Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Condition: Gaucher Disease
MeSH terms: Gaucher Disease
Intervention: Velaglucerase alfa; Vitamin D
Intervention description: Participants will receive 60-minute intravenous infusion of 60 U/kg velaglucerase alfa EOW.; Participants will receive 800 IU vitamin D orally daily.
Intervention MeSH terms: Vitamin D
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 21.0
Sex: All
Minimum age: 18 Years
Maximum age: 70 Years
Healthy volunteers: No

Summary:

      The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per
      kilogram [U/kg] every other week [EOW]) in treatment-naive participants with type 1 Gaucher
      disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as
      measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.
    

Eligibility criteria:

        Inclusion Criteria:

          -  The participant has a documented diagnosis of type 1 Gaucher disease, as documented by
             deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts.
             Diagnosis by only dry blood spot test is insufficient. Diagnosis may be based on
             results obtained prior to screening if documented in the participant's medical
             history.

          -  Participants must have a LS BMD Z-score less than (<) -1 or BMD T-score of < -1 as
             measured by DXA during the screening phase.

          -  Participant is treatment-naive, that is (ie,) has not received ERT or SRT in the 12
             months prior to enrollment.

          -  The participant is greater than or equal to (>=) 18 and less than or equal to (<=) 70
             years of age.

          -  Female participants of childbearing potential must agree to use a medically acceptable
             method of contraception at all times during the study.

          -  The participant, or participant's legally authorized representative(s), if applicable,
             understands the nature, scope, and possible consequences of the study and has provided
             written informed consent that has been approved by the Institutional Review
             Board/Independent Ethics Committee (IRB/IEC).

          -  The participant must be sufficiently cooperative to participate in this clinical study
             as judged by the investigator.

        Exclusion criteria

          -  Neurological symptoms indicating that the participant may have type 3 Gaucher disease.

          -  A significant comorbidity, which, as determined by the investigator, might affect
             study data or confound the study results (eg, malignancies, primary biliary cirrhosis,
             autoimmune liver disease, etc).

          -  Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with
             erythropoietin (or erythropoietin-like substances) during the past year.

          -  Structural, joint-associated bone damage of such extent and severity that the
             investigator deems it could impact participation in the study and assessment of
             relevant study endpoints (example, pain).

          -  The participant is pregnant or lactating.

          -  The participant has had a splenectomy. (This criterion is not meant to exclude
             participants who have accessory spleens.)

          -  The participant is enrolled in another clinical study that involves clinical
             investigations or use of any investigational product (drug or device) within 30 days
             prior to study enrollment or at any time during the study.

          -  Severe vitamin D deficiency to the level that would be expected to result in
             osteomalacia (vitamin D < 10 nanograms per milliliter [ng/mL] [25 nanomoles per liter
             {nmol/L}]). If there is mild vitamin D insufficiency at screening (vitamin D greater
             than [>] 10 and < 30 ng/mL) treat with 4000 IU vitamin D per day for 1 month and
             rescreen.

          -  The participant has previously interrupted ERT for safety reasons.

          -  The participant has had hypersensitivity to the active substance or to any of the
             excipients.