Dallas, Texas 75226


Purpose:

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.


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.


NCT ID:

NCT02528617


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

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