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

Baylor Research Institute

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Gaucher Disease Type 1 Gaucher Disease Type 3

Interventions

Velaglucerase alfaDRUG

Enzyme replacement therapy

Eligibility

Sex: ALLMin age: 4 YearsMax age: 14 Years

Medical Language ↔ Plain English

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.

Locations (1)

Baylor Research Institute

Dallas, Texas, United States

Outcomes

Primary Outcomes

Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.

Time frame: Baseline pre-intervention and yearly thereafter for 3 years

Secondary Outcomes

Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.

Time frame: Baseline pre intervention and yearly thereafter for 3 years

Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.

Time frame: Baseline pre intervention and yearly thereafter for 3 years

Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.

Time frame: Baseline pre-intervention and yearly thereafter for 3 years

Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.

Time frame: Baseline pre-intervention and yearly thereafter for 3 years.

Data from ClinicalTrials.gov

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