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

Phase 4

Withdrawn

• Conditions: Gaucher Disease Type 1; Gaucher Disease Type 3
• Interventions: Drug: Velaglucerase alfa

• Registration Number: NCT02528617
• Lead Sponsor: Baylor Research Institute

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-07
• Study First Submitted: 2015-07-28
• Study First Submitted QC: 2015-08-18
• Study First Posted: 2015-08-19
• Primary Completion: 2017-10
• Study Completion: 2017-10
• Status Verified: 2018-02
• Last Update Submitted: 2018-02-28
• Last Update Posted: 2018-03-02

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Gaucher Type 1 or 3	Velaglucerase alfa	Velaglucerase alfa IV 60 units/kg every other week for duration of the study.

Primary Outcome Measures

Name	Time	Method
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.	Baseline pre-intervention and yearly thereafter for 3 years

Secondary Outcome Measures

Name	Time	Method
Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.	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.	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.	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.	Baseline pre-intervention and yearly thereafter for 3 years

Trial Locations

Locations (1)

Baylor Research Institute; 🇺🇸 Dallas, Texas, United States