WithdrawnPhase 4

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

Stopped: No subjects accrued and no potential subjects were identified..

United States0Started 2015-07

Plain-language 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.

Who can participate

Age range4 Years – 14 Years

SexALL

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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.

What they're measuring

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.

Timeframe: Baseline pre-intervention and yearly thereafter for 3 years

Trial details

NCT IDNCT02528617

SponsorBaylor Research Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-10

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