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NCT02574286 · ClinicalTrials.gov registry record · Phase 4
Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
A Phase 4 study of Gaucher Disease, sponsored by Shire.
- Completed
- Registry status
- Phase 4
- Development phase
- 21
- Enrollment target
- 15
- Study locations
NCT02574286: Completed Phase 4 study of Gaucher Disease, sponsored by Shire.
NCT02574286 is a Phase 4 study of Gaucher Disease that has completed, run by Shire. The registered enrollment target is 21 participants, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (98% lower). The trial reports 15 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02574286, a Phase 4 study of Gaucher Disease, has completed, sponsored by Shire.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 21 participants
- Enrollment target
- 15
- Study locations
Study Summary
The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram \[U/kg\] every other week \[EOW\]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment.
Primary Outcome
Bone mineral density of the lumbar spine was measured by dual energy x-ray absorptiometry (DXA), and the results was converted to Z-scores appropriate for age, sex, and race. The Z-score indicated the number of standard deviations away from a reference population in the same age range, race and with the same sex. A Z-score of 0 was equal to the mean. Negative numbers indicated values lower than the mean and positive numbers indicated values higher than the mean. Baseline was defined as last data
Conditions Studied
Interventions
- DIETARY_SUPPLEMENT Vitamin D
- DRUG Velaglucerase alfa
Study Locations (15)
Other
- Rambam Health Care Campus - Haifa
- Shaare Zedek Medical Center - Jerusalem
- Rabin Medical Center - Petah Tikva
- Hospital Universitario Ramon y Cajal - Madrid
- Hospital Quironsalud Zaragoza - Zaragoza
- Addenbrooke's Hospital - Cambridge
- Royal Free Hospital - London
California
- Cedars Sinai Medical Center - Beverly Hills
- Kaiser Permanente - Los Angeles
Georgia
- Emory Genetics - Atlanta
Illinois
- Ann and Robert H Lurie Childrens Hospital of Chicago - Chicago
New York
- NYU School of Medicine - New York
North Carolina
- Duke University Medical Center - Durham
Pennsylvania
- The Children's Hospital of Philadelphia - Philadelphia
Virginia
- Lysosomal and Rare Disorders Research and Treatment Center - Fairfax
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2016-06-29 |
| Est. Completion | 2020-11-30 |
| Phase | Phase 4 |
What the finished NCT02574286 record still lists
NCT02574286 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 21 participants, a relatively small participant target, below the 1,057-participant average among 13 other Gaucher Disease trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Gaucher Disease appearing as the primary indexed condition, and to 2 interventions - of which Vitamin D is the first listed.
NCT02574286 lists 15 locations in 8 states (Other, California, Georgia).
Frequently Asked Questions
What is clinical trial NCT02574286 about?
NCT02574286 is a clinical study titled "Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease". The primary purpose of this study is to evaluate the effect of VPRIV therapy (60 units per kilogram \[U/kg\] every other week \[EOW\]) in treatment-naive participants with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energ...
What is the current status of trial NCT02574286?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 21 participants. The study started on 2016-06-29. Estimated completion is 2020-11-30.
What conditions does trial NCT02574286 study?
This clinical trial studies the following conditions: Gaucher Disease.
What interventions are being tested in trial NCT02574286?
The interventions under investigation include: Vitamin D (DIETARY_SUPPLEMENT), Velaglucerase alfa (DRUG).
Who is sponsoring clinical trial NCT02574286?
This trial is sponsored by Shire, which has 245 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02574286 being conducted?
This trial has 15 study locations across California, Georgia, Illinois, New York, North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT02574286's enrollment target sits among peer trials
21 11th of 13 higher than 3 of 13 other Gaucher Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Gaucher Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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