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