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

Recruitment Status
COMPLETED - HAS RESULTS
(See Contacts and Locations)Verified June 2021 by Novartis Pharmaceuticals
Sponsor
Novartis Pharmaceuticals
Information Provided by (Responsible Party)
Novartis Pharmaceuticals
Clinicaltrials.gov Identifier
NCT00845871
Other Study ID Numbers:
CICL670AUS32
First Submitted
February 15, 2009
First Posted
February 17, 2009
Results First Posted
May 10, 2021
Last Update Posted
July 19, 2021
Last Verified
June 2021

ClinicalTrials.gov processed this data on July 2021. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study 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.

Condition or DiseaseIntervention/Treatment
Transfusional Hemosiderosis
Drug: deferasirox:

Study Design

Study TypeInterventional
Actual Enrollment65 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleA 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 DateApril 30, 2009
Actual Primary Completion DateJuly 31, 2010
Actual Study Completion DateJuly 31, 2010

Groups and Cohorts

Group/CohortIntervention/Treatment
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.

Outcome Measures

Primary Outcome Measures
  1. 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.
Secondary Outcome Measures
  1. 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.
  2. 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.
  3. 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.

Eligibility Criteria

Ages Eligible for Study(Child, Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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
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

Sponsors and CollaboratorsNovartis Pharmaceuticals
Locations
Children's Hospital and Research Center | Oakland California, United States, 94609Stanford University | Palo Alto California, United States, 94304-1812Bay Area Cancer Research Group | Pleasant Hill California, United States, 94523University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center | Aurora Colorado, United States, 80045Yale University School of Medicine | New Haven Connecticut, United States, 06520Medical College of Georgia | Augusta Georgia, United States, 30912Children's Memorial | Chicago Illinois, United States, 60614Tulane University Health Sciences Center | New Orleans Louisiana, United States, 70118University of Maryland Greenebaum Cancer Center | Baltimore Maryland, United States, 21201Children's Hospital of Boston | Boston Massachusetts, United States, 02115Boston Medical Center | Boston Massachusetts, United States, 02118Washington University School of Medicine | St Louis Missouri, United States, 63110The Cancer Center at Hackensack University Medical Center | Hackensack New Jersey, United States, 07601Cancer Institute of New Jersey | New Brunswick New Jersey, United States, 08901St Joseph Children's Hospital | Paterson New Jersey, United States, 07503Schneider Children's Hospital | New Hyde Park New York, United States, 11040New York Presbyterian Hospital | New York New York, United States, 10065New York Medical College | Valhalla New York, United States, 10595Wake Forest University Health Sciences | Winston-Salem North Carolina, United States, 27157University of Oklahoma | Oklahoma City Oklahoma, United States, 73104Penn State Children's Hospital | Hershey Pennsylvania, United States, 17033St Christopher's Hospital for Children | Philadelphia Pennsylvania, United States, 19134Texas Children's Cancer Center and Hematology Services | Houston Texas, United States, 77030
Investigators
Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals