The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease
| Status: | Withdrawn |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | 4 - 14 |
| Updated: | 4/17/2018 |
| Start Date: | July 2015 |
| End Date: | October 2017 |
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.
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.
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.
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