Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food

Sponsor
Novartis Pharmaceuticals (Industry)
Overall Status
Completed
CT.gov ID
NCT00845871
Collaborator
(none)
65
Enrollment
23
Locations
1
Arm
15
Duration (Months)
2.8
Patients Per Site
0.2
Patients Per Site Per Month

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 DiseaseIntervention/TreatmentPhase
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

Study Type:
Interventional
Actual Enrollment :
65 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
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
Study Start Date :
May 1, 2009
Actual Primary Completion Date :
Aug 1, 2010
Actual Study Completion Date :
Aug 1, 2010

Arms and Interventions

ArmIntervention/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:
  • Exjade
  • Deferasirox
  • Outcome Measures

    Primary Outcome Measures

    1. 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

    1. 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.

    2. 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.

    3. 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

    Ages Eligible for Study:
    2 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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

    SiteCityStateCountryPostal Code
    1Children's Hospital and Research CenterOaklandCaliforniaUnited States94609
    2Stanford UniversityPalo AltoCaliforniaUnited States94304-1812
    3Bay Area Cancer Research GroupPleasant HillCaliforniaUnited States94523
    4University of Colorado Denver, Colorado Sickle Cell Treatment and Research CenterAuroraColoradoUnited States80045
    5Yale University School of MedicineNew HavenConnecticutUnited States06520
    6Medical College of GeorgiaAugustaGeorgiaUnited States30912
    7Children's MemorialChicagoIllinoisUnited States60614
    8Tulane University Health Sciences CenterNew OrleansLouisianaUnited States70118
    9University of Maryland Greenebaum Cancer CenterBaltimoreMarylandUnited States21201
    10Children's Hospital of BostonBostonMassachusettsUnited States02115
    11Boston Medical CenterBostonMassachusettsUnited States02118
    12Washington University School of MedicineSaint LouisMissouriUnited States63110
    13The Cancer Center at Hackensack University Medical CenterHackensackNew JerseyUnited States07601
    14Cancer Institute of New JerseyNew BrunswickNew JerseyUnited States08901
    15St Joseph Children's HospitalPatersonNew JerseyUnited States07503
    16Schneider Children's HospitalNew Hyde ParkNew YorkUnited States11040
    17New York Presbyterian HospitalNew YorkNew YorkUnited States10065
    18New York Medical CollegeValhallaNew YorkUnited States10595
    19Wake Forest University Health SciencesWinston-SalemNorth CarolinaUnited States27157
    20University of OklahomaOklahoma CityOklahomaUnited States73104
    21Penn State Children's HospitalHersheyPennsylvaniaUnited States17033
    22St Christopher's Hospital for ChildrenPhiladelphiaPennsylvaniaUnited States19134
    23Texas Children's Cancer Center and Hematology ServicesHoustonTexasUnited States77030

    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.
    Responsible Party:
    Novartis Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT00845871
    Other Study ID Numbers:
    • CICL670AUS32
    • 2011-004217-17
    First Posted:
    Feb 18, 2009
    Last Update Posted:
    Jul 20, 2021
    Last Verified:
    Jul 1, 2021

    Study Results

    Participant Flow

    Recruitment DetailsThe study was conducted at 17 centres in United States.
    Pre-assignment Detail82 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 TitleDeferasirox
    Arm/Group DescriptionParticipants 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
    STARTED65
    COMPLETED65
    NOT COMPLETED0
    Period Title: Run-in Period
    STARTED65
    COMPLETED58
    NOT COMPLETED7

    Baseline Characteristics

    Arm/Group TitleDeferasirox
    Arm/Group DescriptionParticipants 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 Participants65
    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

    1. Primary Outcome
    TitlePercentage of Participants With Differing Palatability Scores at Week 8 and Week 12
    DescriptionPalatability 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 FrameWeek 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 TitleBreakfast (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 DescriptionParticipants 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 Participants16116104017
    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
    2. Secondary Outcome
    TitleNumber of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
    DescriptionAdverse 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 FrameDay 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 TitleDeferasirox (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 DescriptionParticipants 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 Participants62201814173930
    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
    3. Secondary Outcome
    TitleTrough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16
    DescriptionBlood 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 FramePre-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 TitleDeferasirox
    Arm/Group DescriptionParticipants 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 Participants57
    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
    4. Secondary Outcome
    TitleChange From Baseline in Serum Ferritin at Week 16
    DescriptionFerritin 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 FrameBaseline, 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 TitleDeferasirox
    Arm/Group DescriptionParticipants 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 Participants64
    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 FrameFirst 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 TitleDeferasirox (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 DescriptionParticipants 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 (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# Events
    Total0/62 (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 (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# Events
    Total5/62 (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 syndrome1/62 (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 crisis3/62 (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 acute1/62 (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 complication0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Pyrexia1/62 (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
    Cholelithiasis1/62 (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
    Influenza0/62 (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 abscess0/62 (0%) 0/20 (0%) 0/18 (0%) 1/14 (7.1%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Pneumonia1/62 (1.6%) 0/20 (0%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Sepsis0/62 (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 infection0/62 (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
    Hypotension0/62 (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 (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# EventsAffected / at Risk (%)# Events
    Total30/62 (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
    Thrombocytopenia0/62 (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
    Palpitations0/62 (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 icterus1/62 (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%)
    Photophobia0/62 (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 distension0/62 (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 pain6/62 (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 upper2/62 (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%)
    Constipation1/62 (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%)
    Diarrhoea15/62 (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 hypermotility0/62 (0%) 0/20 (0%) 1/18 (5.6%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Nausea9/62 (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%)
    Vomiting4/62 (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
    Asthenia1/62 (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 pain0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Chest discomfort0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Chest pain0/62 (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 decreased0/62 (0%) 0/20 (0%) 1/18 (5.6%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Fatigue2/62 (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 peripheral0/62 (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%)
    Pyrexia7/62 (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
    Hypersensitivity0/62 (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
    Bronchiolitis0/62 (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 media1/62 (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 infection2/62 (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 infection1/62 (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 bite0/62 (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%)
    Contusion0/62 (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 increased2/62 (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 increased0/62 (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 increased0/62 (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 decreased0/62 (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 decreased0/62 (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 increased0/62 (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
    Anorexia3/62 (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%)
    Hypokalaemia1/62 (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%)
    Hypomagnesaemia0/62 (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
    Arthralgia0/62 (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 pain0/62 (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 lesion0/62 (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 pain0/62 (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 pain0/62 (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 extremity1/62 (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 erythematosus0/62 (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
    Dizziness0/62 (0%) 0/20 (0%) 2/18 (11.1%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 2/65 (3.1%)
    Headache5/62 (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%)
    Migraine0/62 (0%) 0/20 (0%) 0/18 (0%) 0/14 (0%) 1/17 (5.9%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Paraesthesia0/62 (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
    Insomnia0/62 (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
    Pollakiuria0/62 (0%) 0/20 (0%) 1/18 (5.6%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Proteinuria0/62 (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
    Asthma0/62 (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 hyperreactivity0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Cough2/62 (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%)
    Dyspnoea1/62 (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 pain1/62 (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%)
    Rhinorrhoea0/62 (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 congestion0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Wheezing0/62 (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 ulcer0/62 (0%) 0/20 (0%) 0/18 (0%) 1/14 (7.1%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Ecchymosis0/62 (0%) 0/20 (0%) 1/18 (5.6%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Eczema0/62 (0%) 1/20 (5%) 0/18 (0%) 0/14 (0%) 0/17 (0%) 0/39 (0%) 0/30 (0%) 1/65 (1.5%)
    Pruritus0/62 (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 irritation1/62 (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%)
    Urticaria0/62 (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

    [Not Specified]

    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/TitleStudy Director
    OrganizationNovartis pharmaceuticals
    Phone862-778-8300
    EmailNovartis.email@novartis.com
    Responsible Party:
    Novartis Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT00845871
    Other Study ID Numbers:
    • CICL670AUS32
    • 2011-004217-17
    First Posted:
    Feb 18, 2009
    Last Update Posted:
    Jul 20, 2021
    Last Verified:
    Jul 1, 2021