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
Summary For Families
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.
- Will I get the real drug or a placebo?
- It depends on the trial design. Some Parkinson's trials are placebo controlled, meaning a portion of participants get an inactive comparison while others get the experimental treatment. Some use crossover designs so everyone eventually receives the active treatment at some point. Observational trials do not use a placebo at all because they are not testing a new treatment. The consent form for any specific trial spells out the design and your odds of being assigned to placebo before you enroll.
- How often will I need to visit the study site?
- Visit frequency varies by trial. Many Parkinson's studies require an in person visit every 4 to 12 weeks during the active treatment phase, with shorter or longer gaps depending on the design. The site coordinator can give you the full visit schedule before you sign anything, so you know what the time commitment looks like.
- Can I leave the trial if I change my mind?
- Yes. You can withdraw from any clinical trial at any time, for any reason, without affecting your standard medical care. Trials are voluntary by law. The team may ask if you are willing to do a brief exit visit so they can collect safety information, but you are not obligated.
- Will travel or parking be reimbursed?
- Many trials reimburse for parking, mileage, and sometimes lodging if the site is far from your home. Reimbursement policies vary by sponsor and site. When you contact the trial team, ask specifically what is covered and how reimbursement is processed.
- Can my spouse or care partner come with me to visits?
- In most cases yes, and it is often encouraged. Care partners can help with notes, questions, and getting home safely after a long visit. Some study assessments do need to happen one on one, but care partners are usually welcome for the rest of the appointment.