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

Sponsor
Baylor Research Institute (Other)
Overall Status
Withdrawn
CT.gov ID
NCT02528617
Collaborator
Texas Scottish Rite Hospital for Children (Other)
0
Enrollment
1
Location
1
Arm
27
Anticipated Duration (Months)
0
Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease Intervention/Treatment Phase
  • Drug: Velaglucerase alfa
 Phase 4

Study Design

Study Type:
Interventional
Actual Enrollment :
0 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Anticipated Study Start Date :
Jul 1, 2015
Actual Primary Completion Date :
Oct 1, 2017
Actual Study Completion Date :
Oct 1, 2017

Arms and Interventions

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. 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. [Baseline pre-intervention and yearly thereafter for 3 years]

    Secondary Outcome Measures

    1. Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa. [Baseline pre intervention and yearly thereafter for 3 years]

    2. Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa. [Baseline pre intervention and yearly thereafter for 3 years]

    3. Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa. [Baseline pre-intervention and yearly thereafter for 3 years]

    4. Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa. [Baseline pre-intervention and yearly thereafter for 3 years.]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    4 Years to 14 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Baylor Research Institute Dallas Texas United States 75226

    Sponsors and Collaborators

    • Baylor Research Institute
    • Texas Scottish Rite Hospital for Children

    Investigators

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

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Baylor Research Institute
    ClinicalTrials.gov Identifier:
    NCT02528617
    Other Study ID Numbers:
    • 014-285
    First Posted:
    Aug 19, 2015
    Last Update Posted:
    Mar 2, 2018
    Last Verified:
    Feb 1, 2018
    Additional relevant MeSH terms:
    Gaucher Disease
    Disease

    Study Results

    No Results Posted as of Mar 2, 2018