Transfusional HemosiderosisPhase 4NCT00845871

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

A Phase 4 study testing deferasirox: in people with Transfusional Hemosiderosis, run by Novartis Pharmaceuticals at 23 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 2 years and older
Conditions the study is for: Transfusional Hemosiderosis
What is being tested?: deferasirox:
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 65 participants
Where: 23 sitesUnited States
Study dates: May 2009to Aug 2010
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 2 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

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).

Who cannot join

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.

Trial locations and contacts

23 sites in 1 country.

City, postcode or hospital

23 sites in 1 country

United States23 sites

Medical College of Georgia
Augusta, Georgia, 30912
University of Colorado Denver, Colorado Sickle Cell Treatment and Research Center
Aurora, Colorado, 80045
University of Maryland Greenebaum Cancer Center
Baltimore, Maryland, 21201
Children's Hospital of Boston
Boston, Massachusetts, 02115
Boston Medical Center
Boston, Massachusetts, 02118
Children's Memorial
Chicago, Illinois, 60614
The Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, 07601
Penn State Children's Hospital
Hershey, Pennsylvania, 17033
Texas Children's Cancer Center and Hematology Services
Houston, Texas, 77030
Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Yale University School of Medicine
New Haven, Connecticut, 06520
Schneider Children's Hospital
New Hyde Park, New York, 11040
Tulane University Health Sciences Center
New Orleans, Louisiana, 70118
New York Presbyterian Hospital
New York, New York, 10065
Children's Hospital and Research Center
Oakland, California, 94609
University of Oklahoma
Oklahoma City, Oklahoma, 73104
Stanford University
Palo Alto, California, 94304-1812
St Joseph Children's Hospital
Paterson, New Jersey, 07503
St Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
Bay Area Cancer Research Group
Pleasant Hill, California, 94523
Washington University School of Medicine
St Louis, Missouri, 63110
New York Medical College
Valhalla, New York, 10595
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157

Other trials in United States

About this study

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

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.

Read the 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.

Conditions: Transfusional Hemosiderosis
Being tested: deferasirox:
Keywords: Exjade, deferasirox, palatability, tolerability, food, iron overload, Serum Iron overload due to transfusions
Medical subject headings: Iron Overload

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

deferasirox:Drug
Also called Exjade, Deferasirox
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.

Study groups in NCT00845871
Study group	Role	What they receive
Deferasirox	Experimental	Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day. Receives: Drug: deferasirox:

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Week 8 and Week 12

Secondary outcomes (3)

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.
Measured: Day 1 up to Week 16
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.
Measured: Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
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.
Measured: Baseline, Week 16 (End of study)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2009Aug 2010

First posted18 Feb 2009 (estimated)
Study startMay 2009
Primary completionAug 2010
Study completionAug 2010

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Transfusional HemosiderosisStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 65 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00845871
Sponsor's protocol ID: CICL670AUS32
Other IDs: 2011-004217-17
Status last verified: Jul 2021
First submitted: 16 Feb 2009
Last updated: 20 Jul 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

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.
PubMed 23637051

Study at a glance

From the registry record, last updated by the sponsor on 20 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT00845871
Acronym / sponsor ID: CICL670AUS32
Conditions: Transfusional Hemosiderosis
Keywords: Exjade, deferasirox, palatability, tolerability, food, iron overload, Serum Iron overload due to transfusions
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 65 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: May 2009
Primary completion: Aug 2010 (actual)
Study completion: Aug 2010 (actual)
First posted: 18 Feb 2009 (estimated)
Results: Posted 20 Jul 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Deferasirox	Experimental	Drug: deferasirox:Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.

Who may qualify

Ages: 2 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

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).

Key exclusion criteria (2 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
deferasirox:Also: Exjade, DeferasiroxParticipants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.	Drug	Deferasirox

Primary outcome

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.

3 secondary outcomes

Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
Time frame: Day 1 up to Week 16
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
Change From Baseline in Serum Ferritin at Week 16
Time frame: Baseline, Week 16 (End of study)

Results and safety

Results posted to the registry on 20 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Deferasirox	65	65

Primary outcome: Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12

Time frame: Week 8 and Week 12. Number. Unit: percentage of participants

Measure	Breakfast (Deferasirox With Soft Food) n = 16	Breakfast (Deferasirox With Liquid) n = 11	Dinner (Deferasirox With Soft Food) n = 6	Dinner (Deferasirox With Liquid) n = 10	No Meal (Deferasirox With Liquid) n = 40	No Meal Deferasirox With Soft Food) n = 17
Week 8, Dislike extremely	10	0	0	0	0	0
Week 8, Somewhat dislike	0	28.6	0	14.3	5.9	14.3
Week 8, Neither like or dislike	20	28.6	33.3	28.6	29.4	28.6
Week 8, Somewhat like	30	0	0	28.6	17.6	42.9
Week 8, Like extremely	40	42.9	66.7	28.6	47.1	14.3
Week 12, Dislike extremely	0	0	0	0	4.3	0
Week 12, Somewhat dislike	0	25	0	0	4.3	10
Week 12, Neither like or dislike	16.7	25	0	33.3	56.5	50

Population analysed: The primary analysis was performed in the ITT population defined as all subjects enrolled regardless of any treatment received or not. As per the planned analysis, data were summarized by counts and percentages of each category of response when administered with food or with different methods of administration.

3 secondary outcomes are reported in the full results.

Adverse events

Time frame: First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV), up to 16 weeks. Other adverse events reported at 4.90% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Deferasirox (Run-in Phase)	5 of 62 (8.1%)	30 of 62 (48.4%)	0 of 62
Breakfast (Deferasirox With Soft Food)	5 of 20 (25%)	10 of 20 (50%)	0 of 20
Breakfast (Deferasirox With Liquid)	0 of 18 (0%)	6 of 18 (33.3%)	0 of 18
Dinner (Deferasirox With Soft Food)	1 of 14 (7.1%)	6 of 14 (42.9%)	0 of 14
Dinner (Deferasirox With Liquid)	1 of 17 (5.9%)	6 of 17 (35.3%)	0 of 17
No Meal (Deferasirox With Liquid)	6 of 39 (15.4%)	18 of 39 (46.2%)	0 of 39
No Meal (Deferasirox With Soft Food)	0 of 30 (0%)	9 of 30 (30%)	0 of 30
Deferasirox (Overall)	15 of 65 (23.1%)	52 of 65 (80%)	0 of 65

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 23637051Goldberg 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.
Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.Link provided by the sponsor.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Novartis Pharmaceuticals study director

Novartis Pharmaceuticals

23 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing deferasirox: in people with Transfusional Hemosiderosis, run by Novartis Pharmaceuticals at 23 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 23 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Jul 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 (time frame: Week 8 and Week 12). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00845871, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2021.