Phase 2
N=184
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
Anemia · Hemosiderosis
Bottom Line
View on ClinicalTrials.gov: NCT00303329 ↗Enrolled (actual)
184
Serious AEs
46.7%
Results posted
May 2011
Primary outcome: Primary: The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths — 85; 99; 36; 50 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferasirox (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Oct 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths |
85; 99; 36; 50; 2; 12 | — |
| SECONDARY The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study |
-5.17; -5.10 | — |
| SECONDARY The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study |
0.19; -2.04 | — |
| SECONDARY The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study |
4320.6; 3268.8; 3708.2; 2896.0; -612.4; -382.2 | — |
Summary
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.
The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.
Eligibility Criteria
Inclusion Criteria
- Patients completed the planned 12-month core study
- Female patients who have reached menarche and who are sexually active must 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 legal guardian on the patient's behalf in accordance with the national legislation
Exclusion Criteria
- Pregnant or breast feeding patients
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT00303329). 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.