Last updated on March 2017

If you have Gaucher disease, you may be able to participate in a clinical trial that is taking a deeper look into how the disease is treated. Read more to find out how you can get involved today!


Brief description of study

If you have Gaucher disease, you may be able to participate in a clinical trial that is taking a deeper look into how the disease is treated. Read more to find out how you can get involved today!

Detailed Study Description

ABOUT GAUCHER DISEASE AND THE SHP-GCB-402 STUDY

If you have type I Gaucher disease, glucocerebroside (a fatty substance) builds up in your body because you lack the enzyme glucocerebrosidase (GBC), which is needed to break it down. When this substance accumulates in your bone marrow, it leads to loss of bone density and possible broken bones, as well as extreme pain.

The SHP-GCB-402 study for Gaucher disease is evaluating the effect velaglucerase alfa (VPRIV®) has on bone pain and bone density in people with type I Gaucher disease. VPRIV® is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type I Gaucher disease. This study is designed to look at whether VPRIV® can reduce bone pain and protect bone density.

WHAT WILL PARTICIPANTS IN THE SHP-GCB-402 STUDY BE ASKED TO DO?

While receiving VPRIV®, participants will visit a study clinic approximately 14 times throughout the study. During the dosing period, these visits will be once every 3 months. The study medication will be administered as a 60-minute intravenous infusion (a drip) once every 2 weeks. The first 3 doses will be administered at a study clinic. All doses from thereon may be administered at the participant’s home by a member of the study team, if the study doctor (investigator) sees fit. Overall, participants will receive 51 infusions over the course of 102 weeks.

All study-related office visits, medical examinations, and study medication will be provided to qualified study participants at no cost to them.

DO PARTICIPANTS HAVE TO PAY TO JOIN?

No. VPRIV and all study-related assessments will be provided at no cost to you.

HOW SAFE IS THE STUDY MEDICATION?

As with most medications, some people may experience side effects. Also some unexpected side effects may occur.

WHY PARTICIPATE IN CLINICAL RESEARCH?

Clinical research helps discover whether treatments are safe and effective, and can help doctors find out what treatments work best for either specific illnesses or different kinds of patients. By asking specific questions, the study investigators test new medications or the effects of existing medications on specific disease symptoms. To see if you or someone you know may qualify for this study, to find a study location near you, or to be connected with a study doctor, visit www.GaucherType1Study.com

Clinical Study Identifier: TX147834

Contact Investigators or Research Sites near you

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Research Center

Research Center
New York, NY USA
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Research Center

Research Center
Durham, NC USA
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Research Center
Decatur, GA USA
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Research Center

Research Center
Aurora, CO USA
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Research Center

Research Center
Chicago, IL USA
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