Gaucher disease MedDRA version: 9.1 Level: PT Classification code 10018048 Term: Gaucher's disease
Conditions
Interventions
Product Name: recombinant human glucocerebrosidase
Product Code: prGCD
Pharmaceutical Form: Powder for solution for infusion
Current Sponsor code: prGCD
Sponsors
Protalix Biotherapeutics
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - 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 which 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 =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The change in spleen volume (in procent) from baseline at 9 months measured by MRI.; Secondary Objective: Change from baseline in: - Liver volume - Platelet count - Hemoglobin other secondary analysis parameter: -Biomarkers (chitotriosidase and pulmonary and activation-regulated chemokine (PARC/CCL18) - Proportion of patients with greater than 10% reduction in spleen volume at 9 months ; Primary end point(s): Primary efficacy endpoint: Percent change from baseline in spleen volume measured by MRI at 9 months | — |
Countries
Italy, Spain, United Kingdom
Outcome results
None listed