NCT05702814Completed
A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition
Validation of a Set of Potential Biomarkers Predictive of Bone Complications in Type 1 Gaucher Disease Patients
Source: ClinicalTrials.gov · Last updated 11 Dec 2024
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 125 (actual)
- First submitted
- 18 Jan 2023
- Start date
- 27 Mar 2023
- Primary completion date
- 20 May 2024
- Lead sponsor
- Takeda
- Diseases
- Gaucher disease
- NCT number
- NCT05702814
What you commit to
- Study type: Observational
- Participants enrolled: 125
- From 27 Mar 2023 to the primary completion date, 20 May 2024
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study design.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Evidence of hepatitis B, hepatitis C infection or other chronic infectious diseases.
- For Healthy Volunteers
- Evidence of hepatitis B, hepatitis C infection or other chronic infectious diseases.
- Evidence of bone disease.
Who the study is looking for
- Participants with confirmed diagnosis of GD1.
- Determination of Glucocerebrosidase (GCase) blood activity at diagnosis of GD1.
- DNA analysis result demonstrating pathogenic variants in the Glucocerebrosidase gene (GBA1) gene.
- Available data of clinical state at diagnosis and at 4-5 years from diagnosis, including S-MRI, Dual energy x-ray absorptiometry (DXA), and GD1 severity scoring system (GD1-DS3) indexes (or data to calculate it).
Criteria the registry did not label
- For Participants with GD1:
Age in the registry: 18 years to - · Sex: All · Accepts healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Fundación Española para el Estudio y Tratamiento de la Enfermedad de Gaucher (FEETEG) Coordinator of this site? Confirm its status | Zaragoza | Spain | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.