Clinical trial NCT00171210: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
Official title: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Transfusional Iron Overload in β-thalassemia
Keywords: β-thalassemia
iron overload
deferasirox
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2005-09-15
Enrollment: 506
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A 1-year randomized Phase III core trial (NCT00061750) using deferoxamine as the comparator was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older. Patients who successfully completed this main trial may continue in this extension trial to receive chelation therapy with deferasirox for an additional 4 years.

The objective of this study is to assess the efficacy and long-term safety of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.


Eligibility criteria: Inclusion criteria

* Patients who completed the 12-month core study (NCT00061750)
* Female patients after menarche and who were sexually active, if they used double-barrier contraception, oral contraceptive plus barrier contraceptive, or had undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
* Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation

Exclusion criteria

* Pregnant or breast feeding patients
* Patients with a history of non-compliance to medical regimens or those considered to be potentially unreliable

Primary outcomes:
measure: Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
timeFrame: up to 5 years
Secondary outcomes:
measure: Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
measure: Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study
timeFrame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Oakland
country: United States
facility: Children's Hospital and Research Center at Oakland
state: California
city: Stanford
country: United States
facility: Stanford Hospital, Division of Oncology
state: California
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Boston
country: United States
facility: Children's Hospital Boston, Dept of Hematology
state: Massachusetts
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Buenos Aires
country: Argentina
facility: Novartis Investigative Site
city: Cordoba
country: Argentina
facility: Novartis Investigative Site
city: Bruxelles
country: Belgium
facility: Novartis Investigative Site
city: Laken
country: Belgium
facility: Novartis Investigative Site
city: Liege
country: Belgium
facility: Novartis Investigative Site
city: Mons
country: Belgium
facility: Novartis Investigative Site
city: Campinas
country: Brazil
facility: Novartis Investigative Site
city: Sao Paolo
country: Brazil
facility: Novartis Investigative Site
city: Montreal
country: Canada
facility: Novartis Investigative Site
city: Toronto
country: Canada
facility: Novartis Investigative Site
city: Creteil
country: France
facility: Novartis Investigative Site
city: Marseille
country: France
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Pierre-Benite
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Duesseldorf
country: Germany
facility: Novartis Investigative Site
city: Frankfurt
country: Germany
facility: Novartis Investigative Site
city: Hamburg
country: Germany
facility: Novartis Investigative Site
city: Ulm
country: Germany
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
city: Ioannina
country: Greece
facility: Novartis Investigative Site
city: Patras
country: Greece
facility: Novartis Investigative Site
city: Thessaloniki
country: Greece
facility: Novartis Investigative Site
city: Brindisi
country: Italy
facility: Novartis Investigative Site
city: Cagliari
country: Italy
facility: Novartis Investigative Site
city: Catania
country: Italy
facility: Novartis Investigative Site
city: Ferrara
country: Italy
facility: Novartis Investigative Site
city: Genova
country: Italy
facility: Novartis Investigative Site
city: Milan
country: Italy
facility: Novartis Investigative Site
city: Monza
country: Italy
facility: Novartis Investigative Site
city: Naples
country: Italy
facility: Novartis Investigative Site
city: Palermo
country: Italy
facility: Novartis Investigative Site
city: Pavia
country: Italy
facility: Novartis Investigative Site
city: Roma
country: Italy
facility: Novartis Investigative Site
city: Sassari
country: Italy
facility: Novartis Investigative Site
city: Siracusa
country: Italy
facility: Novartis Investigative Site
city: Turin
country: Italy
facility: Novartis Investigative Site
city: Tunis
country: Tunisia
facility: Novartis Investigative Site
city: Adana
country: Turkey
facility: Novartis Investigative Site
city: Isparta
country: Turkey
facility: Novartis Investigative Site
city: Istanbul
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood. 2011 Jul 28;118(4):884-93. doi: 10.1182/blood-2010-11-316646. Epub 2011 May 31.