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

ClinicalTrials.gov processed this data on July 19, 2021. Link to the current ClinicalTrials.gov record.

Recruitment Status

COMPLETED - HAS RESULTS
 (See Contacts and Locations)
Verified July 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 16, 2009
First Posted: February 18, 2009
Results First Posted: July 20, 2021
Last Update Posted: July 20, 2021
Last Verified: July 2021
History of Changes

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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 Disease Intervention/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 DateMay 2009
Actual Primary Completion DateAugust 2010
Actual Study Completion DateAugust 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 [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 [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.
  2. 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.
  3. 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.

Eligibility Criteria

Ages Eligible for Study 2 Years and Older (Child, Adult, Older Adult)
Sexes Eligible for Study All
Accepts Healthy Volunteers No
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 Collaborators Novartis Pharmaceuticals
Locations
  • Children's Hospital and Research Center | Oakland, California, United States, 94609
  • Stanford University | Palo Alto, California, United States, 94304-1812
  • Bay Area Cancer Research Group | Pleasant Hill, California, United States, 94523
  • University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center | Aurora, Colorado, United States, 80045
  • Yale University School of Medicine | New Haven, Connecticut, United States, 06520
  • Medical College of Georgia | Augusta, Georgia, United States, 30912
  • Children's Memorial | Chicago, Illinois, United States, 60614
  • Tulane University Health Sciences Center | New Orleans, Louisiana, United States, 70118
  • University of Maryland Greenebaum Cancer Center | Baltimore, Maryland, United States, 21201
  • Children's Hospital of Boston | Boston, Massachusetts, United States, 02115
  • Boston Medical Center | Boston, Massachusetts, United States, 02118
  • Washington University School of Medicine | Saint Louis, Missouri, United States, 63110
  • The Cancer Center at Hackensack University Medical Center | Hackensack, New Jersey, United States, 07601
  • Cancer Institute of New Jersey | New Brunswick, New Jersey, United States, 08901
  • St Joseph Children's Hospital | Paterson, New Jersey, United States, 07503
  • Schneider Children's Hospital | New Hyde Park, New York, United States, 11040
  • New York Presbyterian Hospital | New York, New York, United States, 10065
  • New York Medical College | Valhalla, New York, United States, 10595
  • Wake Forest University Health Sciences | Winston-Salem, North Carolina, United States, 27157
  • University of Oklahoma | Oklahoma City, Oklahoma, United States, 73104
  • Penn State Children's Hospital | Hershey, Pennsylvania, United States, 17033
  • St Christopher's Hospital for Children | Philadelphia, Pennsylvania, United States, 19134
  • Texas Children's Cancer Center and Hematology Services | Houston, Texas, United States, 77030
Investigators

    More Information

    Additional Information

    Publications

    Additional Relevant MeSH Terms

    • Hemochromatosis
    • Hemosiderosis
    • Iron Overload
    • Iron Metabolism Disorders
    • Metabolic Diseases
    • Metal Metabolism, Inborn Errors
    • Metabolism, Inborn Errors
    • Genetic Diseases, Inborn