Ensayo clínico de extensión, multicéntrico, doble ciego con dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease
- Conditions
- Enfermedad de GaucherGaucher DiseaseMedDRA version: 9.1Level: PTClassification code 10018048Term: Gaucher's disease
- Registration Number
- EUCTR2008-005826-35-ES
- Lead Sponsor
- Protalix Biotherapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
1. Completion of Protocol PB-06-001
2. The patient signs an informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1. Currently taking another experimental drug for any condition.
2. Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease.
3. Pregnant or nursing.
4. Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient?s compliance with the requirements of the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To extend the assessment of the safety and efficacy of prGCD in patients completing 9 months of treatment in Protocol PB-06-001;Secondary Objective: NA;Primary end point(s): Efficacy Endpoints:<br>-Spleen volume<br>-Liver volume<br>-Platelet counts<br>-Hemoglobin<br>-Biomarkers: chitotriosidase and PARC/CCL18<br>-QCSI (a long-term follow-up)<br>-Change in bone mineral density DEXA<br><br>Safety Measures:<br>-Adverse events<br>-Anti human prGCD antibodies<br>-Hypersensitity reactions
- Secondary Outcome Measures
Name Time Method