Takeda
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.
Gaucher Disease
No Intervention
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.}}
Study Type : | Observational |
Estimated Enrollment : | 125 participants |
Official Title : | Validation of a Set of Potential Biomarkers Predictive of Bone Complications in Type 1 Gaucher Disease Patients |
Actual Study Start Date : | March 27, 2023 |
Estimated Primary Completion Date : | May 31, 2024 |
Estimated Study Completion Date : | May 31, 2024 |
Information not available for Arms and Intervention/treatment
Ages Eligible for Study: | 18 Years |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Accepts Healthy Volunteers |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
Not yet recruiting
Spanish Foundation for the Study and Treatment of Gaucher Disease (FEETEG)
Zaragoza, Aragon, Spain, 50006