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

Gaucher Disease Type 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02528617
• Other study ID #: 014-285
• Status: Withdrawn
• Phase: Phase 4
• First received: July 28, 2015
• Last updated: February 28, 2018
• Start date: July 2015
• Est. completion date: October 2017

Study information

• Verified date: February 2018
• Source: Baylor Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: October 2017
• Est. primary completion date: October 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 14 Years

Eligibility

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.

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease Type 1
• Gaucher Disease Type 3

Intervention

Drug:
• Velaglucerase alfa
Enzyme replacement therapy

Locations

Country	Name	City	State
United States	Baylor Research Institute	Dallas	Texas

Sponsors (2)

Lead Sponsor	Collaborator
Baylor Research Institute	Texas Scottish Rite Hospital for Children

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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.