The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Withdrawn before enrolling · Phase 4

Conditions studied: Gaucher Disease Type 1, Gaucher Disease Type 3

In brief

The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

Key facts

Study ID

NCT02528617

Run by

Baylor Research Institute

People needed

0

Starts

2015-07-01

Expected to finish

2017-10-01

Last updated by the study team

2018-03-02

Who can join

Age: 4 and older, up to 14. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Enzyme Replacement Therapy naive,
• confirmed diagnosis of Gaucher disease type 1 or 3,
• able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years,
• able to tolerate all study procedures,
• skeleton not fully formed as confirmed by DXA and MRI),
• and willing to receive velaglucerase alfa infusions every other week for the duration of the study.

You may not qualify if…

• Clinically unstable,
• taking or have taken bisphosphonates,
• Gaucher type 2,
• pregnant female,
• or deemed inappropriate for participation by the principal investigator.

Where it is running

• Baylor Research Institute — Dallas, Texas, United States

Full record on ClinicalTrials.gov

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