Parkinson DiseaseNCT05536388
Drug Discovery for Parkinson's With Mutations in the GBA Gene
An observational study of Parkinson Disease and Gaucher Disease, run by New York Stem Cell Foundation Research Institute at 1 site in United States.
Trial summary
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Parkinson's disease research and drug development by using cells from the body (such as skin or blood cells) to make stem cells and other types of cells, conduct research on the samples, perform genetic testing… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Parkinson Disease, Gaucher Disease, Healthy
- What is being tested?
- Biological Sample Collection
- How many people
- 60 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jul 2022to 15 Jul 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Individuals with a diagnosis of one or more of the listed conditions. Individuals without a condition to serve as healthy controls (a comparison group for those with conditions).
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
The New York Stem Cell Foundation Research Institute
New York, New York, 10019
Clinical Research Office, 212-927-1801, clinicalresearch@nyscf.org
Laura Andres-Martin
About this study
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Parkinson's disease research and drug development by using cells from the body (such as skin or blood cells) to make stem cells and other types of cells, conduct research on the samples, perform genetic testing, and/or store the samples for future use.
Through this research, researchers hope to identify future treatments or even cures for Parkinson's disease.
Read the detailed description
Researchers at the New York Stem Cell Foundation (NYSCF) Research Institute study diverse diseases, conditions, and traits by creating stem cells from biological samples. These "pluripotent" stem cells can become any cell in the human body, including cells that may be difficult, invasive, or impossible to obtain directly.
Additionally, researchers perform genetic testing to learn more about DNA, a material in most cells that contains instructions for the body's development and functions (such as traits like eye color and risk of certain diseases). A piece of DNA that determines the specific role of a cell is called a "gene." If the instructions in a gene are abnormal, this can lead to disease.
Participation in the study involves: (1) completion of health questionnaires, (2) providing a skin and/or blood sample from which stem cells may be created, (3) collection of a saliva sample for genetic analysis, and (4) possible future follow-up to provide additional information or learn about other research studies.
This study is not a clinical trial.
- Conditions
- Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
- Being tested
- Biological Sample Collection
- Keywords
- Parkinson Disease, GBA Gene Mutation
- Medical subject headings
- Parkinson Disease, Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biological Sample CollectionOther
Also called Biological sample collection for biobanking and research
Skin biopsy (2-3mm) and/or blood (up to 50 mL); saliva; excess/leftover biospecimens that were (or will be) collected for other purposes (e.g., medical procedure)
| Study group | Role | What they receive |
|---|---|---|
| Parkinson's Disease | Subjects in this group will have a diagnosis of Parkinson's disease with or without a known GBA gene mutation. Receives: Other: Biological Sample Collection | |
| Gaucher Disease | Subjects in this group with have a diagnosis of Gaucher disease with or without a known GBA gene mutation. Receives: Other: Biological Sample Collection | |
| Healthy Control | Subjects in this group will serve as healthy controls. Receives: Other: Biological Sample Collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Establishment of a diverse bank of biological samples, stem cell lines, derivatives, and associated information.
Establish a robust disease signature using a cohort of PD and carefully matched control samples to screen a library of approximately 3,000 compounds to determine whether any compounds improve or reverse disease phenotypes.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2022
- Study start15 Jul 2022
- Primary completion15 Jul 2025 (estimated)
- Study completion15 Jul 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- New York Stem Cell Foundation Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Silverstein Foundation
- Responsible party
- Sponsor
- Investigators
- Laura Andres-Martin, Principal investigator, New York Stem Cell Foundation Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05536388
- Sponsor's protocol ID
- 10-008
- Status last verified
- Jul 2024
- First submitted
- 7 Sep 2022
- Last updated
- 3 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Drug Discovery for Parkinson's With Mutations in the GBA Gene
- ClinicalTrials.gov ID
- NCT05536388
- Acronym / sponsor ID
- 10-008
- Conditions
- Parkinson Disease, Gaucher Disease, Healthy, GBA Gene Mutation
- Keywords
- Parkinson Disease, GBA Gene Mutation
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- New York Stem Cell Foundation Research InstituteUniversity, hospital or other
- Collaborators
- Silverstein Foundation
- Study start
- 15 Jul 2022 (actual)
- Primary completion
- 15 Jul 2025 (estimated)
- Study completion
- 15 Jul 2025 (estimated)
- First posted
- 10 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Parkinson's disease research and drug development by using cells from the body (such as skin or blood cells) to make stem cells and other types of cells, conduct research on the samples, perform genetic testing, and/or store the samples for future use.
Through this research, researchers hope to identify future treatments or even cures for Parkinson's disease.
Detailed description
Researchers at the New York Stem Cell Foundation (NYSCF) Research Institute study diverse diseases, conditions, and traits by creating stem cells from biological samples. These "pluripotent" stem cells can become any cell in the human body, including cells that may be difficult, invasive, or impossible to obtain directly.
Additionally, researchers perform genetic testing to learn more about DNA, a material in most cells that contains instructions for the body's development and functions (such as traits like eye color and risk of certain diseases). A piece of DNA that determines the specific role of a cell is called a "gene." If the instructions in a gene are abnormal, this can lead to disease.
Participation in the study involves: (1) completion of health questionnaires, (2) providing a skin and/or blood sample from which stem cells may be created, (3) collection of a saliva sample for genetic analysis, and (4) possible future follow-up to provide additional information or learn about other research studies.
This study is not a clinical trial.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Parkinson's Disease | – | Other: Biological Sample CollectionSubjects in this group will have a diagnosis of Parkinson's disease with or without a known GBA gene mutation. |
| Gaucher Disease | – | Other: Biological Sample CollectionSubjects in this group with have a diagnosis of Gaucher disease with or without a known GBA gene mutation. |
| Healthy Control | – | Other: Biological Sample CollectionSubjects in this group will serve as healthy controls. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Individuals with a diagnosis of one or more of the listed conditions. Individuals without a condition to serve as healthy controls (a comparison group for those with conditions).
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biological Sample CollectionAlso: Biological sample collection for biobanking and researchSkin biopsy (2-3mm) and/or blood (up to 50 mL); saliva; excess/leftover biospecimens that were (or will be) collected for other purposes (e.g., medical procedure) | Other | Gaucher Disease, Healthy Control, Parkinson's Disease |
Primary outcome
Establishment of a diverse bank of biological samples, stem cell lines, derivatives, and associated information.
Time frame: Baseline
Establish a robust disease signature using a cohort of PD and carefully matched control samples to screen a library of approximately 3,000 compounds to determine whether any compounds improve or reverse disease phenotypes.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2025.
- Individual participant dataSharing planned: No
Related trials for Parkinson Disease
Not yet recruitingPhase 1/2
STAR0602 With Nivolumab in Non Small Cell Lung Cancer and in Metastatic Castrate-Resistant Prostate Cancer, and STAR0602 Alone in Thymic Carcinoma
NSCLC, Non-Small Cell Lung Cancer
Enrolling by invitationObservational study
Early Molecular Biomarkers for Differentiating Parkinsonian Syndromes
PARKINSON DISEASE (Disorder), Atypical Parkinsonism
RecruitingPhase 3
A Clinical Study of Sacituzumab Tirumotecan (MK-2870) in Combination With Pembrolizumab (MK-3475) as First-line Maintenance Treatment of Cervical Cancer (MK-2870-036/TroFuse-036/GOG-3123/ENGOT-cx22)
Cervical Cancer
Active, not recruitingPhase 1/2
Clinical Safety and Efficacy of Umbilical Cord Mesenchymal Stromal Cells-derived Exosomes in Patients With Parkinson's Disease
Parkinson Disease (PD)
Active, not recruitingObservational study
Adaptive DBS to Offset STN Dysregulation During Exercise in PD
Parkinson Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laura Andres-Martin principal investigator
New York Stem Cell Foundation Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Parkinson Disease and Gaucher Disease, run by New York Stem Cell Foundation Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Clinical Research Office (212-927-1801, clinicalresearch@nyscf.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Establishment of a diverse bank of biological samples, stem cell lines, derivatives, and associated information. (time frame: Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Mar 2025.