Additional identifiers
EudraCT number
ClinicalTrials.gov number
NCT00061750
Protocol/serial number
CICL670 0107
Study information
Scientific title
Acronym
ICL107
Study hypothesis
This study was undertaken to investigate the hypothesis that deferasirox (ICL670) was noninferior to deferoxamine (DFO).
Ethics approval
This trial was conducted in accordance with good clinical practices. Institutional review board or ethics committee approval was obtained at each participating institution and written informed consent was obtained from all patients or their legal guardians prior to participation in any study procedures.
Study design
Randomised controlled trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
β-thalassaemia
Intervention
Patients meeting the eligibility requirements were randomised to receive deferasirox or deferoxamine. Randomisation was stratified by age groups:
1. 2 to younger than 12 years
2. 12 to younger than 18 years
3. 18 years or older
After randomisation, patients were assigned by the investigator to a dose dependent on their baseline liver iron concentrations (LIC). Once-daily treatment with deferasirox at the assigned dose was administered as a suspension in water half an hour prior to breakfast 7 days a week. Deferoxamine was administered as a slow subcutaneous infusion using electronic Microject Chrono infusion pumps (Cane Medical Technology, Torino, Italy) over 8 to 12 hours, 5 days a week.
Treatment with either therapy was continued for 1 year.
Intervention type
Drug
Phase
Phase III
Drug names
Deferasirox (ICL670), Deferoxamine (DFO)
Primary outcome measure
Maintenance or reduction of LIC.
Secondary outcome measures
1. Safety and tolerability
2. Change in serum ferritin level
3. Net body iron balance
Overall trial start date
01/03/2003
Overall trial end date
01/11/2003
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
1. β-thalassaemia outpatients 2 years old or greater
2. Transfusional haemosiderosis
3. Previously treated with DFO, or never treated with any iron chelator
4. Without any contra-indications to either trial medication
Participant type
Patient
Age group
Not Specified
Gender
Both
Target number of participants
586
Participant exclusion criteria
1. Alanine aminotransferase (ALT) level greater than 250 U/L during the year prior to enrolment
2. Chronic hepatitis B infection
3. Active hepatitis C infection
4. A history of a positive human immunodeficiency virus (HIV) test
5. Serum creatinine above the upper limit of normal (ULN)
6. A urinary protein-creatinine ratio of greater than 0.5 mg/mg
7. Nephrotic syndrome
8. Uncontrolled systemic hypertension
9. A prolonged corrected QT interval
10. Systemic infection within the 10 days prior to entry
11. Gastrointestinal conditions preventing absorption of an oral medication
12. Concomitant conditions preventing therapy with deferasirox or deferoxamine
13. A history of ocular toxicity related to iron chelation therapy
14. A poor response to deferoxamine
15. Noncompliance with prescribed therapy
Recruitment start date
01/03/2003
Recruitment end date
01/11/2003
Locations
Countries of recruitment
Argentina, Belgium, Brazil, Canada, France, Germany, Greece, Italy, Tunisia, Turkey, United Kingdom, United States of America
Trial participating centre
Children's Hospital & Research Center at Oakland
Oakland
94609-1809
United States of America
Sponsor information
Organisation
Novartis Pharmaceuticals Corporation (USA)
Sponsor details
One Health Plaza
East Hanover
07936
United States of America
+1 862 778 7042
jerry.retkwa@pharma.novartis.com
Sponsor type
Industry
Website
Funders
Funder type
Industry
Funder name
Novartis Pharmaceuticals Corporation (USA)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list
Results:
1. http://www.ncbi.nlm.nih.gov/pubmed/16352812
2. http://www.ncbi.nlm.nih.gov/pubmed/18469351 (ancillary study)
Publication citations
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Cappellini MD, Cohen A, Piga A, Bejaoui M, Perrotta S, Agaoglu L, Aydinok Y, Kattamis A, Kilinc Y, Porter J, Capra M, Galanello R, Fattoum S, Drelichman G, Magnano C, Verissimo M, Athanassiou-Metaxa M, Giardina P, Kourakli-Symeonidis A, Janka-Schaub G, Coates T, Vermylen C, Olivieri N, Thuret I, Opitz H, Ressayre-Djaffer C, Marks P, Alberti D, A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia., Blood, 2006, 107, 9, 3455-3462, doi: 10.1182/blood-2005-08-3430.
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Walter PB, Macklin EA, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Vichinsky E, Olivieri N, Alberti D, Holland J, Harmatz P, , Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial., Haematologica, 2008, 93, 6, 817-825, doi: 10.3324/haematol.11755.