Find drugs boosting cell cleanup
- Trial ID
- NCT05536388
- Official Title
- Drug Discovery for Parkinson's With Mutations in the GBA Gene
- Goal
- Find drugs boosting cell cleanup
- Status
- RECRUITING
- Sponsor
- New York Stem Cell Foundation Research Institute
- Study Type
- OBSERVATIONAL
- Enrollment
- 60 participants
- Conditions
- Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
- Interventions
- Biological Sample Collection
Plain-Language Summary
The goal is to speed discovery of drugs for people whose Parkinson's is linked to mutations in the GBA gene, which increases risk by weakening an enzyme called glucocerebrosidase and impairing the cell’s cleanup system, the lysosome. Researchers collect blood and skin samples to grow patients' cells in the lab, study how GBA mutations harm cell health, and screen compounds that might boost the enzyme or improve lysosomal function. Adults 18 and older with Parkinson's, people with Gaucher disease, and healthy volunteers can join if they can consent; people with bleeding disorders, a history of keloids for skin biopsies, allergy to local anesthetic, contraindicating anticoagulation, or AIDS with very low CD4 counts are excluded.
Locations
- The New York Stem Cell Foundation Research Institute, New York, New York, United States
Frequently Asked Questions
- What is this trial testing?
- This trial is studying Biological Sample Collection. The goal is to speed discovery of drugs for people whose Parkinson's is linked to mutations in the GBA gene, which increases risk by weakening an enzyme called glucocerebrosidase and impairing the cell’s cleanup system, the lysosome. Researchers collect blood and skin samples to grow patients' cells in the lab, study how GBA mutations harm cell health, and screen compounds that might boost the enzyme or improve lysosomal function. Adults 18 and older with Parkinson's, people with Gaucher disease, and healthy volunteers can join if they can consent; people with bleeding disorders, a history of keloids for skin biopsies, allergy to local anesthetic, contraindicating anticoagulation, or AIDS with very low CD4 counts are excluded.
- Who can participate?
- Participants must be at least 18 Years.
- Where is this trial located?
- This trial is recruiting at 1 location.
- Does it cost anything to join?
- No. There is no cost to participate. Study-related care and treatment are provided at no charge.
- How long does the trial last?
- This trial is estimated to last approximately 3 years.