Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5+
SponsorNew York Stem Cell Foundation Research Institute
About this trial
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Charcot-Marie-Tooth 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 Charcot-Marie-Tooth disease.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
Age 5 years or older.
Diagnosis of Charcot-Marie-Tooth (CMT) disease or healthy controls.
Adults must provide written informed consent unless physical limitations preclude signing.
Minors undergoing skin collection for research purposes must have a CMT diagnosis and must participate with parent/guardian consent.
Disqualifiers
Wards of the state.
For prospective skin samples: history of keloid formation, coagulation disorder, allergy to the anesthetic, or anticoagulation use that precludes sample collection.
For prospective blood samples: coagulation disorder or other medical conditions that increase the risks associated with blood collection.
For all prospective sample collections: Subjects who refuse to adhere to NYSCF's and/or a collaborating site's safety protocols will be excluded. Subjects with an AIDS diagnosis and CD4 count of less than 200 cells per microliter of blood will be excluded due to increased risk of infection.
Trial population
Individuals with a Charcot-Marie-Tooth (CMT) diagnosis. Individuals without a condition to serve as healthy controls (a comparison group for CMT subjects).
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Biological Sample Collection
Other interventionSkin biopsy (2-3mm) and/or blood (up to 50mL); saliva; excess/leftover biospecimens that were (or will be) collected for other purposes (e.g., medical procedure).
Treatment groups
50 Participants
are divided into 2 treatment groupsGroup A: Charcot-Marie-Tooth Disease1 intervention
Group B: Healthy Control1 intervention
Trial outcomes
Primary outcomes
1
Biobank
Establishment of a bank of stem cell lines and associated information to advance understanding of the biology, etiology, manifestations, progression, risk factors, genetic underpinnings, and treatment of Charcot-Marie-Tooth diseases.
Time frame
BaselineSponsors and contacts
Click on the lead sponsor to view all of their trials.
New York Stem Cell Foundation Research Institute
Lead sponsor
Charcot-Marie-Tooth Association
Collaborator
Source ClinicalTrials.gov