Health Condition 1: E752- Other sphingolipidosis Health Condition 2: null- Type 3 Gaucher Disease
Conditions
Interventions
Intervention1: Velaglucerase Alfa (VPRIV®): Each infusion of Velaglucerase Alfa will be administered at the clinical site over an interval of 60 minutes for 51 weeks
Control Intervention1: Not applic
Sponsors
Shire Human Genetic Therapies Inc
Eligibility
Inclusion criteria
Inclusion criteria: Each patient must meet the following criteria to be enrolled in this study. 1. The patient has a confirmed diagnosis of type 3 Gaucher disease. 2. The patient is >=2 and 3. The patient is either naive to treatment or has not received treatment (investigational or approved) for gaucher disease within 12 months prior to study entry. 4. The patient has Gaucher disease-related anemia, defined as hemoglobin concentration below the lower limit of normal for age and sex. AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA a. The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation. b. The patient has Gaucher disease-related thrombocytopenia, defined as platelet count less than120 multiply to 1000 platelets/mm3. c. The patient has a Gaucher disease-related readily palpable enlarged liver. 5. Patients who have undergone splenectomy may still be eligible to participate in the study. 6. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy testing will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partnerâ??s pregnancy to the Investigator. 7. The patientâ??s parent(s) or the patientâ??s legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
Exclusion criteria
Exclusion criteria: Patients who meet any of the following criteria will be excluded from this study. 1. The patient is suspected of having type 2 or type 1 Gaucher disease. 2. The patient is less than 2 years of age. 3. The patient has experienced a severe (Grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any enzyme replacement therapy for Gaucher disease (approved or investigational). 4. The patient has received any non-Gaucher disease-related treatment with an investigational drug within 30 days prior to study entry. 5. The patient is a pregnant and/or lactating female.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Baseline to 12 months in hemoglobin concentration.Timepoint: Baseline to 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary efficacy endpoints include evaluations of change in platelet count, liver and spleen volume, and neurological symptoms. Mean within-patient changes in secondary efficacy assessments from Baseline to 12 months will be evaluated.Timepoint: Baseline to 12 months | — |
Countries
India, Tunisia
Contacts
Public ContactSuchela Srivatsa
IQVIA RDS (India) Private Limited
Outcome results
None listed