Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food
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
2 other identifiers
interventional
65
1 country
23
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started May 2009
23 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 16, 2009
CompletedFirst Posted
Study publicly available on registry
February 18, 2009
CompletedStudy Start
First participant enrolled
May 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2010
CompletedResults Posted
Study results publicly available
July 20, 2021
CompletedJuly 20, 2021
July 1, 2021
1.3 years
February 16, 2009
May 11, 2021
July 19, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (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.
Week 8 and Week 12
Secondary Outcomes (3)
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
Day 1 up to Week 16
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
Change From Baseline in Serum Ferritin at Week 16
Baseline, Week 16 (End of study)
Study Arms (1)
Deferasirox
EXPERIMENTALParticipants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Interventions
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Eligibility Criteria
You may qualify if:
- 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).
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (23)
Children's Hospital and Research Center
Oakland, California, 94609, United States
Stanford University
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
Related Publications (1)
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.
PMID: 23637051DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 16, 2009
First Posted
February 18, 2009
Study Start
May 1, 2009
Primary Completion
August 1, 2010
Study Completion
August 1, 2010
Last Updated
July 20, 2021
Results First Posted
July 20, 2021
Record last verified: 2021-07