E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
- HSCT (Hematopoietic stem cell transplantation)
-beta-thalassemia major |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•Safety of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major [ Time Frame: 12 months ]
• 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 |
|
E.2.2 | Secondary objectives of the trial |
• Efficacy of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major by 12th month. [ Time Frame: 12 Months ]
• 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. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
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
|
|
E.4 | Principal exclusion criteria |
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
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters : 12 months |
|
E.5.2 | Secondary end point(s) |
- 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. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- 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. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
21-10-2015 (last patient last visit) |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 2 |