| 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) |
|
| E.1.1.1 | Medical condition in easily understood language |
| Activated PI3K delta Syndrome (APDS) |
| Síndrome de Fosfoinositida 3-Cinasa (PI3K) Delta Activada (APDS) |
|
| 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 of the study is to evaluate the long-term safety and tolerability of UCB5857 in subjects with APDS. |
| El objetivo principal del estudio es evaluar la seguridad y la tolerabilidad a largo plazo del UCB5857 en sujetos con APDS |
|
| E.2.2 | Secondary objectives of the trial |
| The secondary objective is to assess the long-term PK profile of UCB5857 in subjects with APDS through sparse sampling. |
| El objetivo secundario es evaluar el perfil farmacocinético a largo plazo del UCB5857 en sujetos con APDS mediante muestreo reducido |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
2. Subject/legal representative is considered reliable and capable of adhering to the protocol (eg, able to understand and complete diaries), visit schedule, and medication intake according to the judgment of the investigator; and be able to give written informed consent to participate in the study.
5. Subject must complete the full Treatment Period of APD001 and must be able to expect a reasonable benefit from the long-term administration of UCB5857 in the medical judgment of the Investigator following review of the subject?s overall response in APD001. The SMC from APD001 will confirm each subject?s eligibility based on an evaluation of individual benefit-risk balance.
8. Contraception methods for male subjects and their female partners:
- Male subjects with a partner of childbearing potential must be willing to use a condom when sexually active during the study and for 3 months after the last administration of study drug (anticipated 5 half lives).
- In addition the female partner of childbearing potential of a male subject must be willing to use a highly effective method of contraception (as above) during the study period and for 3 months after the last administration of study drug.
Additional inclusion criteria are outlined in section 6.1 of the clinical trial protocol. |
2. 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 y la administración de la medicación, a juicio del investigador; y es capaz de otorgar su consentimiento informado por escrito para participar en el estudio.
5. El sujeto debe haber concluido todo el periodo de tratamiento del estudio APD001 y, en su caso, debe esperarse que vaya a obtener un beneficio razonable de la administración a largo plazo de UCB5857, según el juicio médico del CVS tras la revisión de la respuesta global del sujeto en el estudio APD001. El CVS del estudio APD001 confirmará la elegibilidad de cada sujeto según una evaluación individual de la relación beneficio-riesgo.
Métodos anticonceptivos para los participantes varones y sus parejas femeninas:
-Los sujetos varones con parejas potencialmente fértiles deben estar dispuestos a utilizar preservativos en las relaciones sexuales que mantengan durante el estudio y los 3 meses siguientes a la última administración del fármaco del estudio (periodo previsto de 5 semividas del fármaco).
-Además, las mujeres potencialmente fértiles que sean pareja de un sujeto varón deben estar dispuestas a utilizar un método anticonceptivo de gran eficacia (como se ha señalado anteriormente) durante el estudio y los 3 meses siguientes a la última administración del fármaco del estudio.
Criterios de inclusión adicionales están descritos en la sección 6.1 del protocolo. |
|
| E.4 | Principal exclusion criteria |
1. Subject has participated in a clinical study with an IMP within 3 months of randomization into the current study (excluding participation in APD001) or subject is currently participating in another study of an IMP (or a medical device).
2. Subject has a history of chronic alcohol or drug abuse within the previous 6 months.
3. Subject has a history of allogenic bone marrow transplant.
4. Subject has any medical or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject?s ability to participate in this study.
5. Subject has any new significant uncontrolled condition or an ongoing SAE from the previous study (APD001) that was assessed as related to IMP.
6. Subject has a known hypersensitivity to any components of the IMP or comparative drugs as stated in this protocol.
7. 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.
11. Subject has >=3x upper limit of normal (ULN) alanine aminotransferase (ALT) or alkaline phosphatase (ALP), or >ULN bilirubin (>=1.5xULN bilirubin if known Gilbert?s syndrome). If subject only has >1.5xULN bilirubin, fractionate bilirubin to identify possible undiagnosed Gilbert?s syndrome (ie, direct bilirubin <35%). For subjects with a baseline result >ULN, a baseline diagnosis and/or the cause of any clinically meaningful elevation must be understood and recorded in eCRF . If subject has >ULN that does not meet the exclusion limit for, ALT, or ALP at screening, repeat, if possible, prior to dosing to ensure there is no further ongoing clinically relevant increase. In case of a clinically relevant increase, inclusion of the patient must be discussed with the Medical Monitor[IS18]. Serum ALT results up to 25% above the exclusion limit may be repeated once for confirmation. This includes re-screening.
Additional exclusion criteria are outlined in section 6.2 of the clinical trial protocol. |
1. El sujeto ha participado en otro estudio clínico con un medicamento en investigación (MI) en el plazo de los 3 meses anteriores a su entrada en el presente estudio (excluida la participación en el estudio APD001) o se encuentra participando en la actualidad en otro estudio de un MI (o producto sanitario).
2. El sujeto tiene antecedentes de abuso crónico de alcohol o drogas en los 6 meses previos.
3. El sujeto tiene antecedentes de alotrasplante de médula ósea.
4. El sujeto padece alguna afección médica o psiquiátrica que, a juicio del investigador, podría poner en peligro al sujeto o alterar su capacidad para participar en este estudio.
5. El sujeto presenta un trastorno no controlado importante nuevo o un AAG en curso del estudio anterior (APD001) que se consideró relacionado con el UCB5857.
6. El sujeto presenta hipersensibilidad conocida a cualquier componente del UCB5857 o de los fármacos de comparación según lo establecido en este protocolo.
7. Mujer en periodo de lactancia materna o embarazada, o que tiene previsto quedarse embarazada o iniciar la lactancia materna durante el estudio o en el plazo de los 3 meses siguientes a la última dosis de UCB5857.
11. El sujeto presenta un valor de alanina-aminotransferasa (ALT) o de fosfatasa alcalina >=5 × límite superior de la normalidad (LSN) o un valor de bilirrubina >LSN (bilirrubina >=2 × LSN en caso de síndrome de Gilbert conocido). Si el sujeto solo presenta un valor aislado de bilirrubina >LSN y <2 × LSN, se determinarán las fracciones para identificar un posible síndrome de Gilbert no diagnosticado (es decir, bilirrubina directa <35 %). Si un sujeto considerado plenamente elegible a la entrada del estudio APD001 presenta valores de las pruebas funcionales hepáticas fuera de estos criterios, podrá entrar en el presente estudio siempre que no se hayan producido variaciones importantes de dichas pruebas hepáticas desde la entrada en el estudio APD001.
Criterios de exclusión adicionales están descritos en la sección 6.2 del protocolo. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
- The total number of subjects experiencing at least one Treatment Emergent Adverse Event during the study
- The total number of subjects experiencing at least one Serious Adverse Event during the study |
- El número total de sujetos que experimenten al menos un Acontecimiento Adverso relacionado con el tratamiento durante el estudio.
-El número total de sujetos que experimenten al menos un Acontecimiento Adverso Grave durante el estudio. |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| Time Frame: Baseline until the end of study |
| Periodo de tiempo: Desde el inicio hasta el final del estudio |
|
| E.5.2 | Secondary end point(s) |
| Plasma Concentration of UCB5857 |
| Concentración en plasma de UCB5857 |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
| Time Frame: Baseline until the end of study |
| Periodo de tiempo: Desde el inicio hasta el final del estudio |
|
| 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 | Yes |
| E.6.13.1 | Other scope of the trial description |
| Tolerability of UCB5857 |
| Tolerabilidad de UCB5857 |
|
| 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 | 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 | 5 |
| 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 of the last subject in the study. |
| El fin del estudio se define como la fecha de la última visita del último sujeto en el estudio. |
|
| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 5 |
| 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 | 5 |
| 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 |
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