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

NCT02528617 · Status: WITHDRAWN · Phase: PHASE4 · Type: INTERVENTIONAL

Last updated 2018-03-02

No results posted yet for this study

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.

Conditions

  • Gaucher Disease Type 1
  • Gaucher Disease Type 3

Interventions

DRUG

Velaglucerase alfa

Enzyme replacement therapy

Sponsors & Collaborators

  • Texas Scottish Rite Hospital for Children

    collaborator OTHER

  • Baylor Research Institute

    lead OTHER

Principal Investigators

  • Raphael Schiffmann, M.D.,M.H.Sc. · Baylor Research Institute/Institute of Metabolic Disease

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-07-31
Primary Completion
2017-10-31
Completion
2017-10-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02528617 on ClinicalTrials.gov