Clinical Trial: The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
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 Summary:
Sponsor: Baylor Research Institute
Current Primary Outcome: 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 ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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. ]
Original Secondary Outcome: Same as current
Information By: Baylor Research Institute
Dates:
Date Received: July 28, 2015
Date Started: July 2015
Date Completion: July 2021
Last Updated: August 18, 2015
Last Verified: August 2015