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Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

NCT04055831 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 40

Last updated 2019-08-15

No results posted yet for this study

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, osteoporosis, marrow infiltration, avascular necrosis, and osteolysis. However, the underlying cellular/molecular basis of bone involvement and related complications in GD are not fully known. Neither are there any bone-specific markers associated with individual bone pathology. Early diagnosis of bone disease is the key issue for planning individual therapy to prevent and reverse bone disease in GD.

Conditions

  • Gaucher Disease Type 1

Sponsors & Collaborators

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    lead OTHER

Principal Investigators

  • Ozlem Goker-Alpan, MD · Lysosomal and Rare Disorders Research and Treatment Center

Eligibility

Min Age
16 Years
Max Age
90 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2019-05-15
Primary Completion
2020-04-15
Completion
2020-05-15

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04055831 on ClinicalTrials.gov