Overview

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

Status:
Withdrawn
Trial end date:
2017-10-01
Target enrollment:
0
Participant gender:
All
Summary
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.
Phase:
Phase 4
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Baylor Research Institute
Collaborator:
Texas Scottish Rite Hospital for Children
Criteria
Inclusion Criteria:

- 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.

Exclusion Criteria:

- Clinically unstable,

- taking or have taken bisphosphonates,

- Gaucher type 2,

- pregnant female,

- or deemed inappropriate for participation by the principal investigator.