Status:
UNKNOWN
Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease
Lead Sponsor:
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Conditions:
Gaucher Disease Type 1
Eligibility:
All Genders
16-90 years
Brief Summary
Bone-related problems represent the principal unmet medical need in Gaucher disease (GD). 75% of GD type 1 patients develop skeletal complications, including bone remodeling defects, osteopenia, osteo...
Detailed Description
This clinical observational study is designed to identify specific biomarkers for bone involvement in patients with GD1 with decreased bone density and/or bone structural abnormalities Aims: 1. Iden...
Eligibility Criteria
Inclusion
- To be enrolled in this study the subject must meet the following criteria
- Subject is greater than 16 years old but not older than 90 years
- Signed Informed Consent/Assent
- Subject is able and willing to sign informed consent or assent
- If the subject has GD1, the must have a confirmed diagnosis of Gaucher disease by
- GCase enzyme activity
- DNA analysis demonstrating pathogenic variants in the GBA gene
Exclusion
- a) Have evidence of hepatitis B, hepatitis C infection or any other chronic infectious disease b) Be pregnant or breastfeeding
Key Trial Info
Start Date :
May 15 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
May 15 2020
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT04055831
Start Date
May 15 2019
End Date
May 15 2020
Last Update
August 15 2019
Active Locations (1)
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1
LDRTC
Fairfax, Virginia, United States, 22030