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

Transfusional Hemosiderosis Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00845871
• Other study ID #: CICL670AUS32
• Secondary ID: 2011-004217-17
• Status: Completed
• Phase: Phase 4
• Start date: May 2009
• Est. completion date: August 2010

Study information

• Verified date: July 2021
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 65
• Est. completion date: August 2010
• Est. primary completion date: August 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemosiderosis
• Transfusional Hemosiderosis

Intervention

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

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Week 8 and Week 12
Secondary	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.	Day 1 up to Week 16
Secondary	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.	Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
Secondary	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.	Baseline, Week 16 (End of study)