E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Moderate and above aplastic anemia |
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E.1.1.1 | Medical condition in easily understood language |
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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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To investigate the efficacy of PF-06462700 administered intravenously at 40mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia |
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E.2.2 | Secondary objectives of the trial |
To investigate the efficacy of PF-06462700 administered intravenously at 40mg/kg/day for 4 days in Japanese participants with moderate and above aplastic anemia |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
-Male or female participants between the ages of 2 years and more inclusive at visit 1 (screening) -Have a clinical diagnosis of aplastic anemia by bone marrow aspiration/biopsy findings and/or magnetic resonance imaging (MRI) etc. -Must meet the criteria of moderate and above aplastic anemia (stage 2b and above) |
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E.4 | Principal exclusion criteria |
-Eligible and willing to have a sibling allogeneic stem cell transplantation -Evidence of a myelodysplastic syndrome (except for refractory cytopenia in children), as well as other primitive marrow disease -History of clinical suspicion of congenital aplastic anemia (Fanconi anemia, Congenital keratosis, etc). -Previous history of stem cell transplantation - Prior immunosuppressive therapy with lymphocyte-depleting agents/therapies, including both non-B-cell selective and B-cell-depleting agents. However, participants previously treated with rATG may enroll. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Hematologic response at week 12 |
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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) |
- Hematologic response at week 24 -Hematological test values at day 4 and at weeks 1, 2, 4, 6, 8, 10, 12 and 24 (Absolute neutrophil count, Platelet count, Reticulocyte count) -Survival Status -Transfusion independence at weeks 12 and 14 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- week 24 - weeks 1, 2, 4, 6, 8, 10, 12 and 24 - up to week 24 -week 12 - week 24 |
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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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 |
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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
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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 | 1 |