Clinical trial NCT00390858: A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
Official title: A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Transfusional Iron Overload
β-thalassemia Major
Pediatric Rare Anemia
Keywords: β-thalassemia major
iron overload
deferasirox
pediatric rare anemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2006-10-20
Enrollment: 40
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.


Eligibility criteria: Inclusion Criteria:

* Completion of the planned 12-month core trial, (NCT00390858).
* Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
* Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation.

Exclusion Criteria:

* Pregnant or breast feeding patients
* Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.

Other protocol-defined exclusion criteria may apply.

Primary outcomes:
measure: Participants With Adverse Events by Primary System Organ Class (SOC)
timeFrame: 4 year extension + core 1 year
measure: Change in Liver Iron Concentration (LIC)
timeFrame: Baseline of Core Study to End of Extension Study, up to 5 years.
Secondary outcomes:
measure: Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)
timeFrame: Baseline of Core Study to End of Extension Study, up to 5 years
measure: Relative Change in Serum Ferritin Level
timeFrame: Baseline of Core Study to Extension 18 months, up to 2.5 years.
Other outcomes:
Locations:
city: Lyon
country: France
facility: Novartis Investigative Site
city: Cagliari
country: Italy
facility: Novartis Investigative Site
city: Genova
country: Italy
facility: Novartis Investigative Site
city: Torino
country: Italy
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Dr. Gianluca Forni
role: STUDY_DIRECTOR
affiliation: Novartis Pharmaceuticals
name: Prof. Renzo Galanello
role: STUDY_DIRECTOR
affiliation: Novartis Pharmaceuticals
name: Prof. Antonio Piga
role: STUDY_DIRECTOR
affiliation: Novartis Pharmaceuticals
name: Dr. Yves Bertrand
role: STUDY_DIRECTOR
References: