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 further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects ?18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocol PB-06-001 and were enrolled in extension study PB-06-003. |
Ampliar la evaluación de la seguridad y la eficacia de la taliglucerasa alfa en sujetos adultos (? 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?001 y que se hayan enrolado en el estudio de extensión PB?06?003. |
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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 |
Eligible subjects must fulfill the following inclusion criteria: 1. Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003 2. The subject signs an informed consent |
Los sujetos elegibles deberán cumplir con los siguientes criterios de inclusión: 1. Haber completado con éxito el Protocolo PB?06?001 y haberse enrolado en el Protocolo PB?06?003. 2. El sujeto deberá firmar un formulario de consentimiento. |
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E.4 | Principal exclusion criteria |
Eligible subjects may not have any of the following exclusion criteria: 1. Currently taking another investigational drug for any condition. 2. Presence of any 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. |
Los sujetos elegibles no pueden poseer ninguno de los criterios de exclusión siguientes: 1. Estar tomando actualmente algún otro medicamento de investigación para cualquier enfermedad. 2. Presencia de cualquier enfermedad 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 or pulmonary and activation-regulated chemokine (PARC/CCL18) - DEXA
2- The safety of taliglucerase alfa will be assessed by the following: - Clinical laboratory - Echocardiography - Adverse events - Anti-taliglucerase alfa antibodies |
Se evaluarán las siguientes variables de eficacia durante la extensión del tratamiento: - Volumen del bazo - Volumen del hígado - Conteo de plaquetas - Hemoglobina - Bio indicadores (cito triosidasa o quimioquina pulmonar y de activación regulada (PARC/CCL18)) - DEXA
2-La seguridad de la taliglucerasa alfa se evaluará por medio del laboratorio clínico, la eco cardiografía, los anticuerpos anti ? taliglucerasa alfa y 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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 |
Canada |
Chile |
Israel |
Italy |
Mexico |
Serbia |
South Africa |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |