Ensayo clinico de fase III, multicéntrico, aleatorizado, doble ciego; para evaluar la seguridad y eficacia de dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher.A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease. - NA
- Conditions
- Enfermedad de Gaucher.Gaucher disease.MedDRA version: 9.1Level: PTClassification code 10018048Term: Gaucher's disease
- Registration Number
- EUCTR2007-000498-42-ES
- Lead Sponsor
- Protalix Biotherapeutics
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 30
-Males and females, 18 years or older.
-Female patients of child-bearing potential or male patients with female partners of child-bearing potential must agree to use two methods of contraception, one of wich must be a barrier method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms.
-Diagnosis of Gaucher disease with leukocyte GCD activity level =3 nmol/mg*hr (=30 % of the mean activity of the reference range)
-Splenomegaly defined as greater than eight times the expected volume [measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis
-Thrombocytopenia (defined as platelet counts <120,000 per mm3) with or without anemia (defined by hemoglobin at least 1 g/dL below normal range according to sex and age).
?Patients who have not received ERT in the past or patients who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody
?Patients who have not received substrate reduction therapy (SRT) in the past 12 months.
?Ability to provide a 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
-Currently taking another experimental drug for any condition
-Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
-Previous anaphylactoid reaction to Cerezyme® or Ceredase®
-History of allergy to carrots
-Pregnant or nursing
-Presence of HIV and/or, HBsAg and/or hepatitis C infections
-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
- Secondary Outcome Measures
Name Time Method