Biological Sample Collection for Parkinson's (NCT05536388)
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?
- You are never charged a fee to join a legitimate clinical trial, and the study treatment and study-specific tests are provided by the trial sponsor at no charge. In the US, routine care you would receive anyway can still be billed to you or your insurance; coverage rules differ by country, and trials in the UK and Canada usually run through public health systems. Travel, parking, and time are real costs, and some trials reimburse them while others do not. Before enrolling, ask the study coordinator to explain in writing what is and is not covered.
- How long does the trial last?
- This trial is estimated to last approximately 3 years.
- 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.
- 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.
- Will I get a placebo in this trial?
- This is an observational study, so there is no placebo and no experimental treatment is assigned. Researchers observe and collect information while you continue your usual care.
- What procedures are involved in this trial?
- Based on the protocol, this trial involves: Blood draw (minimally invasive); Questionnaires & surveys (non-invasive). Confirm the full schedule with the study coordinator.