A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

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About the study

The main purpose of this study is to observe the side effects of VPRIV in participants with type 1 Gaucher disease who are either treatment-naïve (newly diagnosed) or who are currently being treated with enzyme replacement therapy (ERT). Participants will receive VPRIV intravenously during the treatment period (up to 51 weeks), followed by the end-of-treatment (EOT) visit after 2 weeks.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

EXCLUSION CRITERIA

  • Inclusion:
  • * Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator:


    1. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or
    2. Decreased GCB activity level that is >30% of normal, but with confirmation of genetic mutation test
    3. Is at least 2 years of age, inclusive, at screening
    4. Is naive to treatment for Gaucher disease (Has not received treatment for Gaucher disease [investigational or approved products] within the 12 months prior to screening) OR Is receiving or has recently received Imiglucerase ERT (Has received Imiglucerase treatment within the 12 months prior to screening and not within the 14 days prior to screening)
    5. Has Gaucher disease-related hematological abnormalities, defined as

    1. Hemoglobin levels of ≥1 g/dL below the lower limit of normal for their age and gender AND/OR
    2. A platelet count of <90 × 10^9/L below the lower limit of normal for their age and gender
    3. Has Gaucher disease-related viscera abnormalities, defined as the following:

    1. Participant has at least moderate splenomegaly, assessed by palpation (2 to 3 cm below the left costal margin), or by abdominal radiology scan (magnetic resonance imaging [MRI] or computed tomography [CT] scan, with spleen volume >5 times normal) AND/OR
    2. Participant has hepatomegaly, assessed by palpation or by abdominal radiology scan (MRI or CT scan); Participants who have undergone splenectomy must have satisfied these criteria for this study.

    Exclusion:


    1. Has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease as assessed by the investigator
    2. Has had a splenectomy or an active, clinically significant spleen infarction within the 12 months prior to screening
    3. Has received treatment with any investigational drug or device within 30 days prior to screening, or within 5 half-lives of that investigational product, whichever is greater; such treatment during the study will not be permitted
    4. Is currently receiving red blood cell growth factor (eg, erythropoietin), chronic systemic corticosteroids, or has been on such treatment within the 6 months prior to screening
    5. Presents with non-Gaucher disease related exacerbated anemia at screening
    6. Has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)

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    Study Locations

    Enter your ZIP code/Postal code/PIN code to locate study sites near you:

    How to Apply


    Contact the study center to learn if this study is a good match for you.
    Phone iconCall +1-877-825-3327Email iconEmail Study Center

    Study Details


    Contition
    Gaucher Disease
    Age (in years)
    2+
    Phase
    PHASE3
    Participants Needed
    20
    Est. Completion Date
    Aug 5, 2024
    Treatment Type
    INTERVENTIONAL

    Sponsor
    Takeda
    ClinicalTrials.gov NCT Identifier
    NCT05529992
    Study Number
    TAK-669-3001

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