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

Phase 4

Completed

Conditions: Transfusional Hemosiderosis

Registration Number: NCT00845871

Lead Sponsor: Novartis Pharmaceuticals

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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 65

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.

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.
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.
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.

Trial Locations

Locations (23): Children's Hospital and Research Center
🇺🇸
Oakland, California, United States
Schneider Children's Hospital
🇺🇸
New Hyde Park, New York, United States
University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
🇺🇸
Aurora, Colorado, United States
Stanford University
🇺🇸
Palo Alto, California, United States
St Joseph Children's Hospital
🇺🇸
Paterson, New Jersey, United States
Yale University School of Medicine
🇺🇸
New Haven, Connecticut, United States
Bay Area Cancer Research Group
🇺🇸
Pleasant Hill, California, United States
The Cancer Center at Hackensack University Medical Center
🇺🇸
Hackensack, New Jersey, United States
Cancer Institute of New Jersey
🇺🇸
New Brunswick, New Jersey, United States
St Christopher's Hospital for Children
🇺🇸
Philadelphia, Pennsylvania, United States
New York Presbyterian Hospital
🇺🇸
New York, New York, United States
Texas Children's Cancer Center and Hematology Services
🇺🇸
Houston, Texas, United States
New York Medical College
🇺🇸
Valhalla, New York, United States
Medical College of Georgia
🇺🇸
Augusta, Georgia, United States
Tulane University Health Sciences Center
🇺🇸
New Orleans, Louisiana, United States
Children's Memorial
🇺🇸
Chicago, Illinois, United States
Boston Medical Center
🇺🇸
Boston, Massachusetts, United States
Wake Forest University Health Sciences
🇺🇸
Winston-Salem, North Carolina, United States
University of Maryland Greenebaum Cancer Center
🇺🇸
Baltimore, Maryland, United States
University of Oklahoma
🇺🇸
Oklahoma City, Oklahoma, United States
Children's Hospital of Boston
🇺🇸
Boston, Massachusetts, United States
Washington University School of Medicine
🇺🇸
Saint Louis, Missouri, United States
Penn State Children's Hospital
🇺🇸
Hershey, Pennsylvania, United States