Condition category
Haematological Disorders
Date applied
23/07/2003
Date assigned
05/09/2003
Last edited
28/09/2012
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Elliot Vichinsky

ORCID ID

Contact details

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

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 measures

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

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

Results - basic reporting

Publication summary

Results:
1. http://www.ncbi.nlm.nih.gov/pubmed/16352812
2. http://www.ncbi.nlm.nih.gov/pubmed/18469351 (ancillary study)

Publication citations

  1. 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.

  2. 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.

Additional files

Editorial Notes