Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major Clinical Trial
Official title:
A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
| Verified date | February 2016 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Turkey: Ministry of Health |
| Study type | Interventional |
This is a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study is to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.
| Status | Completed |
| Enrollment | 27 |
| Est. completion date | October 2015 |
| Est. primary completion date | October 2015 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 2 Years and older |
| Eligibility |
Inclusion Criteria - Patients who had HSCT for beta-thalassemia major - HSCT was performed minimum 6 months and maximum 2 years ago - The washout period after the immunosuppressive therapy should be at least 3 months. - Signifacant IOL should be present including: A. Serum ferritin >1000 µg/L or B. cardiac MRI <20 ms or C. liver iron concentration = 5 mg/g dry weight measured by R2* MRI Exclusion Criteria - Patients who have any contraindication for treatment with deferasirox according to the prescribing information •Patients who depend on transfusion - Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias) - Patients who are experiencing severe complication of HSCT (e.g. acute GVHD) - Significant proteinuria / Increase in serum creatinine |
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Turkey | Novartis Investigative Site | Ankara | |
| Turkey | Novartis Investigative Site | Ankara | |
| Turkey | Novartis Investigative Site | Antalya | |
| Turkey | Novartis Investigative Site | Antalya | |
| Turkey | Novartis Investigative Site | Istanbul | |
| Turkey | Novartis Investigative Site | Izmir |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
Turkey,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | •Safety of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major | • To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period | 12 months | Yes |
| Secondary | • Efficacy of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major by 12th month. | • To evaluate change the serum ferritin level and change from baseline to 12th month • To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month • To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52. | 12 Months | No |