Actively Recruiting
Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking
Led by New York Stem Cell Foundation Research Institute · Updated on 2025-03-03
50
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
N
New York Stem Cell Foundation Research Institute
Lead Sponsor
C
Charcot-Marie-Tooth Association
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers at the New York Stem Cell Foundation Research Institute are studying Charcot-Marie-Tooth disease by collecting biological samples from people with the disease as well as from healthy volunteers. The goal is to create stem cells from these samples, perform genetic testing, and store samples for future research. This work aims to accelerate the development of new treatments or potential cures for Charcot-Marie-Tooth disease. Participants provide skin biopsies, blood samples, saliva samples, or leftover biological samples collected for other purposes. These samples are used to generate induced pluripotent stem cells (iPSCs) which can develop into any cell type. This is an observational study involving people diagnosed with Charcot-Marie-Tooth disease and healthy controls. During the study, participants complete health questionnaires and provide biological samples for research. Genetic analysis and biobanking of samples are key components. There may be future follow-up to collect additional information or participate in related research. The main outcome is the establishment of a biobank at baseline. This study is not a clinical trial and participation may continue as long as needed for research purposes.
CONDITIONS
Brief Title
Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 5 years or older
- Diagnosis of Charcot-Marie-Tooth (CMT) disease or healthy control status
- Adults must provide written informed consent unless unable to sign
- Minors with CMT must participate with parent or guardian consent for skin sample collection
- Minors with CMT or healthy controls may participate with parent or guardian consent for blood or saliva collection or transfer of biological samples collected outside the study
You will not qualify if you...
- Wards of the state
- For skin samples: history of keloid formation, bleeding disorders, allergy to anesthetics, or anticoagulation use that prevents sample collection
- For blood samples: bleeding disorders or other medical conditions increasing risks of blood collection
- Refusal to follow safety protocols of NYSCF or collaborating sites
- AIDS diagnosis with CD4 count below 200 cells per microliter of blood due to infection risk
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single day
Participants provide biological samples including skin biopsy, blood, and saliva for stem cell creation and genetic analysis.
1 visit (in-person)
Duration - Ongoing
Participants may be contacted for future follow-up to provide additional information or learn about other research studies.
Follow-up visits as needed
Trial Site Locations
Total: 1 location
1
New York Stem Cell Foundation Research Institute
New York, New York, United States, 10019
Actively Recruiting
Research Team
C
Clinical Research Office
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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