Type I Gaucher disease Enfermedad de Gaucher tipo 1 MedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's disease
Conditions
Interventions
Product Name: Gene Activated Human glucocerebrosidase
Product Code: GA-GCB
Pharmaceutical Form: Powder for infusion*
Current Sponsor code: GA-GCB
Other descriptive name: Gene activated human glucocere
Sponsors
Shire Human Genetic Therapies Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis. The patient is at least 2 years of age. The patient has Gaucher disease-related anemia, defined as hemoglobin concentration of at least 0.5 g/dL below the lower limit of normal for age and gender (based on the results obtained during Screening and at Baseline). AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA: The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation. (Patients who have undergone splenectomy must satisfy either inclusion criterion 3c or inclusion criterion 3d to be eligible for this study.) OR The patient has Gaucher disease-related thrombocytopenia (defined as a platelet count less than or equal to 120 x 10000/cu mm). OR The patient has a Gaucher disease-related readily palpable enlarged liver. The patient has not received treatment for Gaucher disease (investigational products, miglustat, or imiglucerase) within 12 months prior to study entry, as documented in the patient’s medical history. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study. The patient, the patient’s parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC). The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator. Are the trial subjects under 18? yes 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
Exclusion criteria: The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease. The patient is antibody-positive to imiglucerase or GA-GCB at Screening or the patient has experienced an anaphylactic reaction to imiglucerase or GA-GCB or the patient has required premedication use to manage infusion reactions to imiglucerase or GA-GCB. The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted. The patient is currently receiving red blood cell growth factor, chronic systemic corticosteroids, or has been on such treatment within the last 6 months. The patient is known to be positive for human immunodeficiency virus (HIV), i.e., has a documented positive result. Patients who do not have a documented positive result will be tested for HIV at screening. The patient is known to be positive for hepatitis B and/or C, i.e., has a documented positive result. Patients who do not have a documented positive result will be tested for hepatitis at screening. The patient presents with exacerbated anemia at Screening (e.g., due to folic acid and/or vitamin B12 deficiency). The patient presents with serum transferrin saturation <20 micrograms/dL and serum ferritin < 50 micrograms/dL The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study. The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.). The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the effects of GA-GCB and imiglucerase on hemoglobin concentration in patients with type 1 Gaucher disease.;Secondary Objective: To compare the effects of GA-GCB and imiglucerase on platelet count To compare the effects of GA-GCB and imiglucerase on liver and spleen volumes (by MRI) To compare the effects of GA-GCB and imiglucerase on Gaucher disease-specific biomarkers (plasma chitotriosidase and CCL18 levels) To evaluate the safety of GA-GCB and imiglucerase in patients with type 1 Gaucher disease, as measured by standard clinical laboratory assessments (including rates of antibody formation and enzyme neutralizing antibody activity) and safety evaluations (including rates of infusion-related adverse events and the proportion of patients requiring premedication use to manage infusion-related adverse events) for each treatment group To compare the effects of GA-GCB and imiglucerase on the earliest time to response for hemoglobin (defined as a greater than or equal to 1 g/dL improvement in hemoglobin levels relative to Baseline) ;Primary end point(s): The primary endpoint of this study is to compare the mean change from Baseline in hemoglobin concentration between the two treatment groups. | — |
Countries
Italy, Spain, United Kingdom
Outcome results
None listed