E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Sickle Cell Disease/ Iron Overload |
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E.1.1.1 | Medical condition in easily understood language |
Patients with sickle cell disease to too much iron from request blood transfusions |
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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 |
To assess the safety of ICL670 compared to deferoxamine during 24 weeks in patients with sickle cell disease and iron overload from repeated blood transfusions |
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E.2.2 | Secondary objectives of the trial |
Long term safety of ICL670 for up to 104 weeks in patients with sickle cell disease and iron overload from repeated blood transfusions |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Sub study conducted within main protocol consists of exploratory analysis of utility of MRI vs biopsy for the assessment of liver iron concentration values as well as assessing the correlation between liver iron concentration and ferritin. |
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E.3 | Principal inclusion criteria |
•Age greater than or equal to 2 years
•Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)
•Iron overload from repeated blood transfusion |
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E.4 | Principal exclusion criteria |
•Serum creatinine above the upper limit of normal
•Significant proteinuria•History of nephrotic syndrome
•Alanine aminotransferase (ALT) ≥ 250 U/L at screening
•Clinical evidence of active hepatitis B or hepatitis C |
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E.5 End points |
E.5.1 | Primary end point(s) |
-The Number of Participants with Adverse Events (AEs) in the First 24 Weeks of Treatment
-The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Absolute Change in Serum Ferritin From Baseline to Week 24 [ Time Frame: Baseline, 24 Weeks ]
-Absolute change from baseline serum ferritin after 24 weeks of treatment with Deferasirox (ICL670) and absolute change from baseline serum ferritin after 24 weeks of treatment with Deferoxamine. Means were adjusted for the amount of transfused blood
-Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52 [ Time Frame: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks ]
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 24 and the absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 52 for the Deferasirox treatment group and the Deferoxamine then Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
-Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104 [ Time Frame: Start of Deferasirox (ICL670) treatment, 104 Weeks ]
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 104 for the Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
-24 weeks
-24 weeks, 52 weeks
-104 weeks |
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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 | Yes |
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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
E.8.1.6 | Cross over | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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 |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
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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 | 11 |