Biomarkers Related to Bone in Pediatric Gaucher Disease

Launched by LYSOSOMAL AND RARE DISORDERS RESEARCH AND TREATMENT CENTER, INC. · Nov 2, 2023

Keywords

ClinConnect Summary

This clinical trial is studying how certain blood markers can help doctors understand bone issues in children and young adults with Gaucher disease, which is a genetic disorder affecting how the body processes a type of fat. The researchers want to find new ways to measure bone health by looking at specific substances in the blood of participants who have been diagnosed with Gaucher disease. They will also compare these results with healthy children to see how bone health differs between the two groups.

To participate, children and young adults aged 5 to 21 who have been diagnosed with Gaucher disease types 1 or 3 can join the study. Participants will undergo tests to measure their bone health and provide blood samples to check the levels of the targeted markers. It's important that the participants, along with their parents or guardians, understand and agree to take part in the study. Overall, this research aims to improve how we assess and treat bone problems in young patients with Gaucher disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. 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.
• • 2. The participant is 5-21 years of age at the initial visit.
• • 3. The participant has a confirmed diagnosis of GD type 1 or type 3 (biochemically and/or genetically).
• • 4. In the investigator's opinion, the subject is capable of understanding and complying with protocol requirements.
• • 5. 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 Criteria:
• • 1. Any subject who does not meet any of the following criteria will not qualify for the study.
• • 2. Any current active chronic infection such as HIV, Hepatitis B or C.
• • 3. Pregnancy or breastfeeding for females.