Palatability and Tolerability of Deferasirox Taken With M... | NCT00845871 | Trialant
NCT00845871
Sponsor
Novartis Pharmaceuticals
Status
Completed
Last Update Posted
Jul 20, 2021Actual
Enrollment
65Actual
Phase
Phase 4
Conditions
Transfusional Hemosiderosis
Interventions
deferasirox:
Countries
United States
Protocol Section
Identification Module
NCT ID
NCT00845871
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
CICL670AUS32
Secondary IDs
ID
Type
Description
Link
2011-004217-17
EudraCT Number
Brief Title
Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food
Official Title
A Single-arm, Open-label Study of the Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food
Acronym
Not provided
Organization
NovartisINDUSTRY
Status Module
Record Verification Date
Jul 2021
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
May 2009
Primary Completion Date
Aug 2010Actual
Completion Date
Aug 2010Actual
First Submitted Date
Feb 16, 2009
First Submission Date that Met QC Criteria
Feb 17, 2009
First Posted Date
Feb 18, 2009Estimated
Results Waived
Not provided
Results First Submitted Date
May 11, 2021
Results First Submitted that Met QC Criteria
Jul 19, 2021
Results First Posted Date
Jul 20, 2021Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Jul 19, 2021
Last Update Posted Date
Jul 20, 2021Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Novartis PharmaceuticalsINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
No
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
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.
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.
Conditions Module
Conditions
Transfusional Hemosiderosis
Keywords
Exjade
deferasirox
palatability
tolerability
food
iron overload
Serum Iron overload due to transfusions
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 4
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
65Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Deferasirox
Experimental
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Drug: deferasirox:
Interventions
Name
Type
Description
Arm Group Labels
Other Names
deferasirox:
Drug
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Deferasirox
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Percentage of Participants With Differing Palatability Scores at 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.
Week 8 and Week 12
Secondary Outcomes
Measure
Description
Time Frame
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
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.
Other Outcomes
Not provided
Eligibility Module
Eligibility 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.
Goldberg SL, Giardina PJ, Chirnomas D, Esposito J, Paley C, Vichinsky E. The palatability and tolerability of deferasirox taken with different beverages or foods. Pediatr Blood Cancer. 2013 Sep;60(9):1507-12. doi: 10.1002/pbc.24561. Epub 2013 Apr 23.
See Also Links
Label
URL
Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.
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.
Recruitment Details
The study was conducted at 17 centres in United States.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Deferasirox
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.
Periods
Title
Milestones
Reasons Not Completed
Run-in Period
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
4.90
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
No data available
No data is available for this block.
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
N/A
Intervention Model
Single Group Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
Exjade
Deferasirox
Day 1 up to Week 16
Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 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.
Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
Change From Baseline in Serum Ferritin at Week 16
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.
Baseline, Week 16 (End of study)
Palo Alto
California
94304-1812
United States
Bay Area Cancer Research Group
Pleasant Hill
California
94523
United States
University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
Aurora
Colorado
80045
United States
Yale University School of Medicine
New Haven
Connecticut
06520
United States
Medical College of Georgia
Augusta
Georgia
30912
United States
Children's Memorial
Chicago
Illinois
60614
United States
Tulane University Health Sciences Center
New Orleans
Louisiana
70118
United States
University of Maryland Greenebaum Cancer Center
Baltimore
Maryland
21201
United States
Children's Hospital of Boston
Boston
Massachusetts
02115
United States
Boston Medical Center
Boston
Massachusetts
02118
United States
Washington University School of Medicine
St Louis
Missouri
63110
United States
The Cancer Center at Hackensack University Medical Center
Hackensack
New Jersey
07601
United States
Cancer Institute of New Jersey
New Brunswick
New Jersey
08901
United States
St Joseph Children's Hospital
Paterson
New Jersey
07503
United States
Schneider Children's Hospital
New Hyde Park
New York
11040
United States
New York Presbyterian Hospital
New York
New York
10065
United States
New York Medical College
Valhalla
New York
10595
United States
Wake Forest University Health Sciences
Winston-Salem
North Carolina
27157
United States
University of Oklahoma
Oklahoma City
Oklahoma
73104
United States
Penn State Children's Hospital
Hershey
Pennsylvania
17033
United States
St Christopher's Hospital for Children
Philadelphia
Pennsylvania
19134
United States
Texas Children's Cancer Center and Hematology Services
Houston
Texas
77030
United States
FG00065 subjects
COMPLETED
FG00065 subjects
NOT COMPLETED
FG0000 subjects
Assessment Period
Type
Comment
Milestone Data
STARTED
FG00065 subjects
COMPLETED
FG00058 subjects
NOT COMPLETED
FG0007 subjects
Type
Comment
Reasons
Adverse Event
FG0002 subjects
Abnormal laboratory values
FG0001 subjects
Abnormal test procedure results
FG0001 subjects
Analysis was performed in Intent-to-treat (ITT) population defined as all enrolled patients regardless of any treatment received or not. As per the planned analysis, data was summarized as overall by combining all Arms/Groups.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Deferasirox
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.
Denominators
Units
Counts
Participants
BG00065
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
Title
Measurements
BG00027.0± 22.88
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG00027
Male
BG00038
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
Caucasian
Title
Measurements
BG00026
Black
Title
Measurements
BG000
Taking Exjade prior to the study
Count of Participants
Participants
Title
Denominators
Categories
Yes
Title
Measurements
BG00044
No
Title
Measurements
BG000
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Percentage of Participants With Differing Palatability Scores at 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.
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.
Posted
Number
percentage of participants
Week 8 and Week 12
ID
Title
Description
OG000
Breakfast (Deferasirox With Soft Food)
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.
OG001
Breakfast (Deferasirox With Liquid)
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.
OG002
Dinner (Deferasirox With Soft Food)
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.
OG003
Dinner (Deferasirox With Liquid)
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.
OG004
No Meal (Deferasirox With Liquid)
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.
OG005
No Meal Deferasirox With Soft Food)
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.
Units
Counts
Participants
OG00016
OG00111
OG0026
OG003
Title
Denominators
Categories
Week 8, Dislike extremely
ParticipantsOG00010
ParticipantsOG0017
ParticipantsOG0023
ParticipantsOG003
Secondary
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
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.
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.
Posted
Count of Participants
Participants
Day 1 up to Week 16
ID
Title
Description
OG000
Deferasirox (Run-in Phase)
Participants were administered with deferasirox starting dose of 20 milligram/kilogram/day (mg/kg/day), daily 30 minutes before meal.
OG001
Breakfast (Deferasirox With Soft Food)
Secondary
Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 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.
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.
Posted
Median
Full Range
micromoles per litre (μmol/L)
Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
ID
Title
Description
OG000
Deferasirox
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.
Units
Counts
Participants
OG000
Secondary
Change From Baseline in Serum Ferritin at Week 16
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.
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.
Posted
Mean
Standard Deviation
Nanogram/mlillilitre
Baseline, Week 16 (End of study)
ID
Title
Description
OG000
Deferasirox
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.
Units
Counts
Participants
OG000
Time Frame
First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV), up to 16 weeks
Description
The analysis was performed in the Safety Set (SAF) population, defined as all participants who received at least one dose of study medication.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Deferasirox (Run-in Phase)
Participants were administered with deferasirox starting dose of 20 milligram/kilogram/day (mg/kg/day), daily 30 minutes before meal.
0
62
5
62
30
62
EG001
Breakfast (Deferasirox With Soft Food)
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.
0
20
5
20
10
20
EG002
Breakfast (Deferasirox With Liquid)
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.
0
18
0
18
6
18
EG003
Dinner (Deferasirox With Soft Food)
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.
0
14
1
14
6
14
EG004
Dinner (Deferasirox With Liquid)
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.
0
17
1
17
6
17
EG005
No Meal (Deferasirox With Liquid)
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.
0
39
6
39
18
39
EG006
No Meal (Deferasirox With Soft Food)
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.
0
30
0
30
9
30
EG007
Deferasirox (Overall)
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.
0
65
15
65
52
65
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Acute chest syndrome
Blood and lymphatic system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG0030 affected14 at risk
EG004
Sickle cell anaemia with crisis
Congenital, familial and genetic disorders
MedDRA (Unspecified)
Systematic Assessment
EG0003 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Pancreatitis acute
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Catheter related complication
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Pyrexia
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Cholelithiasis
Hepatobiliary disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Influenza
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Perirectal abscess
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Sepsis
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Viral infection
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Hypotension
Vascular disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG0030 affected14 at risk
EG004
Palpitations
Cardiac disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Ocular icterus
Eye disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Photophobia
Eye disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Abdominal distension
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0006 affected62 at risk
EG0011 affected20 at risk
EG0022 affected18 at risk
EG003
Abdominal pain upper
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0002 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG00015 affected62 at risk
EG0011 affected20 at risk
EG0021 affected18 at risk
EG003
Gastrointestinal hypermotility
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0009 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0004 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Asthenia
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Catheter site pain
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Chest discomfort
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Chest pain
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Exercise tolerance decreased
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Fatigue
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0002 affected62 at risk
EG0011 affected20 at risk
EG0022 affected18 at risk
EG003
Oedema peripheral
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0021 affected18 at risk
EG003
Pyrexia
General disorders
MedDRA (Unspecified)
Systematic Assessment
EG0007 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Hypersensitivity
Immune system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Bronchiolitis
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Otitis media
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0002 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Arthropod bite
Injury, poisoning and procedural complications
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0022 affected18 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0002 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Blood creatinine increased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Blood pressure diastolic decreased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Urine output decreased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Urine protein/creatinine ratio increased
Investigations
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Anorexia
Metabolism and nutrition disorders
MedDRA (Unspecified)
Systematic Assessment
EG0003 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Hypokalaemia
Metabolism and nutrition disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Hypomagnesaemia
Metabolism and nutrition disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Bone lesion
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Groin pain
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Neck pain
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0012 affected20 at risk
EG0021 affected18 at risk
EG003
Systemic lupus erythematosus
Musculoskeletal and connective tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Dizziness
Nervous system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0022 affected18 at risk
EG003
Headache
Nervous system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0005 affected62 at risk
EG0012 affected20 at risk
EG0022 affected18 at risk
EG003
Migraine
Nervous system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Paraesthesia
Nervous system disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Proteinuria
Renal and urinary disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Bronchial hyperreactivity
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0002 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0012 affected20 at risk
EG0020 affected18 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0021 affected18 at risk
EG003
Upper respiratory tract congestion
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Wheezing
Respiratory, thoracic and mediastinal disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Decubitus ulcer
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0011 affected20 at risk
EG0020 affected18 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Skin irritation
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0001 affected62 at risk
EG0010 affected20 at risk
EG0020 affected18 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA (Unspecified)
Systematic Assessment
EG0000 affected62 at risk
EG0010 affected20 at risk
EG0021 affected18 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
Not provided
Results Disclosure Restriction on PI(s)?
No
Other Details
Not provided
Point of Contact
Title
Organization
Phone
Extension
Email
Study Director
Novartis pharmaceuticals
862-778-8300
Novartis.email@novartis.com
ID
Term
D019190
Iron Overload
Ancestor Terms
ID
Term
D019189
Iron Metabolism Disorders
D008659
Metabolic Diseases
D009750
Nutritional and Metabolic Diseases
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
D000077588
Deferasirox
Ancestor Terms
ID
Term
D001565
Benzoates
D000146
Acids, Carbocyclic
D002264
Carboxylic Acids
D009930
Organic Chemicals
D001555
Benzene Derivatives
D006841
Hydrocarbons, Aromatic
D006844
Hydrocarbons, Cyclic
D006838
Hydrocarbons
D014230
Triazoles
D001393
Azoles
D006573
Heterocyclic Compounds, 1-Ring
D006571
Heterocyclic Compounds
Browse Leaves
Not provided
Browse Branches
Not provided
Patient withdrew consent
FG0003 subjects
25
Asian
Title
Measurements
BG00010
Other
Title
Measurements
BG0004
21
10
OG00440
OG00517
7
ParticipantsOG00417
ParticipantsOG0057
Title
Measurements
OG00010
OG0010
OG0020
OG0030
OG0040
OG0050
Week 8, Somewhat dislike
ParticipantsOG00010
ParticipantsOG0017
ParticipantsOG0023
ParticipantsOG0037
ParticipantsOG00417
ParticipantsOG0057
Title
Measurements
OG0000
OG00128.6
OG0020
OG003
Week 8, Neither like or dislike
ParticipantsOG00010
ParticipantsOG0017
ParticipantsOG0023
ParticipantsOG0037
ParticipantsOG00417
ParticipantsOG0057
Title
Measurements
OG00020
OG00128.6
OG00233.3
OG003
Week 8, Somewhat like
ParticipantsOG00010
ParticipantsOG0017
ParticipantsOG0023
ParticipantsOG0037
ParticipantsOG00417
ParticipantsOG0057
Title
Measurements
OG00030
OG0010
OG0020
OG003
Week 8, Like extremely
ParticipantsOG00010
ParticipantsOG0017
ParticipantsOG0023
ParticipantsOG0037
ParticipantsOG00417
ParticipantsOG0057
Title
Measurements
OG00040
OG00142.9
OG00266.7
OG003
Week 12, Dislike extremely
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0023
ParticipantsOG0033
ParticipantsOG00423
ParticipantsOG00510
Title
Measurements
OG0000
OG0010
OG0020
OG003
Week 12, Somewhat dislike
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0023
ParticipantsOG0033
ParticipantsOG00423
ParticipantsOG00510
Title
Measurements
OG0000
OG00125
OG0020
OG003
Week 12, Neither like or dislike
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0023
ParticipantsOG0033
ParticipantsOG00423
ParticipantsOG00510
Title
Measurements
OG00016.7
OG00125
OG0020
OG003
Week 12, Somewhat like
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0023
ParticipantsOG0033
ParticipantsOG00423
ParticipantsOG00510
Title
Measurements
OG00033.3
OG0010
OG00266.7
OG003
Week 12, Like extremely
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0023
ParticipantsOG0033
ParticipantsOG00423
ParticipantsOG00510
Title
Measurements
OG00050
OG00150
OG00233.3
OG003
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.
OG002
Breakfast (Deferasirox With Liquid)
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.
OG003
Dinner (Deferasirox With Soft Food)
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.
OG004
Dinner (Deferasirox With Liquid)
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.
OG005
No Meal (Deferasirox With Liquid)
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.
OG006
No Meal Deferasirox With Soft Food)
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.