NCT02574286

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of Treatment With Velaglucerase Alfa on Bone-related Pathology in Treatment-naïve Patients With Type 1 Gaucher Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Velaglucerase alfa	Participants will receive 60-minute intravenous infusion of 60 U/kg velaglucerase alfa EOW.
DIETARY_SUPPLEMENT	Vitamin D	Participants will receive 800 IU vitamin D orally daily.

Timeline

• Start date: 2016-06-29
• Primary completion: 2020-11-12
• Completion: 2020-11-30
• First posted: 2015-10-12
• Last updated: 2022-02-01
• Results posted: 2022-02-01

Locations

15 sites across 4 countries: United States, Israel, Spain, United Kingdom

Regulatory

• FDA-regulated drug study