Trial Outcomes & Findings for Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients (NCT NCT00110617)
NCT ID: NCT00110617
Last Updated: 2011-05-26
Results Overview
The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
212 participants
Primary outcome timeframe
24 Weeks
Results posted on
2011-05-26
Participant Flow
212 participants were enrolled in the study; however, 9 participants from Site 512 were excluded due to severe Good Clinical Practice (GCP) violations. 203 participants are included in the Full Analysis Set 1.
Participant milestones
| Measure |
Deferasirox (ICL670)
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
Overall Study
STARTED
|
135
|
68
|
|
Overall Study
Safety Set 1; Received Study Drug
|
135
|
56
|
|
Overall Study
Full Analysis 2; Received ICL670
|
135
|
53
|
|
Overall Study
On Going at Week 24
|
126
|
53
|
|
Overall Study
COMPLETED
|
96
|
39
|
|
Overall Study
NOT COMPLETED
|
39
|
29
|
Reasons for withdrawal
| Measure |
Deferasirox (ICL670)
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
Overall Study
Adverse Event
|
2
|
4
|
|
Overall Study
Abnormal Laboratory Value
|
4
|
2
|
|
Overall Study
Lack of Efficacy
|
1
|
0
|
|
Overall Study
Patient no longer requires study drug
|
1
|
0
|
|
Overall Study
Protocol Violation
|
5
|
0
|
|
Overall Study
Withdrawal by Subject
|
10
|
4
|
|
Overall Study
Lost to Follow-up
|
9
|
1
|
|
Overall Study
Administrative problems
|
6
|
5
|
|
Overall Study
Death
|
1
|
1
|
|
Overall Study
Did not receive study drug
|
0
|
12
|
Baseline Characteristics
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
Baseline characteristics by cohort
| Measure |
Deferasirox (ICL670)
n=135 Participants
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
n=68 Participants
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
Total
n=203 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
16.4 years
STANDARD_DEVIATION 10.31 • n=5 Participants
|
16.2 years
STANDARD_DEVIATION 10.15 • n=7 Participants
|
16.3 years
STANDARD_DEVIATION 10.23 • n=5 Participants
|
|
Age, Customized
2- < 6 Years
|
6 participants
n=5 Participants
|
4 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Age, Customized
6- <12 Years
|
42 participants
n=5 Participants
|
21 participants
n=7 Participants
|
63 participants
n=5 Participants
|
|
Age, Customized
12- <16 Years
|
35 participants
n=5 Participants
|
18 participants
n=7 Participants
|
53 participants
n=5 Participants
|
|
Age, Customized
16- < 50 Years
|
50 participants
n=5 Participants
|
24 participants
n=7 Participants
|
74 participants
n=5 Participants
|
|
Age, Customized
50- <65 Years
|
2 participants
n=5 Participants
|
1 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Age, Customized
= 65 Years
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
56 Participants
n=5 Participants
|
33 Participants
n=7 Participants
|
89 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
79 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
114 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black
|
130 Participants
n=5 Participants
|
65 Participants
n=7 Participants
|
195 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Oriental
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Weight Group
<15 kg
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Weight Group
15- <35 kg
|
39 Participants
n=5 Participants
|
23 Participants
n=7 Participants
|
62 Participants
n=5 Participants
|
|
Weight Group
35- < 55 kg
|
43 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
61 Participants
n=5 Participants
|
|
Weight Group
55- <75 kg
|
42 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
64 Participants
n=5 Participants
|
|
Weight Group
=75 kg
|
10 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Weight Group
Missing
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 WeeksPopulation: Safety-1 set: All participants, except participants enrolled in Center 512, who received at least one dose of study medication during the first 24 weeks.
The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.
Outcome measures
| Measure |
Deferasirox (ICL670)
n=135 Participants
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
n=56 Participants
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Headache
|
30 participants
|
17 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Sickle cell anaemia with crisis
|
30 participants
|
8 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Diarrhoea
|
30 participants
|
5 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Vomiting
|
21 participants
|
9 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Pyrexia
|
19 participants
|
9 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Nausea
|
20 participants
|
4 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Abdominal pain
|
16 participants
|
6 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Upper respiratory tract infection
|
10 participants
|
9 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Rash
|
14 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Cough
|
10 participants
|
7 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Constipation
|
11 participants
|
2 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Pain in Extremity
|
10 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Back Pain
|
8 participants
|
4 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Chest Pain
|
7 participants
|
5 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Oropharyngeal pain
|
7 participants
|
5 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Pruritus
|
7 participants
|
5 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Abdominal pain upper
|
8 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Nasal congestion
|
6 participants
|
4 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Urinary tract infection
|
9 participants
|
0 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Arthralgia
|
7 participants
|
1 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Nasopharyngitis
|
4 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Insomnia
|
3 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Dizziness
|
2 participants
|
3 participants
|
|
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Injection site pain
|
0 participants
|
3 participants
|
SECONDARY outcome
Timeframe: Baseline, 24 WeeksPopulation: Per protocol- 1 defined as all participants who had study drug and had an assessment of serum ferritin at Baseline and at Week 24.
Absolute change from baseline serum ferritin after 24 weeks of treatment with Deferasirox (ICL670) and absolute change from baseline serum ferritin after 24 weeks of treatment with Deferoxamine. Means were adjusted for the amount of transfused blood.
Outcome measures
| Measure |
Deferasirox (ICL670)
n=117 Participants
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
n=50 Participants
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
Absolute Change in Serum Ferritin From Baseline to Week 24
|
-173.2 mg/mL
Interval -691.5 to 345.1
|
-868.7 mg/mL
Interval -1661.9 to 75.5
|
SECONDARY outcome
Timeframe: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 WeeksPopulation: Per Protocol- 2 set defined as all participants who received study drug and had an assessment of serum ferritin at Start of ICL670 treatment and at 24 weeks or 52 weeks.
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 24 and the absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 52 for the Deferasirox treatment group and the Deferoxamine then Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
Outcome measures
| Measure |
Deferasirox (ICL670)
n=113 Participants
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
n=49 Participants
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
24 weeks from ICL670 treatment start (n=111,47)
|
-146.7 mg/mL
Interval -673.7 to 380.2
|
-204.7 mg/mL
Interval -1014.7 to 605.3
|
|
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
52 weeks from ICL670 treatment start (n=113,40)
|
-487.3 mg/mL
Interval -761.6 to -213.0
|
-545.7 mg/mL
Interval -1007.1 to -84.2
|
SECONDARY outcome
Timeframe: Start of Deferasirox (ICL670) treatment, 104 WeeksPopulation: Per Protocol- 2 set defined as all participants who received study drug and had assessment of serum ferritin at Start of ICL670 treatment and at 104 weeks.
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 104 for the Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
Outcome measures
| Measure |
Deferasirox (ICL670)
n=87 Participants
Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks.
|
Deferoxamine (DFO) Then ICL670
Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy.
|
|---|---|---|
|
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104
|
-682.6 mg/mL
Interval -1090.3 to -274.9
|
—
|
Adverse Events
Period 1 Deferasirox (ICL670)
Serious events: 40 serious events
Other events: 91 other events
Deaths: 0 deaths
Period 1 Deferoxamine (DFO)
Serious events: 20 serious events
Other events: 39 other events
Deaths: 0 deaths
Period 2 Deferasirox (ICL670)
Serious events: 78 serious events
Other events: 118 other events
Deaths: 0 deaths
Period 2 Deferoxamine (DFO) Then ICL670
Serious events: 22 serious events
Other events: 44 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Period 1 Deferasirox (ICL670)
n=135 participants at risk
Period 1 (first 24 weeks) Deferasirox (ICL670) 20 mg/kg orally once daily.
|
Period 1 Deferoxamine (DFO)
n=56 participants at risk
Period 1 (first 24 weeks) Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg.
|
Period 2 Deferasirox (ICL670)
n=135 participants at risk
Period 2 (after 24 weeks) Deferasirox (ICL670) 20 mg/kg orally once daily.
|
Period 2 Deferoxamine (DFO) Then ICL670
n=53 participants at risk
Period 2 (After 24 weeks) DFO group cross over to Deferasirox (ICL670) orally 20 mg/kg completing 52 weeks on therapy and then entering an extension period.
|
|---|---|---|---|---|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Investigations
Blood culture positive
|
1.5%
2/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Investigations
Blood pressure diastolic decreased
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
Culture throat positive
|
0.74%
1/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Investigations
Haemoglobin decreased
|
0.00%
0/135
|
1.8%
1/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
Heart rate increased
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
Hepatic enzyme increased
|
1.5%
2/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Investigations
Liver function test abnormal
|
1.5%
2/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Investigations
Oxygen saturation decreased
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Investigations
Serum ferritin increased
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
Urine protein/creatinine ratio increased
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Investigations
White blood cells urine positive
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Metabolism and nutrition disorders
Diabetes mellitus inadequate control
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Haemosiderosis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Hyperglycaemic hyperosmolar nonketotic syndrome
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Type 2 diabetes mellitus
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.74%
1/135
|
1.8%
1/56
|
1.5%
2/135
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.74%
1/135
|
1.8%
1/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
1.5%
2/135
|
0.00%
0/56
|
3.7%
5/135
|
0.00%
0/53
|
|
Nervous system disorders
Altered state of consciousness
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Ataxia
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Balance disorder
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Carotid artery occlusion
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Cerebral ischaemia
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Nervous system disorders
Cerebrovascular accident
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Nervous system disorders
Convulsion
|
0.74%
1/135
|
0.00%
0/56
|
3.0%
4/135
|
0.00%
0/53
|
|
Nervous system disorders
Dysarthria
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Nervous system disorders
Headache
|
1.5%
2/135
|
3.6%
2/56
|
3.7%
5/135
|
1.9%
1/53
|
|
Nervous system disorders
Hypokinesia
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Loss of consciousness
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Migraine
|
1.5%
2/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Nervous system disorders
Moyamoya disease
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Nervous system disorders
Nervous system disorder
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Nervous system disorders
Syncope
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Nervous system disorders
Transient ischaemic attack
|
1.5%
2/135
|
0.00%
0/56
|
1.5%
2/135
|
1.9%
1/53
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Psychiatric disorders
Confusional state
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Psychiatric disorders
Delirium
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Renal and urinary disorders
Haematuria
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Renal and urinary disorders
Renal failure acute
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Renal and urinary disorders
Renal papillary necrosis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/135
|
0.00%
0/56
|
2.2%
3/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.74%
1/135
|
0.00%
0/56
|
2.2%
3/135
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Acute chest syndrome
|
0.00%
0/135
|
3.6%
2/56
|
1.5%
2/135
|
1.9%
1/53
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.74%
1/135
|
0.00%
0/56
|
2.2%
3/135
|
1.9%
1/53
|
|
Cardiac disorders
Cardio-respiratory arrest
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Cardiac disorders
Conduction disorder
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Cardiac disorders
Intracardiac thrombus
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
|
20.0%
27/135
|
10.7%
6/56
|
28.1%
38/135
|
20.8%
11/53
|
|
Endocrine disorders
Adrenocortical insufficiency acute
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Eye disorders
Photophobia
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Gastrointestinal disorders
Abdominal pain
|
1.5%
2/135
|
0.00%
0/56
|
4.4%
6/135
|
1.9%
1/53
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
1.9%
1/53
|
|
Gastrointestinal disorders
Inguinal hernia
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Gastrointestinal disorders
Nausea
|
3.7%
5/135
|
0.00%
0/56
|
4.4%
6/135
|
0.00%
0/53
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Gastrointestinal disorders
Vomiting
|
3.0%
4/135
|
0.00%
0/56
|
5.2%
7/135
|
1.9%
1/53
|
|
General disorders
Asthenia
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
General disorders
Catheter site pain
|
0.00%
0/135
|
1.8%
1/56
|
0.74%
1/135
|
0.00%
0/53
|
|
General disorders
Chest pain
|
0.74%
1/135
|
0.00%
0/56
|
5.2%
7/135
|
7.5%
4/53
|
|
General disorders
Chills
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
1.9%
1/53
|
|
General disorders
Device breakage
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
General disorders
Device malfunction
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
1.9%
1/53
|
|
General disorders
Implant site reaction
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
General disorders
Oedema peripheral
|
0.74%
1/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
General disorders
Pain
|
1.5%
2/135
|
0.00%
0/56
|
2.2%
3/135
|
1.9%
1/53
|
|
General disorders
Pyrexia
|
4.4%
6/135
|
5.4%
3/56
|
12.6%
17/135
|
11.3%
6/53
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/135
|
1.8%
1/56
|
2.2%
3/135
|
1.9%
1/53
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
3.8%
2/53
|
|
Infections and infestations
Bacterial infection
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Bronchitis
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Infections and infestations
Catheter site infection
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Infections and infestations
Cellulitis
|
0.00%
0/135
|
3.6%
2/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Infections and infestations
Device related infection
|
0.74%
1/135
|
0.00%
0/56
|
2.2%
3/135
|
0.00%
0/53
|
|
Infections and infestations
Gastroenteritis
|
0.74%
1/135
|
0.00%
0/56
|
2.2%
3/135
|
1.9%
1/53
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Infected skin ulcer
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Lobar pneumonia
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Mycoplasma infection
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Parvovirus infection
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/135
|
0.00%
0/56
|
2.2%
3/135
|
0.00%
0/53
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.74%
1/135
|
1.8%
1/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Infections and infestations
Pneumonia
|
3.0%
4/135
|
0.00%
0/56
|
6.7%
9/135
|
7.5%
4/53
|
|
Infections and infestations
Pneumonia klebsiella
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Pneumonia streptococcal
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Pyelonephritis
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Infections and infestations
Rotavirus infection
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Sepsis
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Infections and infestations
Septic shock
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Sinusitis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Staphylococcal bacteraemia
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
1.9%
1/53
|
|
Infections and infestations
Streptococcal infection
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/135
|
3.6%
2/56
|
3.0%
4/135
|
1.9%
1/53
|
|
Infections and infestations
Urinary tract infection
|
1.5%
2/135
|
0.00%
0/56
|
3.0%
4/135
|
1.9%
1/53
|
|
Infections and infestations
Viral infection
|
0.74%
1/135
|
1.8%
1/56
|
1.5%
2/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Fall
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Fibula fracture
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Injury
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Patella fracture
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.74%
1/135
|
1.8%
1/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.74%
1/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal inflammation
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/135
|
1.8%
1/56
|
0.00%
0/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
0.00%
0/53
|
|
Vascular disorders
Poor peripheral circulation
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
|
Vascular disorders
Superior vena caval occlusion
|
0.00%
0/135
|
0.00%
0/56
|
0.00%
0/135
|
1.9%
1/53
|
Other adverse events
| Measure |
Period 1 Deferasirox (ICL670)
n=135 participants at risk
Period 1 (first 24 weeks) Deferasirox (ICL670) 20 mg/kg orally once daily.
|
Period 1 Deferoxamine (DFO)
n=56 participants at risk
Period 1 (first 24 weeks) Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg.
|
Period 2 Deferasirox (ICL670)
n=135 participants at risk
Period 2 (after 24 weeks) Deferasirox (ICL670) 20 mg/kg orally once daily.
|
Period 2 Deferoxamine (DFO) Then ICL670
n=53 participants at risk
Period 2 (After 24 weeks) DFO group cross over to Deferasirox (ICL670) orally 20 mg/kg completing 52 weeks on therapy and then entering an extension period.
|
|---|---|---|---|---|
|
Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
|
5.2%
7/135
|
7.1%
4/56
|
12.6%
17/135
|
13.2%
7/53
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/135
|
0.00%
0/56
|
3.0%
4/135
|
7.5%
4/53
|
|
Gastrointestinal disorders
Abdominal pain
|
10.4%
14/135
|
10.7%
6/56
|
22.2%
30/135
|
15.1%
8/53
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.2%
7/135
|
5.4%
3/56
|
9.6%
13/135
|
9.4%
5/53
|
|
Gastrointestinal disorders
Constipation
|
8.1%
11/135
|
3.6%
2/56
|
17.8%
24/135
|
7.5%
4/53
|
|
Gastrointestinal disorders
Diarrhoea
|
22.2%
30/135
|
8.9%
5/56
|
26.7%
36/135
|
17.0%
9/53
|
|
Gastrointestinal disorders
Nausea
|
11.9%
16/135
|
7.1%
4/56
|
24.4%
33/135
|
18.9%
10/53
|
|
Gastrointestinal disorders
Vomiting
|
13.3%
18/135
|
16.1%
9/56
|
22.2%
30/135
|
20.8%
11/53
|
|
General disorders
Chest pain
|
4.4%
6/135
|
8.9%
5/56
|
11.9%
16/135
|
17.0%
9/53
|
|
General disorders
Injection site pain
|
0.00%
0/135
|
5.4%
3/56
|
0.00%
0/135
|
0.00%
0/53
|
|
General disorders
Oedema peripheral
|
0.00%
0/135
|
0.00%
0/56
|
5.2%
7/135
|
1.9%
1/53
|
|
General disorders
Pain
|
0.00%
0/135
|
0.00%
0/56
|
8.1%
11/135
|
1.9%
1/53
|
|
General disorders
Pyrexia
|
10.4%
14/135
|
12.5%
7/56
|
25.2%
34/135
|
28.3%
15/53
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/135
|
0.00%
0/56
|
5.2%
7/135
|
5.7%
3/53
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/135
|
0.00%
0/56
|
0.74%
1/135
|
7.5%
4/53
|
|
Infections and infestations
Influenza
|
0.00%
0/135
|
0.00%
0/56
|
3.0%
4/135
|
5.7%
3/53
|
|
Infections and infestations
Nasopharyngitis
|
3.0%
4/135
|
5.4%
3/56
|
13.3%
18/135
|
11.3%
6/53
|
|
Infections and infestations
Otitis media
|
0.00%
0/135
|
0.00%
0/56
|
3.0%
4/135
|
5.7%
3/53
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/135
|
0.00%
0/56
|
4.4%
6/135
|
9.4%
5/53
|
|
Infections and infestations
Pneumonia
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
7.5%
4/53
|
|
Infections and infestations
Sinusitis
|
0.00%
0/135
|
0.00%
0/56
|
6.7%
9/135
|
3.8%
2/53
|
|
Infections and infestations
Upper respiratory tract infection
|
7.4%
10/135
|
12.5%
7/56
|
18.5%
25/135
|
13.2%
7/53
|
|
Infections and infestations
Urinary tract infection
|
5.2%
7/135
|
0.00%
0/56
|
12.6%
17/135
|
11.3%
6/53
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
0.00%
0/135
|
0.00%
0/56
|
1.5%
2/135
|
5.7%
3/53
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/135
|
0.00%
0/56
|
5.2%
7/135
|
1.9%
1/53
|
|
Investigations
Blood pressure diastolic increased
|
0.00%
0/135
|
0.00%
0/56
|
5.9%
8/135
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.2%
7/135
|
1.8%
1/56
|
14.8%
20/135
|
9.4%
5/53
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.2%
7/135
|
5.4%
3/56
|
14.8%
20/135
|
20.8%
11/53
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.00%
0/135
|
0.00%
0/56
|
6.7%
9/135
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.4%
10/135
|
5.4%
3/56
|
23.0%
31/135
|
17.0%
9/53
|
|
Nervous system disorders
Dizziness
|
1.5%
2/135
|
5.4%
3/56
|
5.9%
8/135
|
0.00%
0/53
|
|
Nervous system disorders
Headache
|
20.7%
28/135
|
26.8%
15/56
|
34.8%
47/135
|
24.5%
13/53
|
|
Psychiatric disorders
Insomnia
|
2.2%
3/135
|
5.4%
3/56
|
5.2%
7/135
|
1.9%
1/53
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/135
|
0.00%
0/56
|
5.2%
7/135
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.4%
10/135
|
12.5%
7/56
|
21.5%
29/135
|
26.4%
14/53
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/135
|
0.00%
0/56
|
5.2%
7/135
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/135
|
0.00%
0/56
|
5.9%
8/135
|
1.9%
1/53
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
4.4%
6/135
|
7.1%
4/56
|
14.8%
20/135
|
13.2%
7/53
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
4.4%
6/135
|
8.9%
5/56
|
17.8%
24/135
|
9.4%
5/53
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/135
|
0.00%
0/56
|
6.7%
9/135
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.00%
0/135
|
0.00%
0/56
|
2.2%
3/135
|
5.7%
3/53
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.2%
7/135
|
8.9%
5/56
|
9.6%
13/135
|
3.8%
2/53
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.4%
14/135
|
5.4%
3/56
|
15.6%
21/135
|
7.5%
4/53
|
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