Phase 2 N=40 Treatment

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.

Transfusional Iron Overload · β-thalassemia Major · Pediatric Rare Anemia

Bottom Line

View on ClinicalTrials.gov: NCT00390858 ↗

Enrolled (actual)

Serious AEs

30.0%

Results posted

Aug 2011

Primary outcome: Primary: Participants With Adverse Events by Primary System Organ Class (SOC) — 20; 20; 19; 20 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Deferasirox (Drug)
Age: Pediatric, Adult · 1+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Feb 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Participants With Adverse Events by Primary System Organ Class (SOC)	20; 20; 19; 20; 19; 17	—
PRIMARY Change in Liver Iron Concentration (LIC)	6.25; 5.73; 5.46; 4.66; -0.9; -1.10	—
SECONDARY Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)	0.4292; 0.4083; 0.4939; 0.4286	—
SECONDARY Relative Change in Serum Ferritin Level	62.4; 54.9	—

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00390858). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.