Ensayo clinico de fase 3, multicéntrico, en abierto y con cambio de tratamiento para evaluar la seguridad y eficacia de Glucocerebrosidasa Humana Recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher tratados con Imiglucerasa (Cerezyme®) como terapia de reemplazo enzimatico. A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease Treated with Imiglucerase (Cerezyme®) Enzyme Replacement Therapy - NA

• Conditions: MedDRA version: 9.1Level: PTClassification code 10018048Term: Gaucher's disease; Enfermedad de GaucherGaucher Disease

• Registration Number: EUCTR2008-005825-12-ES
• Lead Sponsor: Protalix Biotherapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-03-06
• Last Update Posted: 18 April 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Males and females, 18 years or older
2. Female patients of child-bearing potential and male patients with female partners of child-bearing potential must agree to use a medically acceptable method of contraception, not including the rhythm method
3. Diagnosis of Gaucher disease with leukocyte GCD activity level ?3 nmol/mg*hr (?30 % of the mean activity of the reference range)
4.Stable Gaucher disease, defined as:
a. Hemoglobin during Stability Evaluation Period is stable with no value more than 15% below or above the mean value
b. Platelets count during Stability Evaluation Period is stable with no values more than 40% below or above the mean value if the mean value is >120,000, or more than 20% below or above the mean value if the mean value is ? 120,000
c. No major surgery in the last year
d. No blood transfusion or major bleeding episode in the last year
e. No acute avascular necrosis event in the last year
f. No evidence of spleen or liver increasing enlargement while being treated with enzyme replacement therapy by palpation, ultrasound, or MRI over the last year
5. Receiving imiglucerase therapy for at least 2 years and on a stable maintenance regimen (dose unchanged) for at least last six months
6. Able to provide written 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

Exclusion Criteria

1. Currently taking another experimental drug for any condition
2. Pregnant or nursing or planning to become pregnant
3. History of allergy to carrots
4. Presence of anti-glucocerebrosidase (GCD) antibodies
5. Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions
6. Presence of HIV and/or HBsAg and/or hepatitis C infection
7. Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency
8. Presence of any significant comorbidity that could confound the interpretation of the clinical response to prGCD
9. 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