Trial Outcomes & Findings for Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO). (NCT NCT01090323)
NCT ID: NCT01090323
Last Updated: 2011-06-06
Results Overview
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
185 participants
Primary outcome timeframe
0 - 60 months
Results posted on
2011-06-06
Participant Flow
Participant milestones
| Measure |
ICL670
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Overall Study
STARTED
|
132
|
53
|
|
Overall Study
COMPLETED
|
43
|
19
|
|
Overall Study
NOT COMPLETED
|
89
|
34
|
Reasons for withdrawal
| Measure |
ICL670
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Overall Study
Adverse Event
|
12
|
2
|
|
Overall Study
Abnormal Lab Values
|
3
|
1
|
|
Overall Study
Abnormal Test Procedure Results
|
2
|
0
|
|
Overall Study
Unsatisfactory therapeutic effect
|
4
|
2
|
|
Overall Study
Condition no longer requires drug
|
5
|
4
|
|
Overall Study
Protocol Violation
|
2
|
1
|
|
Overall Study
Withdrew consent
|
33
|
11
|
|
Overall Study
Lost to Follow-up
|
11
|
6
|
|
Overall Study
Administrative problems
|
6
|
4
|
|
Overall Study
Death
|
1
|
2
|
|
Overall Study
Stopped end of core
|
7
|
0
|
|
Overall Study
Stopped end of extension 1#
|
3
|
1
|
Baseline Characteristics
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
Baseline characteristics by cohort
| Measure |
ICL670
n=132 Participants
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
n=53 Participants
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Total
n=185 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
<12 years
|
33 participants
n=5 Participants
|
14 participants
n=7 Participants
|
47 participants
n=5 Participants
|
|
Age, Customized
12 - <16 years
|
32 participants
n=5 Participants
|
11 participants
n=7 Participants
|
43 participants
n=5 Participants
|
|
Age, Customized
16 - <65 years
|
67 participants
n=5 Participants
|
28 participants
n=7 Participants
|
95 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
80 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
111 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
52 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
74 Participants
n=5 Participants
|
|
Region of Enrollment
France
|
10 participants
n=5 Participants
|
2 participants
n=7 Participants
|
12 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
110 participants
n=5 Participants
|
46 participants
n=7 Participants
|
156 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
5 participants
n=5 Participants
|
1 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
7 participants
n=5 Participants
|
3 participants
n=7 Participants
|
10 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 0 - 60 monthsPopulation: The primary analysis was on Safety Analysis Set which comprised all participants who received at least one dose of ICL670 during the core/extension phase of the study. All participants previously treated with ICL670/DFO for 52weeks in the core study.
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
Outcome measures
| Measure |
ICL670
n=132 Participants
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
n=53 Participants
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Number of Participants With Adverse Events After Start of ICL670
Adverse Events
|
131 participants
10.66
|
49 participants
11.32
|
|
Number of Participants With Adverse Events After Start of ICL670
Deaths
|
1 participants
|
2 participants
|
SECONDARY outcome
Timeframe: 0 - 60 monthsPopulation: The primary analysis was on Full Analysis Set which comprised all participants who received at least one dose of ICL670 during the core or the extension phase of the study. All participants previously treated with ICL670 or DFO for 52 weeks in the core study were eligible for enrollment.
The main efficacy variable was change in serum ferritin in response to therapy with ICL670. Due to variability of serum ferritin, end of study was considered as the mean of at most the last 3 available observations after the start of ICL670.
Outcome measures
| Measure |
ICL670
n=132 Participants
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
n=53 Participants
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Change in Serum Ferritin From Start of ICL670 to End of Study
|
-245.5 µg/L
Interval -8701.3 to 7626.5
|
-134.3 µg/L
Interval -6124.0 to 5136.7
|
Adverse Events
ICL670
Serious events: 97 serious events
Other events: 129 other events
Deaths: 0 deaths
Crossover
Serious events: 34 serious events
Other events: 47 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ICL670
n=132 participants at risk
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
n=53 participants at risk
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Blood and lymphatic system disorders
Acute chest syndrome
|
3.8%
5/132
|
5.7%
3/53
|
|
Blood and lymphatic system disorders
Anaemia
|
0.76%
1/132
|
3.8%
2/53
|
|
Blood and lymphatic system disorders
Anaemia haemolytic autoimmune
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Coagulopathy
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Haemolysis
|
1.5%
2/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Haemolytic anaemia
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Hypersplenism
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Spleen disorder
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Splenomegaly
|
0.76%
1/132
|
0.00%
0/53
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.76%
1/132
|
0.00%
0/53
|
|
Cardiac disorders
Arrhythmia
|
0.76%
1/132
|
0.00%
0/53
|
|
Cardiac disorders
Cardiac failure congestive
|
1.5%
2/132
|
0.00%
0/53
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia
|
0.00%
0/132
|
1.9%
1/53
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
|
36.4%
48/132
|
35.8%
19/53
|
|
Ear and labyrinth disorders
Otorrhoea
|
0.76%
1/132
|
0.00%
0/53
|
|
Ear and labyrinth disorders
Tinnitus
|
0.00%
0/132
|
1.9%
1/53
|
|
Eye disorders
Vitreous haemorrhage
|
0.00%
0/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Abdominal pain
|
10.6%
14/132
|
7.5%
4/53
|
|
Gastrointestinal disorders
Abdominal pain upper
|
2.3%
3/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Ascites
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Constipation
|
1.5%
2/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Dental caries
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Diarrhoea
|
1.5%
2/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Food poisoning
|
0.00%
0/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.5%
2/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Gastrointestinal inflammation
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Nausea
|
2.3%
3/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Pancreatitis
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Pancreatitis acute
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Retroperitoneal haemorrhage
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Tooth disorder
|
0.76%
1/132
|
0.00%
0/53
|
|
Gastrointestinal disorders
Varices oesophageal
|
0.00%
0/132
|
1.9%
1/53
|
|
Gastrointestinal disorders
Vomiting
|
5.3%
7/132
|
9.4%
5/53
|
|
General disorders
Asthenia
|
0.00%
0/132
|
1.9%
1/53
|
|
General disorders
Catheter related complication
|
0.00%
0/132
|
3.8%
2/53
|
|
General disorders
Catheter site haemorrhage
|
0.00%
0/132
|
1.9%
1/53
|
|
General disorders
Catheter site pain
|
0.76%
1/132
|
1.9%
1/53
|
|
General disorders
Catheter thrombosis
|
0.00%
0/132
|
1.9%
1/53
|
|
General disorders
Chest discomfort
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Chest pain
|
6.1%
8/132
|
7.5%
4/53
|
|
General disorders
Chills
|
0.76%
1/132
|
1.9%
1/53
|
|
General disorders
Facial pain
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Granuloma
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Local swelling
|
0.00%
0/132
|
1.9%
1/53
|
|
General disorders
Necrosis
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Non-cardiac chest pain
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Oedema peripheral
|
0.76%
1/132
|
0.00%
0/53
|
|
General disorders
Pain
|
4.5%
6/132
|
1.9%
1/53
|
|
General disorders
Pyrexia
|
18.9%
25/132
|
17.0%
9/53
|
|
Hepatobiliary disorders
Bile duct stone
|
0.76%
1/132
|
0.00%
0/53
|
|
Hepatobiliary disorders
Biliary colic
|
0.76%
1/132
|
0.00%
0/53
|
|
Hepatobiliary disorders
Cholecystitis
|
0.76%
1/132
|
1.9%
1/53
|
|
Hepatobiliary disorders
Cholelithiasis
|
5.3%
7/132
|
3.8%
2/53
|
|
Hepatobiliary disorders
Hepatic cirrhosis
|
0.76%
1/132
|
0.00%
0/53
|
|
Hepatobiliary disorders
Hepatic failure
|
0.76%
1/132
|
0.00%
0/53
|
|
Hepatobiliary disorders
Hepatic sequestration
|
0.76%
1/132
|
0.00%
0/53
|
|
Hepatobiliary disorders
Hyperbilirubinaemia
|
0.00%
0/132
|
1.9%
1/53
|
|
Hepatobiliary disorders
Jaundice
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Abscess limb
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Adenovirus infection
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Appendicitis
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Bacteraemia
|
1.5%
2/132
|
0.00%
0/53
|
|
Infections and infestations
Bronchitis
|
2.3%
3/132
|
3.8%
2/53
|
|
Infections and infestations
Catheter bacteraemia
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Catheter related infection
|
3.8%
5/132
|
3.8%
2/53
|
|
Infections and infestations
Cellulitis
|
2.3%
3/132
|
1.9%
1/53
|
|
Infections and infestations
Central line infection
|
0.76%
1/132
|
1.9%
1/53
|
|
Infections and infestations
Endocarditis
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Gastroenteritis
|
1.5%
2/132
|
5.7%
3/53
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Herpes zoster
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Influenza
|
0.76%
1/132
|
1.9%
1/53
|
|
Infections and infestations
Lobar pneumonia
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Lung infection
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Mononucleosis syndrome
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Osteomyelitis
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Otitis media
|
0.76%
1/132
|
1.9%
1/53
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Pneumonia
|
9.8%
13/132
|
5.7%
3/53
|
|
Infections and infestations
Pneumonia bacterial
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Pyelonephritis
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Pyelonephritis acute
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Respiratory syncytial virus infection
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Sepsis
|
2.3%
3/132
|
1.9%
1/53
|
|
Infections and infestations
Sinusitis
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Staphylococcal bacteraemia
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Staphylococcal infection
|
0.76%
1/132
|
1.9%
1/53
|
|
Infections and infestations
Staphylococcal sepsis
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Subcutaneous abscess
|
0.00%
0/132
|
1.9%
1/53
|
|
Infections and infestations
Tuberculosis
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Upper respiratory tract infection
|
2.3%
3/132
|
5.7%
3/53
|
|
Infections and infestations
Urinary tract infection
|
2.3%
3/132
|
1.9%
1/53
|
|
Infections and infestations
Viral infection
|
0.76%
1/132
|
0.00%
0/53
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Alcohol poisoning
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Arteriovenous fistula thrombosis
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Citrate toxicity
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Collapse of lung
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Device failure
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Drug toxicity
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Excoriation
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Haemolytic transfusion reaction
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Incision site pain
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Jaw fracture
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Medical device complication
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Meniscus lesion
|
0.76%
1/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Overdose
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Patella fracture
|
0.00%
0/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
2.3%
3/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Vascular graft occlusion
|
0.00%
0/132
|
1.9%
1/53
|
|
Investigations
Alanine aminotransferase increased
|
0.76%
1/132
|
1.9%
1/53
|
|
Investigations
Aspartate aminotransferase increased
|
0.76%
1/132
|
1.9%
1/53
|
|
Investigations
Biopsy liver
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Blood alkaline phosphatase
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Blood amylase increased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Blood bilirubin increased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Blood iron increased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Lipase increased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Oxygen saturation decreased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Pregnancy test positive
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Total lung capacity decreased
|
0.76%
1/132
|
0.00%
0/53
|
|
Investigations
Transaminases increased
|
0.76%
1/132
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.76%
1/132
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Dehydration
|
3.0%
4/132
|
1.9%
1/53
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.76%
1/132
|
0.00%
0/53
|
|
Metabolism and nutrition disorders
Iron overload
|
0.76%
1/132
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.3%
3/132
|
3.8%
2/53
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
4.5%
6/132
|
9.4%
5/53
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Costochondritis
|
0.00%
0/132
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
2.3%
3/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Osteolysis
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
3.0%
4/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
4.5%
6/132
|
5.7%
3/53
|
|
Musculoskeletal and connective tissue disorders
Rheumatoid arthritis
|
0.00%
0/132
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Rotator cuff syndrome
|
0.76%
1/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Synovial cyst
|
0.76%
1/132
|
0.00%
0/53
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Chronic myeloid leukaemia
|
0.76%
1/132
|
0.00%
0/53
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung neoplasm
|
0.00%
0/132
|
1.9%
1/53
|
|
Nervous system disorders
Altered state of consciousness
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Complex partial seizures
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Convulsion
|
2.3%
3/132
|
1.9%
1/53
|
|
Nervous system disorders
Dizziness
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Encephalopathy
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Facial palsy
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Haemorrhage intracranial
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Headache
|
5.3%
7/132
|
3.8%
2/53
|
|
Nervous system disorders
Hepatic encephalopathy
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Hypoaesthesia
|
1.5%
2/132
|
0.00%
0/53
|
|
Nervous system disorders
Intraventricular haemorrhage
|
0.76%
1/132
|
3.8%
2/53
|
|
Nervous system disorders
Migraine
|
1.5%
2/132
|
0.00%
0/53
|
|
Nervous system disorders
Paraesthesia
|
0.76%
1/132
|
0.00%
0/53
|
|
Nervous system disorders
Syncope
|
2.3%
3/132
|
0.00%
0/53
|
|
Nervous system disorders
Transient ischaemic attack
|
2.3%
3/132
|
0.00%
0/53
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
0.76%
1/132
|
1.9%
1/53
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
2.3%
3/132
|
1.9%
1/53
|
|
Psychiatric disorders
Abnormal behaviour
|
0.76%
1/132
|
0.00%
0/53
|
|
Psychiatric disorders
Conversion disorder
|
0.76%
1/132
|
0.00%
0/53
|
|
Psychiatric disorders
Mental status changes
|
0.76%
1/132
|
0.00%
0/53
|
|
Renal and urinary disorders
Enuresis
|
0.76%
1/132
|
0.00%
0/53
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/132
|
1.9%
1/53
|
|
Renal and urinary disorders
Nephropathy toxic
|
0.00%
0/132
|
1.9%
1/53
|
|
Renal and urinary disorders
Oliguria
|
0.76%
1/132
|
0.00%
0/53
|
|
Renal and urinary disorders
Renal failure acute
|
1.5%
2/132
|
0.00%
0/53
|
|
Reproductive system and breast disorders
Breast swelling
|
0.76%
1/132
|
0.00%
0/53
|
|
Reproductive system and breast disorders
Epididymitis
|
0.76%
1/132
|
0.00%
0/53
|
|
Reproductive system and breast disorders
Priapism
|
2.3%
3/132
|
1.9%
1/53
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
0.00%
0/132
|
1.9%
1/53
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
3.0%
4/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.0%
4/132
|
1.9%
1/53
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Lung infiltration
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
0.00%
0/132
|
3.8%
2/53
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary thrombosis
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
0.76%
1/132
|
0.00%
0/53
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.76%
1/132
|
0.00%
0/53
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
1.5%
2/132
|
0.00%
0/53
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
0.76%
1/132
|
0.00%
0/53
|
|
Surgical and medical procedures
Catheter placement
|
0.76%
1/132
|
0.00%
0/53
|
|
Surgical and medical procedures
Catheter removal
|
0.76%
1/132
|
0.00%
0/53
|
|
Surgical and medical procedures
Central venous catheterisation
|
0.76%
1/132
|
0.00%
0/53
|
|
Surgical and medical procedures
Cholecystectomy
|
0.76%
1/132
|
0.00%
0/53
|
|
Surgical and medical procedures
Hip arthroplasty
|
0.76%
1/132
|
0.00%
0/53
|
|
Vascular disorders
Haematoma
|
0.76%
1/132
|
0.00%
0/53
|
|
Vascular disorders
Hypotension
|
0.76%
1/132
|
0.00%
0/53
|
|
Vascular disorders
Jugular vein distension
|
0.76%
1/132
|
0.00%
0/53
|
Other adverse events
| Measure |
ICL670
n=132 participants at risk
Participants treated with ICL670 during the Core Study 0109(NCT00067080) and during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
Crossover
n=53 participants at risk
Participants treated with Deferoxamine (DFO) during the Core Study 0109 (NCT00067080) and treated with ICL670 during the extension phase. ICL670 was administered orally once a day based on the participant's body weight.
|
|---|---|---|
|
Blood and lymphatic system disorders
Leukocytosis
|
3.0%
4/132
|
5.7%
3/53
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
6.1%
8/132
|
3.8%
2/53
|
|
Cardiac disorders
Tachycardia
|
1.5%
2/132
|
5.7%
3/53
|
|
Congenital, familial and genetic disorders
Sickle cell anaemia with crisis
|
28.0%
37/132
|
24.5%
13/53
|
|
Ear and labyrinth disorders
Ear pain
|
6.1%
8/132
|
5.7%
3/53
|
|
Eye disorders
Ocular icterus
|
8.3%
11/132
|
11.3%
6/53
|
|
Eye disorders
Vision blurred
|
6.8%
9/132
|
9.4%
5/53
|
|
Gastrointestinal disorders
Abdominal discomfort
|
6.8%
9/132
|
3.8%
2/53
|
|
Gastrointestinal disorders
Abdominal pain
|
24.2%
32/132
|
24.5%
13/53
|
|
Gastrointestinal disorders
Abdominal pain upper
|
20.5%
27/132
|
28.3%
15/53
|
|
Gastrointestinal disorders
Constipation
|
21.2%
28/132
|
13.2%
7/53
|
|
Gastrointestinal disorders
Diarrhoea
|
29.5%
39/132
|
35.8%
19/53
|
|
Gastrointestinal disorders
Dyspepsia
|
6.1%
8/132
|
5.7%
3/53
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
1.5%
2/132
|
5.7%
3/53
|
|
Gastrointestinal disorders
Nausea
|
39.4%
52/132
|
35.8%
19/53
|
|
Gastrointestinal disorders
Toothache
|
6.8%
9/132
|
7.5%
4/53
|
|
Gastrointestinal disorders
Vomiting
|
34.1%
45/132
|
32.1%
17/53
|
|
General disorders
Asthenia
|
5.3%
7/132
|
1.9%
1/53
|
|
General disorders
Chest pain
|
18.2%
24/132
|
28.3%
15/53
|
|
General disorders
Chills
|
6.8%
9/132
|
7.5%
4/53
|
|
General disorders
Fatigue
|
16.7%
22/132
|
13.2%
7/53
|
|
General disorders
Oedema peripheral
|
10.6%
14/132
|
7.5%
4/53
|
|
General disorders
Pain
|
12.1%
16/132
|
11.3%
6/53
|
|
General disorders
Pyrexia
|
34.1%
45/132
|
41.5%
22/53
|
|
Hepatobiliary disorders
Jaundice
|
4.5%
6/132
|
5.7%
3/53
|
|
Infections and infestations
Bronchitis
|
6.1%
8/132
|
1.9%
1/53
|
|
Infections and infestations
Cellulitis
|
1.5%
2/132
|
5.7%
3/53
|
|
Infections and infestations
Ear infection
|
6.8%
9/132
|
1.9%
1/53
|
|
Infections and infestations
Gastroenteritis
|
9.1%
12/132
|
5.7%
3/53
|
|
Infections and infestations
Gastroenteritis viral
|
6.1%
8/132
|
7.5%
4/53
|
|
Infections and infestations
Influenza
|
10.6%
14/132
|
7.5%
4/53
|
|
Infections and infestations
Nasopharyngitis
|
28.0%
37/132
|
30.2%
16/53
|
|
Infections and infestations
Oral herpes
|
6.1%
8/132
|
1.9%
1/53
|
|
Infections and infestations
Pharyngitis
|
9.1%
12/132
|
7.5%
4/53
|
|
Infections and infestations
Pharyngitis streptococcal
|
5.3%
7/132
|
7.5%
4/53
|
|
Infections and infestations
Pneumonia
|
1.5%
2/132
|
5.7%
3/53
|
|
Infections and infestations
Rhinitis
|
6.8%
9/132
|
0.00%
0/53
|
|
Infections and infestations
Sinusitis
|
12.9%
17/132
|
9.4%
5/53
|
|
Infections and infestations
Staphylococcal infection
|
5.3%
7/132
|
1.9%
1/53
|
|
Infections and infestations
Tonsillitis
|
5.3%
7/132
|
1.9%
1/53
|
|
Infections and infestations
Upper respiratory tract infection
|
32.6%
43/132
|
35.8%
19/53
|
|
Infections and infestations
Urinary tract infection
|
15.2%
20/132
|
5.7%
3/53
|
|
Infections and infestations
Viral infection
|
8.3%
11/132
|
1.9%
1/53
|
|
Injury, poisoning and procedural complications
Allergic transfusion reaction
|
0.76%
1/132
|
5.7%
3/53
|
|
Injury, poisoning and procedural complications
Excoriation
|
6.1%
8/132
|
0.00%
0/53
|
|
Injury, poisoning and procedural complications
Skin laceration
|
3.0%
4/132
|
5.7%
3/53
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
6.1%
8/132
|
3.8%
2/53
|
|
Investigations
Alanine aminotransferase increased
|
4.5%
6/132
|
7.5%
4/53
|
|
Investigations
Aspartate aminotransferase increased
|
6.1%
8/132
|
5.7%
3/53
|
|
Investigations
Blood creatinine increased
|
9.1%
12/132
|
7.5%
4/53
|
|
Investigations
Serum ferritin increased
|
6.1%
8/132
|
7.5%
4/53
|
|
Investigations
Transaminases increased
|
6.8%
9/132
|
1.9%
1/53
|
|
Metabolism and nutrition disorders
Decreased appetite
|
5.3%
7/132
|
5.7%
3/53
|
|
Metabolism and nutrition disorders
Dehydration
|
3.8%
5/132
|
7.5%
4/53
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
6.1%
8/132
|
9.4%
5/53
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
27.3%
36/132
|
26.4%
14/53
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
36.4%
48/132
|
28.3%
15/53
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.8%
9/132
|
0.00%
0/53
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
7.6%
10/132
|
1.9%
1/53
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
3.8%
5/132
|
5.7%
3/53
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.1%
12/132
|
17.0%
9/53
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
4.5%
6/132
|
7.5%
4/53
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
2.3%
3/132
|
7.5%
4/53
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
31.1%
41/132
|
24.5%
13/53
|
|
Nervous system disorders
Dizziness
|
12.9%
17/132
|
13.2%
7/53
|
|
Nervous system disorders
Headache
|
46.2%
61/132
|
56.6%
30/53
|
|
Nervous system disorders
Hypoaesthesia
|
5.3%
7/132
|
1.9%
1/53
|
|
Nervous system disorders
Migraine
|
5.3%
7/132
|
7.5%
4/53
|
|
Psychiatric disorders
Anxiety
|
5.3%
7/132
|
5.7%
3/53
|
|
Psychiatric disorders
Depression
|
7.6%
10/132
|
0.00%
0/53
|
|
Psychiatric disorders
Insomnia
|
9.8%
13/132
|
3.8%
2/53
|
|
Renal and urinary disorders
Chromaturia
|
2.3%
3/132
|
5.7%
3/53
|
|
Renal and urinary disorders
Proteinuria
|
5.3%
7/132
|
0.00%
0/53
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
7.6%
10/132
|
7.5%
4/53
|
|
Reproductive system and breast disorders
Ovarian cyst
|
0.76%
1/132
|
5.7%
3/53
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
26.5%
35/132
|
26.4%
14/53
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.1%
12/132
|
11.3%
6/53
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.1%
8/132
|
7.5%
4/53
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
22/132
|
17.0%
9/53
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
30.3%
40/132
|
28.3%
15/53
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
4.5%
6/132
|
5.7%
3/53
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
6.8%
9/132
|
7.5%
4/53
|
|
Skin and subcutaneous tissue disorders
Acne
|
3.8%
5/132
|
5.7%
3/53
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.1%
16/132
|
5.7%
3/53
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.9%
21/132
|
17.0%
9/53
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
5.3%
7/132
|
1.9%
1/53
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
6.8%
9/132
|
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