E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Gaucher disease |
Enfermedad de Gaucher |
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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] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018048 |
E.1.2 | Term | Gaucher's disease |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic 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 extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa). |
Ampliar la evaluación de la seguridad y la eficacia de la taliglucerasa alfa en sujetos pediátricos (de 2 a 18 años de edad) que posean síntomas y manifestaciones clínicas de la enfermedad de Gaucher y que hayan completado el tratamiento del Protocolo PB-06-005 (estudio de cambio a partir de la imiglucerasa) o del Protocolo PB-06-005 (tratamiento naif con taliglucerasa alfa). |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Successful completion of Protocol PB-06-002 or PB-06-005. 2. The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent |
1.Haber completado con éxito el Protocolo PB-06-002 o el Protocolo PB-06-005. 2.Que el sujeto, sus padres o sus tutores legales hayan firmado un formulario de consentimiento y/o de asentimiento. |
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E.4 | Principal exclusion criteria |
1. Subjects currently taking another investigational drug for any condition. 2. The presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy. 3. The presence of a medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with the subject?s compliance with the requirements of the study. |
1.Estar actualmente tomando algún otro medicamento de investigación bajo cualquier circunstancia. 2.Presencia de signos neurológicos y síntomas característicos de la enfermedad de Gaucher con características neuronopáticas complejas que no sean las de la parálisis óculo motora crónica. 3.Presencia de cualquier condición médica, emocional, de comportamiento o psicológica que a juicio del Investigador pudiera interferir con el cumplimiento de los requisitos del estudio por parte del sujeto. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1- The following efficacy variables will be assessed: - Spleen volume - Liver volume - Platelet count - Hemoglobin - Biomarkers (chitotriosidase and PARC/CCL18)
- Exploratory endpoints are: o Change in height and weight o Change in Tanner Stage o Change in bone age by X-ray of left hand and wrist o Change in bone density by DEXA o Occurrence of bone crises o Qualify of Life using the Child Health Questionnaire? (CHQ) PF-28
2- The safety of taliglucerase alfa will be assessed by the following: - Clinical laboratory - Echocardiography - Antitaliglucerase alfa antibodies - Adverse events. |
1- Las siguientes variables de eficacia se evaluarán: - Volumen del bazo - Volumen del hígado - Conteo de plaquetas - Hemoglobina - Bio indicadores (cito triosidasa y PARC/CCL18)
- Los puntos finales exploratorios son: o Cambios en la altura y en el peso o Cambios en la Escala de Tanner o Cambios en la edad ósea mediante una radiografía de la mano y la muñeca izquierda o Cambios en la densidad ósea por DEXA o Ocurrencia de crisis óseas o Calificación de Vida por medio del uso del Cuestionario de Salud Infantil? (CHQ) PF-28
2- La seguridad de la taliglucerasa alfa será evaluada por medio del : -Laboratorio clínico - Eco cardiografía - Los anticuerpos anti - taliglucerasa alfa - Los efectos colaterales adversos. |
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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) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | No |
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 |
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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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Last Patient Last Visit or until the product is commercially available to the subjects |
Pasada la última visita del paciente o hasta que el producto se encuentre disponible comercialmente. |
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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 |