Back to resultsThe Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Primary Purpose
Gaucher Disease Type 1, Gaucher Disease Type 3
Status
Withdrawn
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Velaglucerase alfa
Sponsored by
About this trial
This is an interventional treatment trial for Gaucher Disease Type 1
Eligibility Criteria
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.
Sites / Locations
- Baylor Research Institute
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
Gaucher Type 1 or 3
Arm Description
Velaglucerase alfa IV 60 units/kg every other week for duration of the study.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Change in degrees/second from baseline in saccadic eye movements in children with Gaucher type 3 receiving velaglucerase alfa.
Change from baseline neurodevelopmental testing normalized scores in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Change from baseline brainstem auditory evoked potential results measured in microvolts in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Change from baseline EEG (electroencephalogram) results measured in Hertz in children with Gaucher Disease type 3 receiving velaglucerase alfa.
Full Information
NCT ID
NCT02528617
First Posted
July 28, 2015
Last Updated
February 28, 2018
Sponsor
Baylor Research Institute
Collaborators
Texas Scottish Rite Hospital for Children
1. Study Identification
Unique Protocol Identification Number
NCT02528617
Brief Title
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Official Title
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
Study Type
Interventional
2. Study Status
Record Verification Date
February 2018
Overall Recruitment Status
Withdrawn
Why Stopped
No subjects accrued and no potential subjects were identified..
Study Start Date
July 2015 (Anticipated)
Primary Completion Date
October 2017 (Actual)
Study Completion Date
October 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baylor Research Institute
Collaborators
Texas Scottish Rite Hospital for Children
4. Oversight
Data Monitoring Committee
No
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease Type 1, Gaucher Disease Type 3
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Gaucher Type 1 or 3
Arm Type
Other
Arm Description
Velaglucerase alfa IV 60 units/kg every other week for duration of the study.
Intervention Type
Drug
Intervention Name(s)
Velaglucerase alfa
Other Intervention Name(s)
VPRIV
Intervention Description
Enzyme replacement therapy
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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
Title
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.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Raphael Schiffmann, M.D.,M.H.Sc.
Organizational Affiliation
Baylor Research Institute/Institute of Metabolic Disease
Official's Role
Principal Investigator
Facility Information:
Facility Name
Baylor Research Institute
City
Dallas
State/Province
Texas
ZIP/Postal Code
75226
Country
United States
12. IPD Sharing Statement
Learn more about this trial
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
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