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Completed

NCT Number: NCT00845871

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

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.

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Key information

Conditions

Transfusional Hemosiderosis Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Children's Hospital and Research Center, Oakland, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

deferasirox:

Drug

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

Other names: Exjade, Deferasirox

Primary outcomes

  1. Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12

    Time frame: 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 outcomes

  1. Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption

    Time frame: 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

    Time frame: 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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2009
Primary completion
2010
Study completion
2010
First posted
Feb 18, 2009
Registry last updated
Jul 20, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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