NCT05253560

Prodromal Parkinsonian Features in GBA1 Mutation Carriers

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 600 (estimated)
• Sponsor: Shaare Zedek Medical Center · Academic / Other
• Sex: All
• Age: 40 Years – 75 Years
• Healthy volunteers: Accepted

Summary

Objective of the trial. To define a sub-population which is at increased risk of developing Parkinson, beyond the fact of carrying Gaucher; in this sub-population the investigators shall conduct a comprehensive evaluation that includes a variety of non-invasive tests, whose purpose is to evaluate the state of the pre- Parkinson's disease signs, signs which can appear, even twenty years before the appearance of the disease, and also to compare them to a group of diagnosed Gaucher patients and a group of healthy people who are not carriers of Gaucher disease. A group of those carriers will be available for trial or for treatment, if there will be a medicine for the prevention of the development of Parkinson, obtainable.

Detailed description

The carriers, the healthy people and the Gaucher patients will undergo a number of non-invasive tests, which test the pre-Parkinson signs. The tests are based on various sense tests, blood tests, measuring of blood pressure, lying and standing, ultra-sound of the brain and a neurological test (test of the nervous system and various questionnaires).

Conditions

• Gaucher Disease, Type 1
• Healthy

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	The investigators aim to identify prodromal PD in a cohort of carriers of Gaucher disease.	Tests to help identify prodromal PD in a cohort of carriers of Gaucher disease

Timeline

• Start date: 2017-05-16
• Primary completion: 2024-01-16
• Completion: 2025-12-31
• First posted: 2022-02-24
• Last updated: 2023-11-24

Locations

1 site across 1 country: Israel