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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT02528617 |
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Date of registration:
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28/07/2015 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
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Scientific title:
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The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease |
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Date of first enrolment:
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July 2015 |
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Target sample size:
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0 |
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Recruitment status: |
Withdrawn |
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URL:
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https://clinicaltrials.gov/show/NCT02528617 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Countries of recruitment
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United States
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Contacts
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Name:
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Raphael Schiffmann, M.D.,M.H.Sc. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Baylor Research Institute/Institute of Metabolic Disease |
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Key inclusion & exclusion 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.
Age minimum:
4 Years
Age maximum:
14 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Gaucher Disease Type 3
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Gaucher Disease Type 1
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Intervention(s)
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Drug: Velaglucerase alfa
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Primary Outcome(s)
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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]
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Secondary Outcome(s)
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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]
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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]
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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]
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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.]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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