Clinical trial NCT00845871: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Transfusional Hemosiderosis
Keywords: Exjade
deferasirox
palatability
tolerability
food
iron overload
Serum Iron overload due to transfusions
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-02-18
Enrollment: 65
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
timeFrame: Week 8 and Week 12
Secondary outcomes:
measure: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
timeFrame: Day 1 up to Week 16
measure: Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16
timeFrame: Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
measure: Change From Baseline in Serum Ferritin at Week 16
timeFrame: Baseline, Week 16 (End of study)
Other outcomes:
Locations:
city: Oakland
country: United States
facility: Children's Hospital and Research Center
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Pleasant Hill
country: United States
facility: Bay Area Cancer Research Group
state: California
city: Aurora
country: United States
facility: University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
state: Colorado
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Augusta
country: United States
facility: Medical College of Georgia
state: Georgia
city: Chicago
country: United States
facility: Children's Memorial
state: Illinois
city: New Orleans
country: United States
facility: Tulane University Health Sciences Center
state: Louisiana
city: Baltimore
country: United States
facility: University of Maryland Greenebaum Cancer Center
state: Maryland
city: Boston
country: United States
facility: Children's Hospital of Boston
state: Massachusetts
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Hackensack
country: United States
facility: The Cancer Center at Hackensack University Medical Center
state: New Jersey
city: New Brunswick
country: United States
facility: Cancer Institute of New Jersey
state: New Jersey
city: Paterson
country: United States
facility: St Joseph Children's Hospital
state: New Jersey
city: New Hyde Park
country: United States
facility: Schneider Children's Hospital
state: New York
city: New York
country: United States
facility: New York Presbyterian Hospital
state: New York
city: Valhalla
country: United States
facility: New York Medical College
state: New York
city: Winston-Salem
country: United States
facility: Wake Forest University Health Sciences
state: North Carolina
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Hershey
country: United States
facility: Penn State Children's Hospital
state: Pennsylvania
city: Philadelphia
country: United States
facility: St Christopher's Hospital for Children
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Cancer Center and Hematology Services
state: Texas
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: 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.