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

February 28, 2018 updated by: Baylor Research Institute
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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75226
        • Baylor Research Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 14 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Gaucher Type 1 or 3
Velaglucerase alfa IV 60 units/kg every other week for duration of the study.
Drug: Velaglucerase alfa
Enzyme replacement therapy
Other Names:
  • VPRIV

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Baseline pre-intervention and yearly thereafter for 3 years

Secondary Outcome Measures

Outcome Measure
Time Frame
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
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
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
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.
Baseline pre-intervention and yearly thereafter for 3 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Baylor Research Institute

Collaborators

Texas Scottish Rite Hospital for Children

Investigators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

July 1, 2015

Primary Completion (Actual)

October 1, 2017

Study Completion (Actual)

October 1, 2017

Study Registration Dates

First Submitted

July 28, 2015

First Submitted That Met QC Criteria

August 18, 2015

First Posted (Estimate)

August 19, 2015

Study Record Updates

Last Update Posted (Actual)

March 2, 2018

Last Update Submitted That Met QC Criteria

February 28, 2018

Last Verified

February 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 014-285

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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