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• Status: Withdrawn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.