Phase 4
N=233
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
Beta-thalassemia Major · Hemosiderosis · Iron Overload · Rare Anemia
Bottom Line
View on ClinicalTrials.gov: NCT00171301 ↗Enrolled (actual)
233
Serious AEs
7.4%
Results posted
Jul 2011
Primary outcome: Primary: Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age — 75.0; 100; 72.2; 50.0 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Deferasirox (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- May 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age |
75.0; 100; 72.2; 50.0; 76.6; 73.8 | — |
| PRIMARY Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category |
-1.32; NA; -9.03; -8.39 | — |
| SECONDARY Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study |
-1432.51; -1791.91 | — |
| SECONDARY Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC) |
-369.83; -1619.31 | — |
Summary
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available. To evaluate the long-term safety and efficacy of deferasirox by measuring treatment success, change in liver iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by incidence of adverse events (AEs)and clinically significant lab parameters.
Eligibility Criteria
Inclusion Criteria
- Patients completing the planned 12-month core study (NCT00171171)
- Female patients who have reached menarche and who are sexually active must use an effective method of contraception, 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.
- Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the core study protocol, or the planned extension protocol
Data sourced from ClinicalTrials.gov (NCT00171301). 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.