants
|
99 Participants
|
|
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Serious Adverse Events
|
36 Participants
|
50 Participants
|
|
The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
Deaths
|
2 Participants
|
12 Participants
|
SECONDARY outcome
Timeframe: Core study Baseline to end of extension study (up to 60 months)Population: The full analysis Set (FAS) comprised all participants who received at least one dose of deferasirox during either the core or extension studies.
Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant.
Outcome measures
| Measure |
β-thalassemia Patients
n=55 Participants
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
n=42 Participants
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
|
-5.17 mg Fe/g dw
Standard Deviation 11.659
|
-5.10 mg Fe/g dw
Standard Deviation 7.756
|
SECONDARY outcome
Timeframe: Core study Baseline to end of extension study (up to 60 months)Population: The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies.
Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study.
Outcome measures
| Measure |
β-thalassemia Patients
n=15 Participants
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
n=33 Participants
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
|
0.19 mg Fe/g dw
Standard Deviation 8.577
|
-2.04 mg Fe/g dw
Standard Deviation 4.521
|
SECONDARY outcome
Timeframe: Core study Baseline to end of extension study (up to 60 months)Population: The FAS comprised all participants who received at least one dose of deferasirox during either the core or extension studies.
Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered.
Outcome measures
| Measure |
β-thalassemia Patients
n=85 Participants
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias Patients
n=96 Participants
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Baseline
|
4320.6 μg/L
Standard Deviation 2881.08
|
3268.8 μg/L
Standard Deviation 2082.60
|
|
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
End of Study
|
3708.2 μg/L
Standard Deviation 3018.10
|
2896.0 μg/L
Standard Deviation 2597.22
|
|
The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
Absolute Change
|
-612.4 μg/L
Standard Deviation 2520.87
|
-382.2 μg/L
Standard Deviation 2325.41
|
Adverse Events
Beta-thalassemia
Serious events: 36 serious events
Other events: 84 other events
Deaths: 0 deaths
Rare Anemias
Serious events: 50 serious events
Other events: 96 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Beta-thalassemia
n=85 participants at risk
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias
n=99 participants at risk
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/85
|
5.1%
5/99
|
|
Blood and lymphatic system disorders
Disseminated intravascular coagulation
|
0.00%
0/85
|
1.0%
1/99
|
|
Blood and lymphatic system disorders
Extramedullary haemopoiesis
|
1.2%
1/85
|
0.00%
0/99
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/85
|
2.0%
2/99
|
|
Blood and lymphatic system disorders
Haemolysis
|
0.00%
0/85
|
1.0%
1/99
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
2.4%
2/85
|
0.00%
0/99
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/85
|
2.0%
2/99
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/85
|
1.0%
1/99
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Angina pectoris
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Arrhythmia
|
1.2%
1/85
|
1.0%
1/99
|
|
Cardiac disorders
Atrial fibrillation
|
3.5%
3/85
|
1.0%
1/99
|
|
Cardiac disorders
Cardiac arrest
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Cardiac disorder
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Cardiac failure
|
1.2%
1/85
|
0.00%
0/99
|
|
Cardiac disorders
Cardiac failure congestive
|
2.4%
2/85
|
0.00%
0/99
|
|
Cardiac disorders
Cardio-respiratory arrest
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Cardiogenic shock
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Cardiomyopathy
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Left ventricular failure
|
1.2%
1/85
|
0.00%
0/99
|
|
Cardiac disorders
Mitral valve incompetence
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Palpitations
|
1.2%
1/85
|
0.00%
0/99
|
|
Cardiac disorders
Tachyarrhythmia
|
1.2%
1/85
|
0.00%
0/99
|
|
Cardiac disorders
Ventricular fibrillation
|
0.00%
0/85
|
1.0%
1/99
|
|
Cardiac disorders
Ventricular tachyarrhythmia
|
1.2%
1/85
|
0.00%
0/99
|
|
Ear and labyrinth disorders
Sudden hearing loss
|
0.00%
0/85
|
1.0%
1/99
|
|
Ear and labyrinth disorders
Tympanic membrane perforation
|
0.00%
0/85
|
1.0%
1/99
|
|
Endocrine disorders
Primary hypogonadism
|
0.00%
0/85
|
1.0%
1/99
|
|
Eye disorders
Cataract
|
0.00%
0/85
|
2.0%
2/99
|
|
Gastrointestinal disorders
Abdominal pain
|
3.5%
3/85
|
4.0%
4/99
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/85
|
2.0%
2/99
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
0.00%
0/85
|
2.0%
2/99
|
|
Gastrointestinal disorders
Ileus
|
0.00%
0/85
|
1.0%
1/99
|
|
Gastrointestinal disorders
Melaena
|
1.2%
1/85
|
1.0%
1/99
|
|
Gastrointestinal disorders
Nausea
|
2.4%
2/85
|
0.00%
0/99
|
|
Gastrointestinal disorders
Varices oesophageal
|
0.00%
0/85
|
1.0%
1/99
|
|
Gastrointestinal disorders
Vomiting
|
3.5%
3/85
|
1.0%
1/99
|
|
General disorders
Catheter related complication
|
0.00%
0/85
|
1.0%
1/99
|
|
General disorders
Chest pain
|
2.4%
2/85
|
1.0%
1/99
|
|
General disorders
Chills
|
0.00%
0/85
|
2.0%
2/99
|
|
General disorders
Drug withdrawal syndrome
|
1.2%
1/85
|
0.00%
0/99
|
|
General disorders
General physical health deterioration
|
0.00%
0/85
|
1.0%
1/99
|
|
General disorders
Hyperpyrexia
|
1.2%
1/85
|
0.00%
0/99
|
|
General disorders
Malaise
|
0.00%
0/85
|
1.0%
1/99
|
|
General disorders
Oedema
|
1.2%
1/85
|
0.00%
0/99
|
|
General disorders
Pyrexia
|
3.5%
3/85
|
12.1%
12/99
|
|
Hepatobiliary disorders
Bile duct stone
|
1.2%
1/85
|
0.00%
0/99
|
|
Hepatobiliary disorders
Cholelithiasis
|
1.2%
1/85
|
0.00%
0/99
|
|
Hepatobiliary disorders
Cholelithiasis obstructive
|
1.2%
1/85
|
0.00%
0/99
|
|
Hepatobiliary disorders
Gallbladder disorder
|
1.2%
1/85
|
0.00%
0/99
|
|
Hepatobiliary disorders
Hepatic failure
|
1.2%
1/85
|
0.00%
0/99
|
|
Hepatobiliary disorders
Hepatic haemorrhage
|
0.00%
0/85
|
1.0%
1/99
|
|
Hepatobiliary disorders
Hepatitis
|
0.00%
0/85
|
2.0%
2/99
|
|
Infections and infestations
Abdominal infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Anal abscess
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Arthritis infective
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Bacterial sepsis
|
0.00%
0/85
|
2.0%
2/99
|
|
Infections and infestations
Bronchiolitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Bronchitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Bronchopulmonary aspergillosis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Catheter related infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Erysipelas
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Gastroenteritis
|
2.4%
2/85
|
1.0%
1/99
|
|
Infections and infestations
Gastroenteritis yersinia
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Haematoma infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Hepatitis A
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Influenza
|
1.2%
1/85
|
1.0%
1/99
|
|
Infections and infestations
Klebsiella infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Klebsiella sepsis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Lobar pneumonia
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/85
|
2.0%
2/99
|
|
Infections and infestations
Lung infection
|
0.00%
0/85
|
2.0%
2/99
|
|
Infections and infestations
Meningitis viral
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Neutropenic sepsis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Orchitis
|
1.2%
1/85
|
0.00%
0/99
|
|
Infections and infestations
Peridiverticular abscess
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Pharyngitis streptococcal
|
1.2%
1/85
|
0.00%
0/99
|
|
Infections and infestations
Pneumonia
|
0.00%
0/85
|
5.1%
5/99
|
|
Infections and infestations
Pseudomonal sepsis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Pyelonephritis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Sepsis
|
0.00%
0/85
|
5.1%
5/99
|
|
Infections and infestations
Septic shock
|
0.00%
0/85
|
3.0%
3/99
|
|
Infections and infestations
Staphylococcal sepsis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Subcutaneous abscess
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Urosepsis
|
0.00%
0/85
|
1.0%
1/99
|
|
Infections and infestations
Viral infection
|
1.2%
1/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Contusion
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Device failure
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Fall
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Fibula fracture
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Forearm fracture
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Head injury
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Hepatic haematoma
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Radius fracture
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Skin laceration
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Spinal fracture
|
1.2%
1/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Splenic rupture
|
1.2%
1/85
|
0.00%
0/99
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.00%
0/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
0.00%
0/85
|
1.0%
1/99
|
|
Investigations
Blood phosphorus increased
|
0.00%
0/85
|
1.0%
1/99
|
|
Investigations
Electrocardiogram abnormal
|
0.00%
0/85
|
1.0%
1/99
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/85
|
1.0%
1/99
|
|
Investigations
Transaminases increased
|
1.2%
1/85
|
2.0%
2/99
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/85
|
2.0%
2/99
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/85
|
1.0%
1/99
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
1.2%
1/85
|
0.00%
0/99
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
1.2%
1/85
|
0.00%
0/99
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/85
|
1.0%
1/99
|
|
Metabolism and nutrition disorders
Hypoglycaemic seizure
|
0.00%
0/85
|
1.0%
1/99
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/85
|
1.0%
1/99
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
0.00%
0/85
|
1.0%
1/99
|
|
Metabolism and nutrition disorders
Type 1 diabetes mellitus
|
1.2%
1/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.2%
1/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.2%
1/85
|
1.0%
1/99
|
|
Musculoskeletal and connective tissue disorders
Bone cyst
|
1.2%
1/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Knee deformity
|
2.4%
2/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
1.2%
1/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/85
|
1.0%
1/99
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
1.2%
1/85
|
0.00%
0/99
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
0.00%
0/85
|
1.0%
1/99
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
1.2%
1/85
|
0.00%
0/99
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Acute leukaemia
|
0.00%
0/85
|
2.0%
2/99
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Myelodysplastic syndrome
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Cerebral haemorrhage
|
0.00%
0/85
|
2.0%
2/99
|
|
Nervous system disorders
Coma
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Dementia
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Dizziness
|
1.2%
1/85
|
0.00%
0/99
|
|
Nervous system disorders
Encephalitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Headache
|
1.2%
1/85
|
0.00%
0/99
|
|
Nervous system disorders
Hypokinesia
|
1.2%
1/85
|
0.00%
0/99
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Migraine
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Psychomotor hyperactivity
|
0.00%
0/85
|
1.0%
1/99
|
|
Nervous system disorders
Sciatica
|
1.2%
1/85
|
0.00%
0/99
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
1.2%
1/85
|
0.00%
0/99
|
|
Psychiatric disorders
Adjustment disorder
|
0.00%
0/85
|
1.0%
1/99
|
|
Psychiatric disorders
Anxiety
|
2.4%
2/85
|
0.00%
0/99
|
|
Psychiatric disorders
Delirium
|
1.2%
1/85
|
0.00%
0/99
|
|
Psychiatric disorders
Depression
|
1.2%
1/85
|
3.0%
3/99
|
|
Psychiatric disorders
Illusion
|
1.2%
1/85
|
0.00%
0/99
|
|
Psychiatric disorders
Mental disorder
|
0.00%
0/85
|
1.0%
1/99
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/85
|
1.0%
1/99
|
|
Psychiatric disorders
Suicide attempt
|
0.00%
0/85
|
1.0%
1/99
|
|
Renal and urinary disorders
Anuria
|
1.2%
1/85
|
0.00%
0/99
|
|
Renal and urinary disorders
Calculus bladder
|
1.2%
1/85
|
0.00%
0/99
|
|
Renal and urinary disorders
Calculus ureteric
|
0.00%
0/85
|
1.0%
1/99
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/85
|
1.0%
1/99
|
|
Renal and urinary disorders
Renal colic
|
1.2%
1/85
|
0.00%
0/99
|
|
Renal and urinary disorders
Renal mass
|
1.2%
1/85
|
0.00%
0/99
|
|
Renal and urinary disorders
Urethral haemorrhage
|
0.00%
0/85
|
1.0%
1/99
|
|
Reproductive system and breast disorders
Testicular disorder
|
0.00%
0/85
|
1.0%
1/99
|
|
Reproductive system and breast disorders
Testicular torsion
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/85
|
2.0%
2/99
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
1.2%
1/85
|
0.00%
0/99
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.2%
1/85
|
0.00%
0/99
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal haemorrhage
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Pleurisy
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
1.2%
1/85
|
0.00%
0/99
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.2%
1/85
|
0.00%
0/99
|
|
Skin and subcutaneous tissue disorders
Stasis dermatitis
|
0.00%
0/85
|
1.0%
1/99
|
|
Surgical and medical procedures
Toe amputation
|
0.00%
0/85
|
1.0%
1/99
|
|
Vascular disorders
Hypotension
|
1.2%
1/85
|
1.0%
1/99
|
|
Vascular disorders
Hypovolaemic shock
|
0.00%
0/85
|
1.0%
1/99
|
|
Vascular disorders
Poor venous access
|
0.00%
0/85
|
1.0%
1/99
|
Other adverse events
| Measure |
Beta-thalassemia
n=85 participants at risk
Deferasirox (5-40 mg/kg/day)
|
Rare Anemias
n=99 participants at risk
Deferasirox (5-40 mg/kg/day)
|
|---|---|---|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
5.9%
5/85
|
1.0%
1/99
|
|
Cardiac disorders
Palpitations
|
14.1%
12/85
|
8.1%
8/99
|
|
Cardiac disorders
Tachycardia
|
14.1%
12/85
|
6.1%
6/99
|
|
Ear and labyrinth disorders
Ear pain
|
11.8%
10/85
|
6.1%
6/99
|
|
Ear and labyrinth disorders
Tinnitus
|
7.1%
6/85
|
0.00%
0/99
|
|
Ear and labyrinth disorders
Vertigo
|
7.1%
6/85
|
6.1%
6/99
|
|
Eye disorders
Vision blurred
|
5.9%
5/85
|
1.0%
1/99
|
|
Gastrointestinal disorders
Abdominal discomfort
|
8.2%
7/85
|
3.0%
3/99
|
|
Gastrointestinal disorders
Abdominal distension
|
11.8%
10/85
|
5.1%
5/99
|
|
Gastrointestinal disorders
Abdominal pain
|
37.6%
32/85
|
31.3%
31/99
|
|
Gastrointestinal disorders
Abdominal pain upper
|
35.3%
30/85
|
13.1%
13/99
|
|
Gastrointestinal disorders
Constipation
|
27.1%
23/85
|
14.1%
14/99
|
|
Gastrointestinal disorders
Diarrhoea
|
47.1%
40/85
|
50.5%
50/99
|
|
Gastrointestinal disorders
Dyspepsia
|
10.6%
9/85
|
7.1%
7/99
|
|
Gastrointestinal disorders
Gastritis
|
1.2%
1/85
|
7.1%
7/99
|
|
Gastrointestinal disorders
Gingival bleeding
|
1.2%
1/85
|
5.1%
5/99
|
|
Gastrointestinal disorders
Nausea
|
42.4%
36/85
|
34.3%
34/99
|
|
Gastrointestinal disorders
Toothache
|
14.1%
12/85
|
3.0%
3/99
|
|
Gastrointestinal disorders
Vomiting
|
27.1%
23/85
|
36.4%
36/99
|
|
General disorders
Asthenia
|
16.5%
14/85
|
19.2%
19/99
|
|
General disorders
Chest pain
|
22.4%
19/85
|
9.1%
9/99
|
|
General disorders
Fatigue
|
20.0%
17/85
|
18.2%
18/99
|
|
General disorders
Influenza like illness
|
10.6%
9/85
|
4.0%
4/99
|
|
General disorders
Malaise
|
5.9%
5/85
|
4.0%
4/99
|
|
General disorders
Oedema
|
2.4%
2/85
|
8.1%
8/99
|
|
General disorders
Oedema peripheral
|
8.2%
7/85
|
12.1%
12/99
|
|
General disorders
Pyrexia
|
45.9%
39/85
|
36.4%
36/99
|
|
Hepatobiliary disorders
Hepatomegaly
|
4.7%
4/85
|
5.1%
5/99
|
|
Immune system disorders
Seasonal allergy
|
9.4%
8/85
|
6.1%
6/99
|
|
Infections and infestations
Bronchitis
|
10.6%
9/85
|
20.2%
20/99
|
|
Infections and infestations
Ear infection
|
8.2%
7/85
|
3.0%
3/99
|
|
Infections and infestations
Gastroenteritis
|
16.5%
14/85
|
17.2%
17/99
|
|
Infections and infestations
Influenza
|
16.5%
14/85
|
15.2%
15/99
|
|
Infections and infestations
Nasopharyngitis
|
38.8%
33/85
|
26.3%
26/99
|
|
Infections and infestations
Oral herpes
|
3.5%
3/85
|
8.1%
8/99
|
|
Infections and infestations
Otitis media
|
3.5%
3/85
|
6.1%
6/99
|
|
Infections and infestations
Pharyngitis
|
30.6%
26/85
|
4.0%
4/99
|
|
Infections and infestations
Rhinitis
|
24.7%
21/85
|
12.1%
12/99
|
|
Infections and infestations
Sinusitis
|
3.5%
3/85
|
6.1%
6/99
|
|
Infections and infestations
Tonsillitis
|
10.6%
9/85
|
8.1%
8/99
|
|
Infections and infestations
Tooth abscess
|
8.2%
7/85
|
1.0%
1/99
|
|
Infections and infestations
Tooth infection
|
8.2%
7/85
|
2.0%
2/99
|
|
Infections and infestations
Upper respiratory tract infection
|
27.1%
23/85
|
16.2%
16/99
|
|
Infections and infestations
Urinary tract infection
|
11.8%
10/85
|
10.1%
10/99
|
|
Infections and infestations
Viral infection
|
5.9%
5/85
|
2.0%
2/99
|
|
Infections and infestations
Viral upper respiratory tract infection
|
11.8%
10/85
|
1.0%
1/99
|
|
Injury, poisoning and procedural complications
Fall
|
5.9%
5/85
|
3.0%
3/99
|
|
Injury, poisoning and procedural complications
Procedural pain
|
16.5%
14/85
|
3.0%
3/99
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
15.3%
13/85
|
13.1%
13/99
|
|
Investigations
Blood creatinine increased
|
18.8%
16/85
|
19.2%
19/99
|
|
Investigations
Cardiac murmur
|
5.9%
5/85
|
5.1%
5/99
|
|
Investigations
Transaminases increased
|
3.5%
3/85
|
5.1%
5/99
|
|
Investigations
Weight decreased
|
2.4%
2/85
|
5.1%
5/99
|
|
Metabolism and nutrition disorders
Anorexia
|
8.2%
7/85
|
7.1%
7/99
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
21.2%
18/85
|
12.1%
12/99
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
37.6%
32/85
|
20.2%
20/99
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
9.4%
8/85
|
10.1%
10/99
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
11.8%
10/85
|
9.1%
9/99
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
10.6%
9/85
|
2.0%
2/99
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
9.4%
8/85
|
2.0%
2/99
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
20.0%
17/85
|
9.1%
9/99
|
|
Nervous system disorders
Dizziness
|
5.9%
5/85
|
8.1%
8/99
|
|
Nervous system disorders
Headache
|
52.9%
45/85
|
34.3%
34/99
|
|
Nervous system disorders
Paraesthesia
|
5.9%
5/85
|
1.0%
1/99
|
|
Psychiatric disorders
Anxiety
|
7.1%
6/85
|
4.0%
4/99
|
|
Psychiatric disorders
Depression
|
12.9%
11/85
|
8.1%
8/99
|
|
Psychiatric disorders
Insomnia
|
9.4%
8/85
|
7.1%
7/99
|
|
Renal and urinary disorders
Dysuria
|
8.2%
7/85
|
3.0%
3/99
|
|
Renal and urinary disorders
Proteinuria
|
5.9%
5/85
|
3.0%
3/99
|
|
Renal and urinary disorders
Renal failure
|
0.00%
0/85
|
6.1%
6/99
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
7.1%
6/85
|
2.0%
2/99
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
42.4%
36/85
|
34.3%
34/99
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
11.8%
10/85
|
12.1%
12/99
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.4%
2/85
|
10.1%
10/99
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.6%
9/85
|
3.0%
3/99
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
35.3%
30/85
|
15.2%
15/99
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
12.9%
11/85
|
1.0%
1/99
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
7.1%
6/85
|
3.0%
3/99
|
|
Skin and subcutaneous tissue disorders
Erythema
|
5.9%
5/85
|
3.0%
3/99
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
8.2%
7/85
|
9.1%
9/99
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.5%
14/85
|
13.1%
13/99
|
|
Skin and subcutaneous tissue disorders
Rash pruritic
|
8.2%
7/85
|
1.0%
1/99
|
|
Vascular disorders
Haematoma
|
1.2%
1/85
|
5.1%
5/99
|
|
Vascular disorders
Hypertension
|
1.2%
1/85
|
7.1%
7/99
|
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