Dallas, Texas 75226


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.


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.



Primary Contact:

Principal Investigator
Raphael Schiffmann, M.D.,M.H.Sc.
Baylor Research Institute/Institute of Metabolic Disease

Mary E Wallace, MSRD/LD
Phone: 214-820-4752
Email: marywall@baylorhealth.edu

Backup Contact:

Email: caren.swift@baylorhealth.edu
Caren Swift, BSN
Phone: 214-820-4857

Location Contact:

Dallas, Texas 75226
United States

Mary E Wallace, MSRD/LD
Phone: 214-820-4752
Email: marywall@baylorhealth.edu

Site Status: Recruiting

Data Source: ClinicalTrials.gov

Date Processed: November 17, 2017

Modifications to this listing: Only selected fields are shown, please use the link below to view all information about this clinical trial.

Click to view Full Listing

If you would like to be contacted by the clinical trial representative please fill out the form below.