Refer patients to this study

Velaglucerase alfa (VPRIV®) is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type I Gaucher disease. The SHP-GCB-402 Gaucher disease study will evaluate the effect of VPRIV® on bone pain and density in people with type 1 Gaucher disease. If you are currently treating a patient with type 1 Gaucher disease, please consider referring him or her to this study.

This study is an open-label, multicenter, single-arm, Phase 4 study of the effect of treatment with VPRIV® on bone-related pathology in treatment-naïve patients with type I Gaucher disease. Patients who qualify will meet at least the following inclusion criteria:

KEY INCLUSION CRITERIA

  1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes (whole blood only) or cultured skin fibroblasts.
    • Diagnosis by dry blood spot test is not sufficient.
    • Diagnosis may be based on results obtained prior to screening if documented in the patient's medical history.
  2. Patient is treatment-naïve, ie, has not received ERT or substrate reduction therapy (SRT) in the 12 months prior to enrollment.
  3. The patient is ≥18 and ≤65 years of age.
  4. Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study.

The study doctor will assess additional eligibility criteria during the screening visit.

If you have any questions or are interested in speaking with a study physician about referring patients, please feel free to get in touch with the study team using the contact form on this page. We look forward to hearing from you.

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Your contact information will only be used for study-related communications and will not be shared with anyone outside of the Gaucher study, SHP-GCB-402.