A randomised, comparative, open label phase III trial on efficacy and safety of long-term treatment with ICL670 (5 to 40 mg/kg/day) in comparison with deferoxamine (DFO) (20 to 60 mg/kg/day) in β-thalassaemia patients with transfusional haemosiderosis

ISRCTN	ISRCTN93355192
DOI	https://doi.org/10.1186/ISRCTN93355192
ClinicalTrials.gov number	NCT00061750
Secondary identifying numbers	CICL670 0107

Submission date: 23/07/2003
Registration date: 05/09/2003
Last edited: 23/05/2022

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Elliot Vichinsky
Scientific

Children's Hospital & Research Center at Oakland
747 52nd Street
OPC-PCRC, 1st Floor
Oakland
94609-1809
United States of America

Study information

Study design	Randomised controlled trial
Primary study design	Interventional
Secondary study design	Randomised controlled trial
Study setting(s)	Not specified
Study type	Treatment
Scientific title	A randomised, comparative, open label phase III trial on efficacy and safety of long-term treatment with ICL670 (5 to 40 mg/kg/day) in comparison with deferoxamine (DFO) (20 to 60 mg/kg/day) in β-thalassaemia patients with transfusional haemosiderosis
Study acronym	ICL107
Study objectives	This study was undertaken to investigate the hypothesis that deferasirox (ICL670) was noninferior to deferoxamine (DFO).
Ethics approval(s)	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.
Health condition(s) or problem(s) studied	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
Pharmaceutical study type(s)
Phase	Phase III
Drug / device / biological / vaccine name(s)	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 study start date	01/03/2003
Completion date	01/11/2003

Eligibility

Participant type(s)	Patient
Age group	Not Specified
Sex	Both
Target number of participants	586
Key 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
Key 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
Date of first enrolment	01/03/2003
Date of final enrolment	01/11/2003

Locations

Countries of recruitment

Argentina
Belgium
Brazil
Canada
France
Germany
Greece
Italy
Tunisia
Türkiye
United Kingdom
United States of America

Study participating centre

Children's Hospital & Research Center at Oakland

Oakland
94609-1809
United States of America

Sponsor information

Novartis Pharmaceuticals Corporation (USA)
Industry

One Health Plaza
East Hanover
07936
United States of America

Phone	+1 862 778 7042
Email	jerry.retkwa@pharma.novartis.com
"ROR"	https://ror.org/028fhxy95

Funders

Funder type

Industry

Novartis Pharmaceuticals Corporation (USA)

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
Publication and dissemination plan	Not provided at time of registration
IPD sharing plan	Not provided at time of registration

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Other publications	A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia	01/05/2006		Yes	No
Other publications	Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial	01/06/2008		Yes	No
Results article		15/01/2008	23/05/2022	Yes	No

Editorial Notes

23/05/2022: Publication reference added.