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Validation of a Set of Potential Biomarkers Predictive of Bone Complications in Type 1 Gaucher Disease Patients
Substances in the body, so-called biomarkers, can help predict the severity of Gaucher disease (GD)-related bone problems in adults. The main aim of the study is to determine if certain biomarkers found in the body at the time of diagnosing GD can help predict the risk of bone problems after 4-5 years. There is no treatment involved in this study. The study will review previously collected participants' data using a database. Data from both adults with type 1 Gaucher condition as well as healthy adults will be compared.
This is a non-interventional and retrospective study of participants with GD1 and healthy participants whose data is available in the Aragon Health Systems Biobank (BSSA) from the date of diagnosis to 4-5 years after diagnosis. The main objective of this study is to validate the prognosis value of a set of potential biomarkers related to bone disease in participants with GD1. The study will enroll approximately 120 participants, and it would be divided into 4 groups as given below: * Group A: GD1 with Low-Normal Bone Disease * Group B: GD1 with Mild Bone Disease * Group C: GD1 with Severe Bone Disease * Group D: Healthy Participants This study will have a retrospective data collection from the date of diagnosis of GD1 until 4-5 years from diagnosis by using data available in BSSA. This single-centre trial will be conducted in Spain. The overall time for data collection in this study will be approximately 9 months.
Age
18 - No limit years
Sex
ALL
Healthy Volunteers
Yes
Fundación Española para el Estudio y Tratamiento de la Enfermedad de Gaucher (FEETEG)
Zaragoza, Aragon, Spain
Start Date
March 27, 2023
Primary Completion Date
May 20, 2024
Completion Date
May 24, 2024
Last Updated
December 11, 2024
125
ACTUAL participants
No Intervention
OTHER
Lead Sponsor
Takeda
NCT05992532
NCT06573723
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