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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE4

Study Classification

INTERVENTIONAL

Study Start Date

2015-07-31

Study Completion Date

2017-10-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Gaucher Disease Type 1 Gaucher Disease Type 3

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Gaucher Type 1 or 3

Velaglucerase alfa IV 60 units/kg every other week for duration of the study.

Group Type OTHER

Velaglucerase alfa

Intervention Type DRUG

Enzyme replacement therapy

Interventions

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Velaglucerase alfa

Enzyme replacement therapy

Intervention Type DRUG

Other Intervention Names

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VPRIV

Eligibility Criteria

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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.

Minimum Eligible Age

4 Years

Maximum Eligible Age

14 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No