Research on Parkinson's disease using stem cells from patients with GBA gene mutations
Drug Discovery for Parkinson's With Mutations in the GBA Gene
This study is testing how stem cells from people with GBA gene mutations can help us understand and find new treatments for Parkinson's disease.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 60 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | New York Stem Cell Foundation Research Institute Academic / other |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT05536388 on ClinicalTrials.gov |
What this trial studies
This research conducted by the New York Stem Cell Foundation aims to advance the understanding and treatment of Parkinson's disease by utilizing biological samples, such as skin and blood, to create pluripotent stem cells. These stem cells can differentiate into various cell types, allowing researchers to study the disease mechanisms and potential therapies. Participants will provide health information, biological samples, and may undergo genetic testing to explore the role of the GBA gene mutation in Parkinson's disease. The study also includes the possibility of future follow-ups for additional research opportunities.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older diagnosed with Parkinson's disease, Gaucher disease, or those who are healthy controls.
Not a fit: Patients with coagulation disorders or those who refuse to adhere to safety protocols may not benefit from this study.
Why it matters
Potential benefit: If successful, this research could lead to new treatments or even cures for Parkinson's disease.
How similar studies have performed: Other studies utilizing stem cells for understanding neurodegenerative diseases have shown promise, indicating that this approach is both relevant and potentially impactful.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age 18 years or older. * Diagnosis of Parkinson's disease, Gaucher disease, or healthy control. * Must provide written informed consent unless physical limitations preclude signing. Exclusion Criteria: * For skin samples collected specifically for this study: history of keloid formation, coagulation disorder, allergy to the anesthetic, or anticoagulation use that precludes sample collection. * For blood samples collected specifically for this study: coagulation disorder or other medical condition(s) that increases the risks associated with blood collection. * For all prospective sample collections: Subjects who refuse to adhere to NYSCF's and/or a collection site's safety protocols will be excluded. Subjects with an AIDS diagnosis and CD4 count of less than 200 cells per microliter (mcL) of blood will be excluded due to increased risk of infection.
Where this trial is running
New York, New York
- The New York Stem Cell Foundation Research Institute — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Laura Andres-Martin — New York Stem Cell Foundation Research Institute
- Study coordinator: Clinical Research Office
- Email: clinicalresearch@nyscf.org
- Phone: 212-927-1801
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.