Now Recruiting

Phase 4 Study of the Effect of Velaglucerase alfa on Bone-related Pathology in Patients with Type 1 Gaucher Disease

WHAT IS THE PURPOSE OF THIS STUDY?

The purpose of this study is to evaluate the effect of Velaglucerase alfa therapy in treatment-naïve patients with type 1 Gaucher disease by measuring the change in the lumbar spine (LS) bone mineral density (BMD) after 12 and 24 months of treatment.

TRIAL AT A GLANCE

Condition

Gaucher Disease

Study Intervention

Velaglucerase Alfa

Possibility of Active Treatment

100%

Study Type

Interventional

Phase

Phase 4

IS THIS STUDY RIGHT FOR ME?

AGE

18 to 65

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

PARTICIPATION DURATION/ REQUIREMENTS

Participation will last for approximately 110 weeks, including 102 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 ≤65 years of age

Documented diagnosis of type 1 Gaucher disease

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

Must not have had a splenectomy (spleen removed)

Must have below average bone density (BMD Z-score at the lumbar spine <-1)

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

  • 1.

    Kaiser Permanente

    Los Angeles, California
    United States of America

    dvats@chla.usc.edu

    miles

  • 2.

    Hospital Universitario Ramon y Cajal

    Madrid,
    Spain

    + 34 913 368 686

    jesus.vallarrubia@salud.madrid.org

    miles

  • 3.

    Royal Free Hospital

    London,
    United Kingdom

    020 77943 4154

    rmgvdah@ucl.ac.uk

    miles

  • 4.

    NYU School of Medicine - Hospital

    New York, New York
    United States of America

    212-263-0139

    miles

  • 5.

    Gaucher Clinical Department of Medicine

    Jerusalem,
    Israel

    972-2-6555143

    miles

  • 6.

    Hospital Quironsalud Zaragoza

    Zaragoza, Zaragoza
    Spain

    +34976765546

    miles

  • 7.

    Duke University Medical Center

    Durham, NC
    United States of America

    9196819854

    kishn001@mc.duke.edu

    miles

  • 8.

    Emory Genetics

    Decatur, GA
    United States of America

    404-778-3543

    miles

  • 9.

    Addenbrooke's Hospital

    Cambridge, Cambridge
    United Kingdom

    +4401223274634

    patrick.deegan@addenbrookes.nhs.uk

    miles

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