E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subjects with active Behçet's Disease or Juvenile Psoriatic Arthritis |
Sujetos con enfermedad de Behçet o artritis psoriásica juvenil activa |
|
E.1.1.1 | Medical condition in easily understood language |
Behçet Disease: inflammation in blood vessels throughout the body, manifests as mouth or genital ulcers with eye & skin lesions JPA:Joint inflammation with psoriatic disorder affecting pediatric pts |
EB:inflamación en los vasos sanguineos, se manifiestan ulceras bucales/genitales y lesiones en los ojos/piel AIJ:inflamación de las articulaciones con desorden psoriasico que afecta a pts pediatricos |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10004212 |
E.1.2 | Term | Behcet's disease |
E.1.2 | System Organ Class | 100000004866 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10079454 |
E.1.2 | Term | Systemic juvenile idiopathic arthritis |
E.1.2 | System Organ Class | 100000004859 |
|
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 |
Evaluate the long-term safety of apremilast in subjects 2 years of age or older with oral ulcers associated with Behçets disease or 5 years of age or older with active JPsA that have completed Study 20190530 or Study 20190529 |
Evaluar la seguridad a largo plazo de apremilast en sujetos de 2 años o más con úlceras bucales asociadas a la enfermedad de Behçet o de 5 años o más con APsJ activa que hayan completado el estudio 20190530 o el estudio 20190529. |
|
E.2.2 | Secondary objectives of the trial |
Not applicable |
No aplicable |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subject’s legally authorized representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated. 2. Subject must have completed week 52 (Apremilast Active Treatment Phase) of Study 20190529 (from France and Turkey sites only) or Study 20190530 where drug is not commercially available in their country. 3. Subject must have an age and sex specific body mass index (BMI) value no lower in range than the 5th percentile on the Centers for Disease Control (CDC) growth chart (Appendix 11.7) for children and adolescents (CDC, 2000) at randomization. 4. Subject is willing and able to adhere to the study visit schedule and other protocol requirements. 5. Subject must have acceptable benefit/risk for continued treatment with apremilast. |
1. El representante legal autorizado del sujeto ha dado su consentimiento informado cuando el sujeto es legalmente demasiado joven para dar su consentimiento informado y el sujeto ha dado su asentimiento por escrito de acuerdo con las normativas y/o directrices locales antes de iniciar cualquier actividad/procedimiento específico del estudio. 2. El sujeto debe haber completado la semana 52 (fase de tratamiento activo con apremilast) del estudio 20190529 (solo se aplica a los centros de Francia y Turquía) o del estudio 20190530 cuando el fármaco no esté disponible en el mercado de su país. 3. El sujeto debe tener una edad y un índice de masa corporal (IMC) correspondiente a su sexo que no sea inferior al percentil 5 de la tabla de crecimiento de los Centros para el Control de Enfermedades (CDC) (apéndice 11.7) para niños y adolescentes (CDC, 2000) en el momento de la aleatorización. 4. El sujeto está dispuesto a cumplir el calendario de visitas del estudio y otros requisitos del protocolo y es capaz de hacerlo. 5. El sujeto debe presentar una relación beneficio-riesgo aceptable para continuar el tratamiento con apremilast. |
|
E.4 | Principal exclusion criteria |
1. Answer “Yes” to any question on the Columbia-Suicide Severity Rating Scale (C-SSRS) at the week 52 visit on Study 20190529 (subjects from France and Turkey sites only) or Study 20190530. 2. Scheduled surgery or other interventions that would interrupt the subject’s participation in the study. 3. Female subjects of childbearing potential (for the purpose of this study, a female subject is considered of childbearing potential if she is 12 years old or older or has reached menarche, whichever occurred first) unwilling to use protocol specified method of contraception see Appendix 5 (Section 11.5) during treatment and for an additional 30 days after the last dose of investigational product. 4. Female subjects planning to become pregnant while on study through 30 days after the last dose of investigational product. 5. Female subjects of childbearing potential with a positive pregnancy test assessed at week 0 by a highly sensitive urine or serum pregnancy test. 6. Subject has known sensitivity to any of the products to be administered during dosing. 7. Subject likely to not be available to complete all protocol-required study visits. |
1. Respuesta afirmativa a cualquier pregunta de la escala Columbia para evaluar el riesgo de suicidio (C-SSRS) en la visita de la semana 52 del estudio 20190529 (solo se aplica a los sujetos de los centros de Francia y Turquía) o del estudio 20190530. 2. Cirugía programada u otras intervenciones que requieran que el sujeto interrumpa su participación en el estudio. 3. Mujeres en edad fértil (a efectos de este estudio, una mujer se considera en edad fértil si tiene ≥ 12 años o ha tenido la menarquia, lo que ocurra primero) que no estén dispuestas a utilizar un método anticonceptivo especificado en el protocolo (consulte el apéndice 5; apartado 11.5) durante el tratamiento y durante los 30 días posteriores a la administración de la última dosis del producto en investigación. 4. Mujeres que planean quedarse embarazadas durante el estudio o en los 30 días posteriores a la última dosis del producto en investigación. 5. Mujeres en edad fértil con una prueba de embarazo positiva, evaluada la semana 0 mediante una prueba de embarazo en suero u orina con un alto grado de sensibilidad. 6. El sujeto presenta una sensibilidad conocida a alguno de los productos que se administrarán durante el tratamiento. 7. Es probable que el sujeto no esté disponible para completar todas las visitas del estudio requeridas por el protocolo. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- Adverse events: Type, frequency, severity, and relationship to apremilast - Columbia-Suicide Severity rating Scale (C-SSRS) - Tanner Staging - Body weight, height, and body mass index (BMI) - Vital signs and laboratory parameters |
- Acontecimientos adversos: tipo, frecuencia, gravedad y relación con apremilast. - Escala Columbia para evaluar el riesgo de suicidio (C-SSRS). - Estadio de Tanner. - Peso corporal, altura e índice de masa corporal (IMC). - Signos vitales y parámetros analíticos. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
week 0, 26 , 52, 78, 104, 130 , 156 , 182 , 208 and 212 |
semana 0, 26 , 52, 78, 104, 130 , 156 , 182 , 208 y 212 |
|
E.5.2 | Secondary end point(s) |
Not applicable |
No aplicable |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Not applicable |
No aplicable |
|
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 | No |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 27 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 |
Israel |
France |
Spain |
Switzerland |
Greece |
Italy |
Turkey |
United Kingdom |
|
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
|
LVLS |
Ultima visita del ultimo paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 10 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial months | 11 |