Phase 3 N=555 Treatment

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

Transfusional Iron Overload in β-thalassemia

Bottom Line

View on ClinicalTrials.gov: NCT00171210 ↗

Enrolled (actual)

555

Serious AEs

29.4%

Results posted

May 2011

Primary outcome: Primary: Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event — 109; 113; 85; 110 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Deferasirox (Drug)
Age: Pediatric, Adult, Older Adult · 2+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Apr 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event	109; 113; 85; 110; 81; 84	—
SECONDARY Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy	-2.4; -4.1	—
SECONDARY Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID	-0.5; -0.7	—
SECONDARY Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study	-122.1; -527.8	—
SECONDARY Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study	7.7; 7.0	—
SECONDARY Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study	1.5; 10.3	—
SECONDARY Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study	-5.4; 3.4	—
SECONDARY Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy	-2.8; -3.2	—
SECONDARY Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy	-25.4; -26.0	—
SECONDARY Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID	-1.1; -1.6	—
SECONDARY Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID	-20.8; -33.0	—
SECONDARY Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study	0.37; 0.38	—

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00171210). 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.