Status:
UNKNOWN
Biomarkers Related to Bone in Pediatric Gaucher Disease
Lead Sponsor:
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Conditions:
Gaucher Disease
Bone Diseases
Eligibility:
All Genders
5-21 years
Brief Summary
Aims of the observational study is to establish novel blood-based biomarkers for grading bone disease in pediatric patients with Gaucher disease (GD). Patients with clinically confirmed GD: deficient ...
Detailed Description
Gaucher disease (GD), the most common lysosomal storage disorder, is caused by a deficiency of the enzyme glucocerebrosidase. Clinically, there are three sub-types according to neurological involvemen...
Eligibility Criteria
Inclusion
- The parent or legal guardian and the participant who is eligible to provide assent are able and willing to provide informed consent and assent when applicable.
- The participant is 5-21 years of age at the initial visit.
- The participant has a confirmed diagnosis of GD type 1 or type 3 (biochemically and/or genetically).
- In the investigator's opinion, the subject is capable of understanding and complying with protocol requirements.
- The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
Exclusion
- Any subject who does not meet any of the following criteria will not qualify for the study.
- Any current active chronic infection such as HIV, Hepatitis B or C.
- Pregnancy or breastfeeding for females.
Key Trial Info
Start Date :
November 25 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2025
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT06116071
Start Date
November 25 2023
End Date
December 31 2025
Last Update
March 13 2024
Active Locations (1)
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1
Lysosomal and Rare disorder research and treatment center
Fairfax, Virginia, United States, 22030