E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Activated PI3K delta Syndrome (APDS) |
Síndrome de Fosfoinositida 3-Cinasa (PI3K) Delta Activada (APDS) |
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E.1.1.1 | Medical condition in easily understood language |
Activated PI3K delta Syndrome (APDS) |
Síndrome de Fosfoinositida 3-Cinasa (PI3K) Delta Activada (APDS) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
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 |
The primary objective is to evaluate the safety and tolerability of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS). |
El objetivo principal de este estudio es evaluar la seguridad y la tolerabilidad de UCB5857 en sujetos con Síndrome de fosfoinositida 3-cinasa (PI3K) delta Activada (APDS). |
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E.2.2 | Secondary objectives of the trial |
The secondary objective is to assess the pharmacokinetic (PK) profile of UCB5857 in subjects with Activated phosphoinositide-3 kinase (PI3K) delta Syndrome (APDS). |
El objetivo secundario es determinar el perfil de farmacocinética (FC) de UCB5857 en sujetos con Síndrome de fosfoinositida 3-cinasa (PI3K) delta Activada (APDS). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject/legal representative is considered reliable and capable of adhering to the protocol (e.g. able to understand and complete diaries), visit schedule, or medication intake.
- Subject must have a confirmed genotypic diagnosis of APDS (i.e. proven pathogenic mutation in either the PIK3R1 or PIK3CD gene).
Additional inclusion criteria are outlined in section 6.1 of the clinical trial protocol. |
- Se considera que el sujeto/representante legal es fiable y capaz de cumplir el protocolo (por ejemplo, es capaz de comprender y rellenar los diarios), el calendario de visitas o la administración de la medicación.
- El sujeto debe tener un diagnóstico genotípico confirmado de APDS (es decir, una mutación con valor patógeno demostrada en el gen PIK3R1 o en el PIK3CD).
Criterios de inclusión adicionales se resaltan el la sección 6.1 del protocolo de ensayo clínico. |
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E.4 | Principal exclusion criteria |
- Subject has participated in another study of an investigational medicinal product (IMP) (or a medical device) within the previous 30 days or is currently participating in another study of an IMP (or a medical device). - Subject has a history of allogeneic bone marrow transplantation. - Subject has no history of any clinical manifestations in last 12 months. - Subject tests positive for human immunodeficiency virus Type 1 or Type 2, hepatitis B surface antigen, or hepatitis C virus antibody. - Subject is female and is breast-feeding, pregnant, or plans to become pregnant or to start breastfeeding during the study or within 3 months following last dose of IMP. - Subject has a history of chronic alcohol or drug abuse within the previous 6 months. - Subject has any relevant medical or psychiatric condition that could jeopardize or would compromise the subject?s ability to participate in this study. - Subject has a known hypersensitivity to any components of the IMP or comparative drugs as stated in this protocol.
Additional exclusion criteria are outlined in section 6.2 of the clinical trial protocol. |
- El sujeto ha participado en otro estudio de un medicamento (o un producto sanitario) en investigación (MI) durante los 30 días previos o está participando actualmente en otro estudio de un medicamento (o un producto sanitario) en investigación. - El sujeto tiene antecedentes de un alotrasplante de médula ósea previo. - El sujeto no ha tenido ninguna manifestación clínica en los últimos 12 meses. - El sujeto presenta un resultado positivo en los análisis del virus de la inmunodeficiencia humana de tipo 1 o tipo 2, el antígeno de superficie de la hepatitis B o anticuerpos contra el virus de la hepatitis C. E- l sujeto es una mujer y está en periodo de lactancia materna, embarazada o tiene previsto quedarse embarazada o iniciar la lactancia materna durante la realización del estudio o en un periodo de 3 meses tras la última dosis del MI. - El sujeto tiene antecedentes de abuso crónico de alcohol o drogas en los 6 meses previos. - El sujeto presenta cualquier trastorno médico o psiquiátrico relevante que pudiera poner en peligro o comprometer la capacidad del sujeto para participar en este estudio. - El sujeto tiene hipersensibilidad conocida a cualquiera de los componentes del MI o fármacos de comparación según lo establecido en este protocolo.
Criterios de exclusión adicionales se resaltan en la sección 6.2 del protocolo de ensayo clínico. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety variables The safety variables will include: - Incidence of AEs - Incidence of SAEs - Changes from Baseline in safety laboratory tests at the time points measured - Change from Baseline in vital sign parameters and body weight at the time points measured - Change from Baseline in ECG parameters at the time points measured - Physical examination findings.
Pharmacokinetic variable The PK variable will be the plasma concentration of UCB5857. |
Variables de Seguridad Las variables de seguridad incluirán: - Incidencia de EAs -Incidencia de EAGs -Cambios respecto del valor basal en los tests de seguridad de laboratorio en los tiempos de medida. -Cambios respecto del valor basal en los parámetros de signos vitales y peso corporal en los tiempos de medida. - Cambios respecto del valor basal en los parámetros de ECG en los tiempos de medida. -Resultados de la examinación física.
Variable de Farmacocinética La variable de farmacocinética será la concenración de plasma de UCB5857. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At the time points measured. |
A los tiempos de medida tomados. |
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E.5.2 | Secondary end point(s) |
Please refer to the study variables of the Clinical Trial Protocol. |
Por favor, consulten las variables del estudio del Protocolo de Ensayo Clínico. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Please refer to the study variables of the Clinical Trial Protocol. |
Por favor, consulten las variables del estudio del Protocolo de Ensayo Clínico. |
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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 | No |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
A phase 1b study to evaluate the safety and tolerability of UCB5857 in subjects with APDS |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | No |
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 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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
|
The end of the study is defined as the date of the last visit (or follow-up telephone call) of the last subject in the study. |
El fin del estudio se define como la fecha de la última visita (o llamada telefónica de seguimiento ) del último sujeto en el estudio. |
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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 |