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

Primary Purpose

Transfusional Hemosiderosis

Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
deferasirox:
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Transfusional Hemosiderosis focused on measuring Exjade, deferasirox, palatability, tolerability, food, iron overload, Serum Iron overload due to transfusions

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital and Research Center
  • Stanford University
  • Bay Area Cancer Research Group
  • University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
  • Yale University School of Medicine
  • Medical College of Georgia
  • Children's Memorial
  • Tulane University Health Sciences Center
  • University of Maryland Greenebaum Cancer Center
  • Children's Hospital of Boston
  • Boston Medical Center
  • Washington University School of Medicine
  • The Cancer Center at Hackensack University Medical Center
  • Cancer Institute of New Jersey
  • St Joseph Children's Hospital
  • Schneider Children's Hospital
  • New York Presbyterian Hospital
  • New York Medical College
  • Wake Forest University Health Sciences
  • University of Oklahoma
  • Penn State Children's Hospital
  • St Christopher's Hospital for Children
  • Texas Children's Cancer Center and Hematology Services

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.

Outcomes

Primary Outcome Measures

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

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.
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.
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.

Full Information

First Posted
February 16, 2009
Last Updated
July 19, 2021
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00845871
Brief Title
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

2. Study Status

Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
May 2009 (undefined)
Primary Completion Date
August 2010 (Actual)
Study Completion Date
August 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transfusional Hemosiderosis
Keywords
Exjade, deferasirox, palatability, tolerability, food, iron overload, Serum Iron overload due to transfusions

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
65 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Arm Description
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Intervention Type
Drug
Intervention Name(s)
deferasirox:
Other Intervention Name(s)
Exjade, Deferasirox
Intervention Description
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Primary Outcome Measure Information:
Title
Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
Description
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.
Time Frame
Week 8 and Week 12
Secondary Outcome Measure Information:
Title
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
Description
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.
Time Frame
Day 1 up to Week 16
Title
Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16
Description
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.
Time Frame
Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
Title
Change From Baseline in Serum Ferritin at Week 16
Description
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.
Time Frame
Baseline, Week 16 (End of study)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital and Research Center
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304-1812
Country
United States
Facility Name
Bay Area Cancer Research Group
City
Pleasant Hill
State/Province
California
ZIP/Postal Code
94523
Country
United States
Facility Name
University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Yale University School of Medicine
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
Medical College of Georgia
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Children's Memorial
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614
Country
United States
Facility Name
Tulane University Health Sciences Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118
Country
United States
Facility Name
University of Maryland Greenebaum Cancer Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Children's Hospital of Boston
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Boston Medical Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
The Cancer Center at Hackensack University Medical Center
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
Cancer Institute of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
St Joseph Children's Hospital
City
Paterson
State/Province
New Jersey
ZIP/Postal Code
07503
Country
United States
Facility Name
Schneider Children's Hospital
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
New York Presbyterian Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
Wake Forest University Health Sciences
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
University of Oklahoma
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Penn State Children's Hospital
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
St Christopher's Hospital for Children
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19134
Country
United States
Facility Name
Texas Children's Cancer Center and Hematology Services
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
23637051
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.
Results Reference
derived
Links:
URL
http://www.novartisclinicaltrials.com/etrials/searchTrial.do?trialID=717
Description
Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.

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

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