Now Recruiting

Phase 4 study to evaluate the effect of Velaglucerase alfa (VPRIV®) on patients with Type 1 Gaucher Disease through the IV administration of VPRIV® over 2 years

WHAT IS THE PURPOSE OF THIS STUDY?

To evaluate the effect of VPRIV® therapy on patients with type 1 Gaucher disease by measuring the change in the lumbar spine (LS) and bone mineral density (BMD) after 24 months of treatment.

TRIAL AT A GLANCE

Condition

Gaucher Disease

Study Intervention

Velaglucerase alfa; 800 International Unit (IU) Vitamin D

Possibility of Active Treatment

100%

Study Type

Interventional

Phase

Phase 4

IS THIS STUDY RIGHT FOR ME?

AGE

18 to 70

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

PARTICIPATION DURATION/ REQUIREMENTS

Participation for each participant will last for approximately 110 weeks, including 101 weeks of treatment (51 infusions), additional screening/baseline assessments taking up to 4 weeks, and 4 weeks of safety follow up after treatment is completed.

ENTRY CRITERIA

Males and females ≥18 and ≤70 years of age

Must have documented diagnosis of type 1 Gaucher disease

Must be treatment-naïve, i.e. must not have received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the 12 months prior to enrollment

Cannot have any osteoporosis-specific treatment or treatment with erythropoietin (or erythropoietin-like substances) during the past year

Cannot have had a splenectomy

Cannot have severe vitamin D deficiency to the level that would be expected to result in osteomalacia

Cannot be enrolled in another clinical study within 30 days prior to study enrollment or at any time during the study

Study Locations

We are planning* on conducting this study in 4 countries around the world.

    • Israel
    • Spain
    • United Kingdom
    • United States of America

Enter your city or postal code to find a location near you.

RESULTS

*Planning is an intent to conduct this study in the specified countries and is subject to change.