Active, not 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

This study is no longer recruiting. Please check back for study results.

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

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

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

RESULTS

  • 1.

    Kaiser Permanente

    Los Angeles, California
    United States of America

    miles

  • 2.

    Hospital Universitario Ramon y Cajal

    Madrid,
    Spain

    miles

  • 3.

    Royal Free Hospital

    London,
    United Kingdom

    miles

  • 4.

    NYU School of Medicine

    New York, New York
    United States of America

    miles

  • 5.

    Hospital Quironsalud Zaragoza

    Zaragoza,
    Spain

    miles

  • 6.

    Duke University Medical Center

    Durham, North Carolina
    United States of America

    miles

  • 7.

    Emory Genetics

    Atlanta, Georgia
    United States of America

    miles

  • 8.

    Addenbrooke's Hospital

    Cambridge,
    United Kingdom

    miles

  • 9.

    Shaare Zedek Medical Center

    Jerusalem,
    Israel

    miles

  • 10.

    The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania
    United States of America

    miles

  • 11.

    Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois
    United States of America

    miles

  • 12.

    Cedars Sinai Medical Center

    Beverly Hills, California
    United States of America

    miles

  • 13.

    Rambam Health Care Campus

    Haifa,
    Israel

    miles

  • 14.

    Rabin Medical Center

    Petah Tikva,
    Israel

    miles

  • 15.

    Lysosomal and Rare Disorders Research and Treatment Center

    Fairfax, Virginia
    United States of America

    miles