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An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)

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ClinicalTrials.gov Identifier: NCT01736540
Recruitment Status : Completed
First Posted : November 29, 2012
Results First Posted : September 19, 2016
Last Update Posted : April 12, 2017
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Other
Condition Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia
Intervention Device: MRI scan
Enrollment 243
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Period Title: Overall Study
Started 243
Completed 228
Not Completed 15
Reason Not Completed
Did not complete scan within 45 days             2
Death             1
Abnormal test procedure             1
Withdrawal by Subject             5
Protocol Violation             1
Unable to tolerate MRI; claustrophobic             1
No show for MRI appointment             1
Unable to make MRI appointment             3
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Baseline Participants 243
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 243 participants
52.8  (20.92)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 243 participants
Female
109
  44.9%
Male
134
  55.1%
1.Primary Outcome
Title Percentage of Participants With Cardiac and Liver Iron Overload.
Hide Description Hepatic iron overload (liver siderosis) and cardiac iron overload (cardiac siderosis) in patients with transfusional siderosis (Myelodysplastic syndrome (MDS), thalassaemia major, non-transfusion-dependent thalassaemia (NTDT) and other anaemias) were measured using MRI (R2 by FerriScan and T2*, respectively).
Time Frame 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants with valid T2* by MRI and valid liver iron concentration (LIC) by MRI were included for the cardiac siderosis and liver siderosis analyses, respectively.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 228 76 69 18 65
Measure Type: Number
Unit of Measure: Percentage of participants
Cardiac 10 22 4 0 3
Liver 48 33 55 50 57
2.Primary Outcome
Title Cardiac Siderosis Severity
Hide Description Cardiac siderosis severity was measured by MRI (T2*). The severity grade of siderosis was tiered in 3 levels: mild (T2* >= 20ms), moderate (T2* from 10 to 20ms), and severe (T2* <10ms). Mild cardiac siderosis, by the definitions used in this study, were equivalent to not having cardiac siderosis. Values were compared to published thresholds of iron overload to determine severity of transfusion siderosis in the participant population studied.
Time Frame 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants with valid T2* by MRI were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 228 76 69 18 65
Measure Type: Number
Unit of Measure: Percentage of participants
None 89.9 77.6 95.7 100.0 95.4
Moderate 4.4 11.8 0.0 0.0 1.5
Severe 5.7 10.5 4.3 0.0 3.1
3.Secondary Outcome
Title Comparison of T2* Levels to Evaluate the Severity of Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups
Hide Description Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups).
Time Frame 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants with valid T2* by MRI were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 228 76 69 18 65
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ms
Overall (n=228,76,69,18,65)
30.2
(27.62 to 32.62)
23.95
(21.46 to 26.72)
30.13
(26.78 to 33.90)
32.69
(25.94 to 41.19)
30.76
(27.29 to 34.67)
Chelation naïve/minimally exposed(n=75,0,27,11,37)
32.55
(28.71 to 36.91)
NA [1] 
(NA to NA)
32.05
(26.66 to 38.53)
32.95
(24.70 to 43.97)
32.66
(27.91 to 38.22)
Chelator exposed (n=153,76,42,7,28)
28.25
(25.28 to 31.57)
23.95
(21.46 to 26.72)
28.32
(24.43 to 32.82)
32.43
(22.59 to 46.55)
28.96
(24.17 to 34.70)
[1]
No participants were minimally exposed to chelator treatment.
4.Secondary Outcome
Title Comparison of Liver Iron Concentration (LIC) Levels to Evaluate Iron Overload Due to Transfusion Therapy in Chelation-naïve and Chelation-treated Participant Subgroups
Hide Description Iron overload due to transfusion therapy was assessed based on chelation status of each participant (i.e. minimally exposed to chelator treatment and chelation-treated patient subgroups). The mean data presented are mean estimates of log transformed data.
Time Frame 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants with valid LIC by MRI were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 228 76 69 18 65
Mean (95% Confidence Interval)
Unit of Measure: mg Fe/g
Overall (n=228,76,69,18,65)
11.30
(9.42 to 13.17)
8.09
(5.62 to 10.57)
10.84
(8.18 to 13.50)
12.38
(7.16 to 17.59)
12.28
(9.58 to 14.98)
Chelation naïve/minimally exposed(n=75,0,27,11,37)
8.73
(5.90 to 11.56)
NA [1] 
(NA to NA)
10.28
(6.13 to 14.43)
5.00
(-1.50 to 11.50)
10.92
(7.37 to 14.47)
Chelator exposed (n=153,76,42,7,28)
13.22
(10.72 to 15.72)
8.09
(5.62 to 10.57)
11.40
(8.07 to 14.72)
19.76
(11.60 to 27.91)
13.64
(9.56 to 17.72)
[1]
No participants were minimally exposed.
5.Secondary Outcome
Title Mean Serum Ferritin According to the Presence or Absence of Retrospective Cardiac Events
Hide Description Mean serum ferritin according to the presence or absence of cardiac events was assessed for all participant subgroups.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with serum ferritin values and previous cardiac events data were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 238
Mean (Standard Deviation)
Unit of Measure: ng/mL
Cardiac event in past 12 months = yes (n=12) 2153.6  (1878.6)
Cardiac event in past 12 months = no (n=226) 2150.2  (1914.5)
6.Secondary Outcome
Title Mean Serum Ferritin According to the Presence or Absence of Retrospective Hepatic Events
Hide Description Mean serum ferritin according to the presence or absence of hepatic events was assessed for all participant subgroups.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with serum ferritin values and previous hepatic events data were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 239
Mean (Standard Deviation)
Unit of Measure: ng/mL
Hepatic event in the past 12 months = yes (n=8) 3302.8  (2684.3)
Hepatic event in the past 12 months = no (n=231) 2124.1  (1879.2)
7.Secondary Outcome
Title Mean Cardiac T2* According to the Presence or Absence of Retrospective Cardiac Events
Hide Description Mean cardiac T2* according to the presence or absence of cardiac events was assessed for all participant subgroups. The mean data presented are mean estimates of log transformed data.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with valid T2* by MRI results were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 228
Mean (Standard Deviation)
Unit of Measure: log10 (ms)
Cardiac event in past 12 months = yes (n=12) 1.546  (0.0979)
Cardiac event in the past 12 months = no (n=226) 1.445  (0.2188)
8.Secondary Outcome
Title Mean LIC According to the Presence or Absence of Retrospective Hepatic Events
Hide Description Mean LIC according to the presence or absence of hepatic events was assessed for all participant subgroups.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with LIC by MRI were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 228
Mean (Standard Deviation)
Unit of Measure: mg Fe/g
Hepatic event in past 12 months = yes (n=7) 17.51  (15.292)
Hepatic event in past 12 months = no (n=221) 10.08  (10.950)
9.Secondary Outcome
Title Mean Blood Magnetic Susceptibility (BMS)
Hide Description Blood samples were collected to assess BMS. The measurement represents absolute magnetic susceptibility at 1 month. Whole blood magnetic susceptibility was calculated by the addition of the dry weight susceptibility and the contribution of the water driven from the sample.
Time Frame 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with BMS values were analyzed.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 233
Mean (Standard Deviation)
Unit of Measure: emu/g wet wt/Oe
-6.18  (0.147)
10.Secondary Outcome
Title Percentage of Participants Transfused With Erythrocytes
Hide Description Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with a erythrocyte transfusion history were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 241 81 73 19 68
Measure Type: Number
Unit of Measure: Percentage of participants
95.0 100.0 100.0 36.8 100.0
11.Secondary Outcome
Title Percentage of Participants With Time Since Most Recent Transfuison of <7 Days, 7 to < 14 Days, 14 to < 30 Days, 30 to < 60 Days or >= 60 Days
Hide Description Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with data on time since their most recent transfusion were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 241 81 68 19 65
Measure Type: Number
Unit of Measure: percentage of participants
< 7 days 43.1 72.8 31.0 14.3 22.7
7 - < 14 days 12.9 2.5 28.2 14.3 9.1
14 - < 30 days 20.0 22.2 14.1 0.0 25.8
30 - < 60 days 5.8 2.5 9.9 14.3 4.5
>= 60 days 18.2 0.0 16.9 57.1 37.9
12.Secondary Outcome
Title Mean Number of Erythrocyte Units Transfused in Last 12 Months
Hide Description Transfusion requirement in participants with acquired anaemias with history of receiving chelation therapy was assessed.
Time Frame 12 months - retrospective
Hide Outcome Measure Data
Hide Analysis Population Description
Participants with 'number of units transfused' data were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 228 81 72 7 68
Mean (Standard Deviation)
Unit of Measure: number of units transfused
33.3  (21.0) 41.4  (9.5) 34.7  (25.6) 8.4  (12.2) 24.8  (21.5)
13.Secondary Outcome
Title Mean Quality of Life (QOL) Scores
Hide Description Quality of life was assessed using the Short Form 36 (SF-36) Health Survey. The SF-36 consists of 8 sub-scales: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning and mental health. The raw sores of the 8 scales are transformed to a 0 - 100 scale where 0 indicates maximum disability and 100 indicates no disability. There also are two physical and mental health summary measures. Each summary measure is the mean average of the 4 associated sub-scale scores. The range for each summary measure is 0 to 100 where 0 represents maximum disability and 100 represents no disability.
Time Frame 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
Only participants with data for each subscale were included in the analysis for that subscale.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 243 81 74 20 68
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical functioning 43.10  (11.471) 49.79  (9.144) 35.67  (9.791) 49.37  (7.576) 41.56  (11.139)
Role physical 42.54  (10.895) 49.51  (8.836) 36.71  (9.462) 46.46  (8.717) 39.44  (10.229)
Bodily pain 48.41  (10.969) 50.48  (11.084) 45.52  (10.750) 54.66  (7.773) 47.60  (10.815)
General health 41.65  (9.909) 44.82  (9.365) 39.48  (9.595) 43.23  (8.893) 39.80  (10.239)
Vitality 45.59  (10.267) 50.18  (9.277) 42.41  (9.190) 48.95  (8.424) 42.67  (10.757)
Social functioning 44.99  (11.075) 48.98  (9.869) 42.37  (10.486) 46.43  (9.905) 42.67  (12.043)
Role emotional 43.83  (12.977) 48.99  (11.236) 40.11  (13.101) 47.03  (10.552) 40.89  (13.320)
Mental health 47.84  (11.149) 50.09  (11.240) 46.69  (9.495) 48.74  (9.846) 46.12  (12.632)
Physical component summary 43.48  (9.808) 48.75  (7.809) 37.55  (8.748) 50.34  (6.392) 41.93  (9.524)
Mental component summary 46.76  (11.622) 49.58  (10.354) 45.91  (10.349) 47.47  (10.832) 44.08  (13.822)
14.Secondary Outcome
Title Percentage of Participants With Low Medium or High Adherence to Iron Chelator Therapy
Hide Description Adherence of participants was assessed using an adherence questionnaire. Adherence questionnaires were completed only by participants who received chelating agents. Participants answered yes or no to 6 statements such as "Forgot to take pills". Based on the responses to these questions, adherence was classified as low, medium or high.
Time Frame 1 month
Hide Outcome Measure Data
Hide Analysis Population Description
Participants who were on iron chelator therapy at screening, and had answered at least one question on the questionnaire and had sufficient information to score the questionnaire, were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI) Thalassemia Major Melodysplastic Syndrome (MDS) Non-transfusion-dependent Anaemia (NTDT) Other Anaemias
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Subset of overall participants with thalassemia major
Subset of overall participants with MDS
Subset of overall participants with NTDT
Subset of overall participants with other types of anaemia
Overall Number of Participants Analyzed 141 78 34 4 25
Measure Type: Number
Unit of Measure: Percentage of participants
Low 27.7 32.1 11.8 75.0 28.0
Medium 61.7 57.7 73.5 25.0 64.0
High 10.6 10.3 14.7 0.0 8.0
15.Secondary Outcome
Title Investigator Treatment Decisions Based on MRI Results
Hide Description Treatment decisions were recorded after the investigator evaluated the MRI results, in order to assess the impact of such diagnostic test on the overall clinical management of participants with iron overload. Investigators answered the following question: "Since the MRI scan, have you changed or are planning to change the management of iron in your subject?".
Time Frame 2 months
Hide Outcome Measure Data
Hide Analysis Population Description
Participants, for whom treatment decision questionnaire results were provided and for whom MRI results were available, were included in the analysis.
Arm/Group Title Magnetic Resonance Imaging (MRI)
Hide Arm/Group Description:
All participants were subjected to a non-invasive hepatic and cardiac MRI within 60 days of enrollment to measure iron overload.
Overall Number of Participants Analyzed 229
Measure Type: Number
Unit of Measure: Percentage of participants
Answer = yes 42.4
Answer = no 57.6
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Thalassaemia Major Myelodysplastic Syndrome (MDS) Other Anaemia Non-transfusion-dependent Anaemia (NTDT)
Hide Arm/Group Description Subset of overall participants with thalassemia major Subset of overall participants with MDS Subset of overall participants with other types of anaemias Subset of overall participants with NTDT
All-Cause Mortality
Thalassaemia Major Myelodysplastic Syndrome (MDS) Other Anaemia Non-transfusion-dependent Anaemia (NTDT)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
Thalassaemia Major Myelodysplastic Syndrome (MDS) Other Anaemia Non-transfusion-dependent Anaemia (NTDT)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/81 (2.47%)   8/74 (10.81%)   2/68 (2.94%)   0/20 (0.00%) 
Blood and lymphatic system disorders         
Anaemia † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Febrile neutropenia † 1  0/81 (0.00%)  1/74 (1.35%)  2/68 (2.94%)  0/20 (0.00%) 
Thrombocytopenia † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Cardiac disorders         
Angina pectoris † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Eye disorders         
Eye swelling † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Gastrointestinal disorders         
Diarrhoea † 1  0/81 (0.00%)  2/74 (2.70%)  1/68 (1.47%)  0/20 (0.00%) 
Rectal haemorrhage † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
General disorders         
Malaise † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Medical device site erosion † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Medical device site pain † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Medical device site swelling † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Pyrexia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Hepatobiliary disorders         
Cholecystitis acute † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Infections and infestations         
Infection † 1  1/81 (1.23%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Klebsiella infection † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Nasopharyngitis † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Pneumonia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Injury, poisoning and procedural complications         
Fall † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Investigations         
C-reactive protein increased † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Electrocardiogram QT prolonged † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Platelet count decreased † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Metabolism and nutrition disorders         
Hypokalaemia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Myelodysplastic syndrome † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Psychiatric disorders         
Anxiety † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Pulmonary oedema † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Thalassaemia Major Myelodysplastic Syndrome (MDS) Other Anaemia Non-transfusion-dependent Anaemia (NTDT)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   7/81 (8.64%)   9/74 (12.16%)   3/68 (4.41%)   0/20 (0.00%) 
Blood and lymphatic system disorders         
Anaemia † 1  0/81 (0.00%)  1/74 (1.35%)  1/68 (1.47%)  0/20 (0.00%) 
Febrile neutropenia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Thrombocytopenia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Cardiac disorders         
Palpitations † 1  0/81 (0.00%)  2/74 (2.70%)  0/68 (0.00%)  0/20 (0.00%) 
Ear and labyrinth disorders         
Hypoacusis † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Eye disorders         
Dry eye † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Gastrointestinal disorders         
Abdominal pain † 1  0/81 (0.00%)  3/74 (4.05%)  1/68 (1.47%)  0/20 (0.00%) 
Abdominal pain upper † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Constipation † 1  0/81 (0.00%)  1/74 (1.35%)  1/68 (1.47%)  0/20 (0.00%) 
Diarrhoea † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Gastrointestinal disorder † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Nausea † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Proctalgia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Rectal haemorrhage † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
General disorders         
Asthenia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Chest pain † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Fatigue † 1  0/81 (0.00%)  2/74 (2.70%)  1/68 (1.47%)  0/20 (0.00%) 
Influenza like illness † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Injection site reaction † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Oedema † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Oedema peripheral † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Pain † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Pyrexia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Infections and infestations         
Lower respiratory tract infection † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Pneumonia † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Respiratory tract infection † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Tonsillitis † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Upper respiratory tract infection † 1  2/81 (2.47%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Urinary tract infection † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Viral infection † 1  1/81 (1.23%)  0/74 (0.00%)  0/68 (0.00%)  0/20 (0.00%) 
Injury, poisoning and procedural complications         
Infusion related reaction † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Investigations         
Heart rate increased † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Metabolism and nutrition disorders         
Decreased appetite † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Dehydration † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Gout † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Hypokalaemia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Arthritis † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Flank pain † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Pain in extremity † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Nervous system disorders         
Headache † 1  0/81 (0.00%)  2/74 (2.70%)  0/68 (0.00%)  0/20 (0.00%) 
Sinus headache † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Psychiatric disorders         
Insomnia † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Sleep disorder † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Renal and urinary disorders         
Haematuria † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Dyspnoea † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Epistaxis † 1  0/81 (0.00%)  1/74 (1.35%)  0/68 (0.00%)  0/20 (0.00%) 
Skin and subcutaneous tissue disorders         
Pruritus † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
Vascular disorders         
Hypotension † 1  0/81 (0.00%)  0/74 (0.00%)  1/68 (1.47%)  0/20 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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 or disclosure of trial results in their entirety.
Results Point of Contact
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Name/Title: Study Director
Organization: Novartis
Phone: 862-778-8300
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01736540    
Other Study ID Numbers: CICL670AAU05
First Submitted: November 26, 2012
First Posted: November 29, 2012
Results First Submitted: May 28, 2016
Results First Posted: September 19, 2016
Last Update Posted: April 12, 2017