E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acquired Thrombotic thrombocytopenic purpura |
Púrpura trombocitopénica trombótica adquirida |
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E.1.1.1 | Medical condition in easily understood language |
TTP is a rare condition in which the blood becomes ?sticky? and forms clots within the blood vessels in different parts of the body. |
La PTT es una enfermedad rara que se caracteriza por la viscosidad excesiva de la sangre y por la formación de coágulos en los vasos sanguíneos de distintas partes del cuerpo. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10043648 |
E.1.2 | Term | Thrombotic thrombocytopenic purpura |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic system disorders |
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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 evaluate efficacy of caplacizumab in more rapidly restoring normal platelet counts as measure of prevention of further microvascular thrombosis. |
Evaluar la eficacia de caplacizumab en la restauración más rápida del número normal de plaquetas como medida de la prevención de trombosis microvascular adicional |
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E.2.2 | Secondary objectives of the trial |
-To evaluate prevention of recurrence of presenting TTP episode after cessation of daily PE, including prolongation of caplacizumab treatment beyond 30 days post-daily PE treatment based on clinical assessment of underlying disease and ADAMTS13 activity -To evaluate mortality rate -To evaluate the effect of caplacizumab on biomarkers for organ damage: LDH, cTnI, and serum creatinine. |
-Evaluar la prevención de recidiva del episodio de PTT de presentación tras el cese del intercambio de plasma (IP) diario, incluyendo la prolongación del tratamiento con caplacizumab después del periodo de 30 días posterior al tratamiento de IP diario, basándose en la evaluación clínica de la enfermedad subyacente y en la actividad de ADAMTS13 -evaluar la tasa de mortalidad -evaluar el efecto de caplacizumab sobre los biomarcadores de daño orgánico: lactato deshidrogenasa (LDH), troponina I cardiaca (TnIc) y creatinina sérica. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Adult male or female ? 18 years of age at the time of signing the informed consent form (ICF) 2.Clinical diagnosis of acquired TTP (initial or recurrent), which includes thrombocytopenia and microscopic evidence of red blood cell fragmentation (e.g. schistocytes) 3.Requires initiation of daily PE treatment and has received PE treatment prior to randomization . |
1-Adulto varón o mujer ? 18 años de edad en el momento de la firma del formulario de consentimiento informado (FCI) 2-Diagnóstico clínico de PTT adquirida (inicial o recurrente), que incluye trombocitopenia y evidencia microscópica de fragmentación eritrocitaria (p. ej., esquistocitos) 3-Requiere instauración del tratamiento de IP diario y ha recibido tratamiento de IP antes de la aleatorización. |
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E.4 | Principal exclusion criteria |
1.Platelet count ?100×109/L 2.Serum creatinine level >200 µmol/L in case platelet count is > 30×109/L 3.Known other causes of thrombocytopenia 4.Congenital TTP (known at the time of study entry) 5.Pregnancy or breast-feeding. |
1.Número de plaquetas ?100×109/l 2.Nivel de creatinina sérica >200 µmol/l en caso de que el número de plaquetas sea > 30×109/l 3.Otras causas conocidas de trombocitopenia 4.PTT congénita (conocida en el momento de la entrada en el estudio) 5.Embarazo o lactancia. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Time to platelet count response defined as initial platelet count ? 150×109/L with subsequent stop of daily PE within 5 days. |
Tiempo hasta la respuesta en el número de plaquetas definida como un número inicial de plaquetas ? 150×109/l con la consiguiente interrupción del IP diario en los siguientes 5 días. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
daily PE period |
Periodo de IP diario |
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E.5.2 | Secondary end point(s) |
1-Proportion of subjects with an exacerbation and/or relapse of TTP as well as the number of such events 2-Proportion of subjects with treatment-emergent clinically significant TTP-related events as well as the number of such events. 3-Area under the curve (AUC) of platelet count 4-Time to lactate dehydrogenase (LDH) ? 2 x upper limit of normal (ULN) 5-Time to cardiac Troponin I (cTnI) ? 1 x ULN 6-Time to serum creatinine ? 1 x ULN 7-Mortality Rate 8-(Serious)adverse events |
1.Proporción de pacientes con exacerbación y/o recidiva de la PTT así como el número de dichos acontecimientos. 2.Proporción de pacientes con acontecimientos relacionados con la PTT clínicamente significativos así como el número de dichos acontecimientos. 3.Área bajo la curva (AUC) del número de plaquetas. 4.Tiempo hasta una lactato deshidrogenasa (LDH) ? 2 x límite superior normal (LSN) 5.Tiempo hasta una troponina I cardiaca (TnIc) ? 1 x ULN. 6.Tiempo hasta una creatinina sérica ? 1 x ULN. 7.Tasa de mortalidad 8. Acontecimientos Adversos (Graves) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1-During study drug treatment and during follow-up (FU) 2, 4, 5 &6- During the overall study period. 3- until Day 5 7-Initial daily PE period, full study drug treatment period, FU period and overall study period 8-During the overall study period |
1.Durante el tratamiento con el fármaco del estudio y durante el seguimiento. 2., 4, 5 y 6. Durante el periodo global del ensayo. 3. hasta el día 5 7. Periodo de IP diario inicial, periodo completo de tratamiento con el fármaco del estudio, periodo de seguimiento y periodo global del estudio. 8. Durante el periodo global del ensayo. |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 47 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
Belgium |
Canada |
Czech Republic |
France |
Germany |
Hungary |
Israel |
Italy |
Netherlands |
New Zealand |
Spain |
Switzerland |
Turkey |
United Kingdom |
United States |
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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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LVLS |
Última visita del último paciente. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |