Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food
Study Details
Study Description
Brief Summary
This single-arm, open-label, multi-center study enrolled 65 patients from approximately 20 centers. All patients who met the study criteria and were taking, beginning or resuming treatment with Deferasirox were allowed. The study will began with a one month run-in phase, where all patients were instructed to take Deferasirox according to their physician's prescribing information.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
Phase 4 |
Detailed Description
Following the run-in phase, patients entered a three month, assessment phase. During the assessment phase, patients were given five general options for taking Deferasirox including with or without meals, crushed and added to a soft food or mixed in a liquid of choice.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: Deferasirox Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day. |
Drug: deferasirox:
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 [Week 8 and Week 12]
Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver.
Secondary Outcome Measures
- Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption [Day 1 up to Week 16]
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively.
- Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 [Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16]
Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated.
- Change From Baseline in Serum Ferritin at Week 16 [Baseline, Week 16 (End of study)]
Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Male or female patients with thalassemia major, sickle cell disease (SCD), low or intermediate 1 (INT 1) risk myelodysplastic syndrome (MDS) or other anemias and transfusional hemosiderosis.
-
Patients who were on, starting, or resuming treatment with Exjade.
-
Patients who were >2 years (i.e., 2 years of age or older).
Exclusion criteria:
-
Serum creatinine above the upper limit of normal (ULN) for age.
-
Alanine aminotransferase (ALT) >2.5 times the ULN.-High risk intermediate-2 or high risk MDS or acute leukemia.
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | Children's Hospital and Research Center | Oakland | California | United States | 94609 |
2 | Stanford University | Palo Alto | California | United States | 94304-1812 |
3 | Bay Area Cancer Research Group | Pleasant Hill | California | United States | 94523 |
4 | University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center | Aurora | Colorado | United States | 80045 |
5 | Yale University School of Medicine | New Haven | Connecticut | United States | 06520 |
6 | Medical College of Georgia | Augusta | Georgia | United States | 30912 |
7 | Children's Memorial | Chicago | Illinois | United States | 60614 |
8 | Tulane University Health Sciences Center | New Orleans | Louisiana | United States | 70118 |
9 | University of Maryland Greenebaum Cancer Center | Baltimore | Maryland | United States | 21201 |
10 | Children's Hospital of Boston | Boston | Massachusetts | United States | 02115 |
11 | Boston Medical Center | Boston | Massachusetts | United States | 02118 |
12 | Washington University School of Medicine | Saint Louis | Missouri | United States | 63110 |
13 | The Cancer Center at Hackensack University Medical Center | Hackensack | New Jersey | United States | 07601 |
14 | Cancer Institute of New Jersey | New Brunswick | New Jersey | United States | 08901 |
15 | St Joseph Children's Hospital | Paterson | New Jersey | United States | 07503 |
16 | Schneider Children's Hospital | New Hyde Park | New York | United States | 11040 |
17 | New York Presbyterian Hospital | New York | New York | United States | 10065 |
18 | New York Medical College | Valhalla | New York | United States | 10595 |
19 | Wake Forest University Health Sciences | Winston-Salem | North Carolina | United States | 27157 |
20 | University of Oklahoma | Oklahoma City | Oklahoma | United States | 73104 |
21 | Penn State Children's Hospital | Hershey | Pennsylvania | United States | 17033 |
22 | St Christopher's Hospital for Children | Philadelphia | Pennsylvania | United States | 19134 |
23 | Texas Children's Cancer Center and Hematology Services | Houston | Texas | United States | 77030 |
Sponsors and Collaborators
- Novartis Pharmaceuticals
Investigators
- Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Study Documents (Full-Text)
None provided.More Information
Additional Information:
Publications
None provided.- CICL670AUS32
- 2011-004217-17
Study Results
Participant Flow
Recruitment Details | The study was conducted at 17 centres in United States. |
---|---|
Pre-assignment Detail | 82 participants were screened, out of which 65 participants enrolled into study. Study design comprised of a one-month run-in period where participants took deferasirox according to the physician, followed by a three-month assessment period. In assessment period, participants were instructed to document the selected method of administration and meal (breakfast, dinner, or no meal). Method of administration and meal type were kept same over week, but allowed to change once a new week began. |
Arm/Group Title | Deferasirox |
---|---|
Arm/Group Description | Participants were administered with deferasirox starting dose of 20 milligram/kilogram/day (mg/kg/day), daily 30 minutes before meal for 4 weeks of Run-in period. Where as, Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. |
Period Title: Run-in Period | |
STARTED | 65 |
COMPLETED | 65 |
NOT COMPLETED | 0 |
Period Title: Run-in Period | |
STARTED | 65 |
COMPLETED | 58 |
NOT COMPLETED | 7 |
Baseline Characteristics
Arm/Group Title | Deferasirox |
---|---|
Arm/Group Description | Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. |
Overall Participants | 65 |
Age (years) [Mean (Standard Deviation) ] | |
Mean (Standard Deviation) [years] |
27.0
(22.88)
|
Sex: Female, Male (Count of Participants) | |
Female |
27
41.5%
|
Male |
38
58.5%
|
Race/Ethnicity, Customized (Count of Participants) | |
Caucasian |
26
40%
|
Black |
25
38.5%
|
Asian |
10
15.4%
|
Other |
4
6.2%
|
Taking Exjade prior to the study (Count of Participants) | |
Yes |
44
67.7%
|
No |
21
32.3%
|
Outcome Measures
Title | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 |
---|---|
Description | Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver. |
Time Frame | Week 8 and Week 12 |
Outcome Measure Data
Analysis Population Description |
---|
The primary analysis was performed in the ITT population defined as all subjects enrolled regardless of any treatment received or not. As per the planned analysis, data were summarized by counts and percentages of each category of response when administered with food or with different methods of administration. |
Arm/Group Title | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal Deferasirox With Soft Food) |
---|---|---|---|---|---|---|
Arm/Group Description | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food a dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to a soft food with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. |
Measure Participants | 16 | 11 | 6 | 10 | 40 | 17 |
Week 8, Dislike extremely |
10
15.4%
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
Week 8, Somewhat dislike |
0
0%
|
28.6
NaN
|
0
NaN
|
14.3
NaN
|
5.9
NaN
|
14.3
NaN
|
Week 8, Neither like or dislike |
20
30.8%
|
28.6
NaN
|
33.3
NaN
|
28.6
NaN
|
29.4
NaN
|
28.6
NaN
|
Week 8, Somewhat like |
30
46.2%
|
0
NaN
|
0
NaN
|
28.6
NaN
|
17.6
NaN
|
42.9
NaN
|
Week 8, Like extremely |
40
61.5%
|
42.9
NaN
|
66.7
NaN
|
28.6
NaN
|
47.1
NaN
|
14.3
NaN
|
Week 12, Dislike extremely |
0
0%
|
0
NaN
|
0
NaN
|
0
NaN
|
4.3
NaN
|
0
NaN
|
Week 12, Somewhat dislike |
0
0%
|
25
NaN
|
0
NaN
|
0
NaN
|
4.3
NaN
|
10
NaN
|
Week 12, Neither like or dislike |
16.7
25.7%
|
25
NaN
|
0
NaN
|
33.3
NaN
|
56.5
NaN
|
50
NaN
|
Week 12, Somewhat like |
33.3
51.2%
|
0
NaN
|
66.7
NaN
|
33.3
NaN
|
8.7
NaN
|
10
NaN
|
Week 12, Like extremely |
50
76.9%
|
50
NaN
|
33.3
NaN
|
33.3
NaN
|
26.1
NaN
|
30
NaN
|
Title | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption |
---|---|
Description | Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively. |
Time Frame | Day 1 up to Week 16 |
Outcome Measure Data
Analysis Population Description |
---|
The analysis was performed in the Safety Set (SAF) population, defined as all participants who received at least one dose of study medication. As per the planned analysis, data were summarized by counts and percentages of each category of response when administered with food or with different methods of administration. |
Arm/Group Title | Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal Deferasirox With Soft Food) |
---|---|---|---|---|---|---|---|
Arm/Group Description | Participants were administered with deferasirox starting dose of 20 milligram/kilogram/day (mg/kg/day), daily 30 minutes before meal. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food a dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to a soft food with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. |
Measure Participants | 62 | 20 | 18 | 14 | 17 | 39 | 30 |
AEs |
39
60%
|
10
NaN
|
6
NaN
|
6
NaN
|
6
NaN
|
24
NaN
|
11
NaN
|
SAEs |
5
7.7%
|
5
NaN
|
0
NaN
|
1
NaN
|
1
NaN
|
6
NaN
|
0
NaN
|
AEs leading to permanent discontinuation |
0
0%
|
2
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
1
NaN
|
AEs leading to temporary interruption |
4
6.2%
|
2
NaN
|
1
NaN
|
0
NaN
|
0
NaN
|
2
NaN
|
0
NaN
|
Death |
0
0%
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
0
NaN
|
Title | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 |
---|---|
Description | Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated. |
Time Frame | Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16 |
Outcome Measure Data
Analysis Population Description |
---|
The analysis was performed in the ITT population defined as all subjects enrolled regardless of any treatment received or not. As per the planned analysis, data was summarized as overall by combining all Arms/Groups. |
Arm/Group Title | Deferasirox |
---|---|
Arm/Group Description | Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. |
Measure Participants | 57 |
Week 8, Deferasirox 20 mg/kg |
36.30
|
Week 12, Deferasirox 20 mg/kg |
66.95
|
Week 16, Deferasirox 20 mg/kg |
27.75
|
Week 8, Deferasirox 30 mg/kg |
20.15
|
Week 12, Deferasirox 30 mg/kg |
34.30
|
Week 16, Deferasirox 30 mg/kg |
54.50
|
Title | Change From Baseline in Serum Ferritin at Week 16 |
---|---|
Description | Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox. |
Time Frame | Baseline, Week 16 (End of study) |
Outcome Measure Data
Analysis Population Description |
---|
The analysis was performed in the ITT population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively. As per the planned analysis, data was summarized as overall by combining all Arms/Groups. |
Arm/Group Title | Deferasirox |
---|---|
Arm/Group Description | Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. |
Measure Participants | 64 |
Age 2 to < 10 years |
-198.1
(653.11)
|
Age 10 to < 60 years |
38.3
(859.45)
|
Age ≥ 60 years |
-593
(1683.39)
|
Adverse Events
Time Frame | First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV), up to 16 weeks | |||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Adverse Event Reporting Description | The analysis was performed in the Safety Set (SAF) population, defined as all participants who received at least one dose of study medication. | |||||||||||||||
Arm/Group Title | Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal (Deferasirox With Soft Food) | Deferasirox (Overall) | ||||||||
Arm/Group Description | Participants were administered with deferasirox starting dose of 20 milligram/kilogram/day (mg/kg/day), daily 30 minutes before meal. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at breakfast for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to soft food a dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) at dinner for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received deferasirox at a minimum starting dose of 20 mg/kg/day dispersed in a beverage of choice (non-carbonated, non-alcoholic liquid) with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | Participants received crushed deferasirox at a minimum starting dose of 20 mg/kg/day added to a soft food with no meal for 12 weeks of assessment period. For participants receiving > 30 mg/kg/ day, a maximum of 40 mg/kg/day was allowed. | Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For subjects receiving > 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | ||||||||
All Cause Mortality |
||||||||||||||||
Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal (Deferasirox With Soft Food) | Deferasirox (Overall) | |||||||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 0/65 (0%) | ||||||||
Serious Adverse Events |
||||||||||||||||
Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal (Deferasirox With Soft Food) | Deferasirox (Overall) | |||||||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 5/62 (8.1%) | 5/20 (25%) | 0/18 (0%) | 1/14 (7.1%) | 1/17 (5.9%) | 6/39 (15.4%) | 0/30 (0%) | 15/65 (23.1%) | ||||||||
Blood and lymphatic system disorders | ||||||||||||||||
Acute chest syndrome | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Congenital, familial and genetic disorders | ||||||||||||||||
Sickle cell anaemia with crisis | 3/62 (4.8%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 1/17 (5.9%) | 5/39 (12.8%) | 0/30 (0%) | 8/65 (12.3%) | ||||||||
Gastrointestinal disorders | ||||||||||||||||
Pancreatitis acute | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
General disorders | ||||||||||||||||
Catheter related complication | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Pyrexia | 1/62 (1.6%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Hepatobiliary disorders | ||||||||||||||||
Cholelithiasis | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Infections and infestations | ||||||||||||||||
Influenza | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Perirectal abscess | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Pneumonia | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Sepsis | 0/62 (0%) | 2/20 (10%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Viral infection | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Vascular disorders | ||||||||||||||||
Hypotension | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Other (Not Including Serious) Adverse Events |
||||||||||||||||
Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) | No Meal (Deferasirox With Soft Food) | Deferasirox (Overall) | |||||||||
Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
Total | 30/62 (48.4%) | 10/20 (50%) | 6/18 (33.3%) | 6/14 (42.9%) | 6/17 (35.3%) | 18/39 (46.2%) | 9/30 (30%) | 52/65 (80%) | ||||||||
Blood and lymphatic system disorders | ||||||||||||||||
Thrombocytopenia | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Cardiac disorders | ||||||||||||||||
Palpitations | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Eye disorders | ||||||||||||||||
Ocular icterus | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 2/39 (5.1%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Photophobia | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Gastrointestinal disorders | ||||||||||||||||
Abdominal distension | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Abdominal pain | 6/62 (9.7%) | 1/20 (5%) | 2/18 (11.1%) | 2/14 (14.3%) | 0/17 (0%) | 1/39 (2.6%) | 1/30 (3.3%) | 8/65 (12.3%) | ||||||||
Abdominal pain upper | 2/62 (3.2%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 1/17 (5.9%) | 2/39 (5.1%) | 3/30 (10%) | 8/65 (12.3%) | ||||||||
Constipation | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 1/17 (5.9%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Diarrhoea | 15/62 (24.2%) | 1/20 (5%) | 1/18 (5.6%) | 1/14 (7.1%) | 0/17 (0%) | 2/39 (5.1%) | 2/30 (6.7%) | 20/65 (30.8%) | ||||||||
Gastrointestinal hypermotility | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Nausea | 9/62 (14.5%) | 1/20 (5%) | 0/18 (0%) | 2/14 (14.3%) | 1/17 (5.9%) | 3/39 (7.7%) | 0/30 (0%) | 15/65 (23.1%) | ||||||||
Vomiting | 4/62 (6.5%) | 1/20 (5%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 2/39 (5.1%) | 2/30 (6.7%) | 10/65 (15.4%) | ||||||||
General disorders | ||||||||||||||||
Asthenia | 1/62 (1.6%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Catheter site pain | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Chest discomfort | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Chest pain | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 1/30 (3.3%) | 2/65 (3.1%) | ||||||||
Exercise tolerance decreased | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Fatigue | 2/62 (3.2%) | 1/20 (5%) | 2/18 (11.1%) | 2/14 (14.3%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 8/65 (12.3%) | ||||||||
Oedema peripheral | 0/62 (0%) | 1/20 (5%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Pyrexia | 7/62 (11.3%) | 2/20 (10%) | 0/18 (0%) | 3/14 (21.4%) | 0/17 (0%) | 4/39 (10.3%) | 1/30 (3.3%) | 15/65 (23.1%) | ||||||||
Immune system disorders | ||||||||||||||||
Hypersensitivity | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Infections and infestations | ||||||||||||||||
Bronchiolitis | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Otitis media | 1/62 (1.6%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Upper respiratory tract infection | 2/62 (3.2%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 1/17 (5.9%) | 5/39 (12.8%) | 1/30 (3.3%) | 8/65 (12.3%) | ||||||||
Urinary tract infection | 1/62 (1.6%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Injury, poisoning and procedural complications | ||||||||||||||||
Arthropod bite | 0/62 (0%) | 0/20 (0%) | 2/18 (11.1%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Contusion | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Investigations | ||||||||||||||||
Alanine aminotransferase increased | 2/62 (3.2%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 1/17 (5.9%) | 0/39 (0%) | 0/30 (0%) | 4/65 (6.2%) | ||||||||
Aspartate aminotransferase increased | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Blood creatinine increased | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Blood pressure diastolic decreased | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Urine output decreased | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Urine protein/creatinine ratio increased | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Metabolism and nutrition disorders | ||||||||||||||||
Anorexia | 3/62 (4.8%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 4/65 (6.2%) | ||||||||
Hypokalaemia | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Hypomagnesaemia | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Musculoskeletal and connective tissue disorders | ||||||||||||||||
Arthralgia | 0/62 (0%) | 2/20 (10%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 1/30 (3.3%) | 3/65 (4.6%) | ||||||||
Back pain | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 2/39 (5.1%) | 1/30 (3.3%) | 4/65 (6.2%) | ||||||||
Bone lesion | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Groin pain | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Neck pain | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Pain in extremity | 1/62 (1.6%) | 2/20 (10%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 2/39 (5.1%) | 0/30 (0%) | 5/65 (7.7%) | ||||||||
Systemic lupus erythematosus | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Nervous system disorders | ||||||||||||||||
Dizziness | 0/62 (0%) | 0/20 (0%) | 2/18 (11.1%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Headache | 5/62 (8.1%) | 2/20 (10%) | 2/18 (11.1%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 1/30 (3.3%) | 7/65 (10.8%) | ||||||||
Migraine | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 1/17 (5.9%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Paraesthesia | 0/62 (0%) | 2/20 (10%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Psychiatric disorders | ||||||||||||||||
Insomnia | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 2/39 (5.1%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Renal and urinary disorders | ||||||||||||||||
Pollakiuria | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Proteinuria | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Respiratory, thoracic and mediastinal disorders | ||||||||||||||||
Asthma | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Bronchial hyperreactivity | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Cough | 2/62 (3.2%) | 2/20 (10%) | 0/18 (0%) | 1/14 (7.1%) | 1/17 (5.9%) | 0/39 (0%) | 0/30 (0%) | 6/65 (9.2%) | ||||||||
Dyspnoea | 1/62 (1.6%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 3/65 (4.6%) | ||||||||
Oropharyngeal pain | 1/62 (1.6%) | 2/20 (10%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 2/39 (5.1%) | 0/30 (0%) | 5/65 (7.7%) | ||||||||
Rhinorrhoea | 0/62 (0%) | 1/20 (5%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Upper respiratory tract congestion | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Wheezing | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Skin and subcutaneous tissue disorders | ||||||||||||||||
Decubitus ulcer | 0/62 (0%) | 0/20 (0%) | 0/18 (0%) | 1/14 (7.1%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Ecchymosis | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Eczema | 0/62 (0%) | 1/20 (5%) | 0/18 (0%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Pruritus | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 1/39 (2.6%) | 0/30 (0%) | 2/65 (3.1%) | ||||||||
Skin irritation | 1/62 (1.6%) | 0/20 (0%) | 0/18 (0%) | 0/14 (0%) | 1/17 (5.9%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) | ||||||||
Urticaria | 0/62 (0%) | 0/20 (0%) | 1/18 (5.6%) | 0/14 (0%) | 0/17 (0%) | 0/39 (0%) | 0/30 (0%) | 1/65 (1.5%) |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Name/Title | Study Director |
---|---|
Organization | Novartis pharmaceuticals |
Phone | 862-778-8300 |
Novartis.email@novartis.com |
- CICL670AUS32
- 2011-004217-17