Genetic Study for Charcot-Marie-Tooth Disease
(INC-6602 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to understand the genetics behind Charcot Marie Tooth disease (CMT). Researchers seek new genes that might cause CMT and others that might influence symptom severity. Individuals with a specific genetic marker (PMP22 duplication) or a family history of CMT1A could be suitable candidates. Additionally, family members of CMT patients, regardless of symptoms, might also qualify to join. The trial involves no treatment, focusing solely on genetic research. As an unphased study, it offers participants the chance to contribute to groundbreaking genetic research that could lead to future breakthroughs in understanding CMT.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to uncover genetic factors that modify the severity of Charcot Marie Tooth disease type 1A (CMT1A) and identify new causes of Charcot Marie Tooth disease type 2 (CMT2). This approach is unique because, instead of focusing on treating symptoms, it seeks to understand the underlying genetic influences that could lead to more personalized and effective treatment strategies in the future. By identifying genetic modifiers and new genetic causes, scientists hope to pave the way for targeted therapies that could offer more precise and potentially transformative options for patients with CMT.
Who Is on the Research Team?
Michael E Shy, MD
Principal Investigator
University of Iowa
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Sample Collection
Blood samples are collected from participants for genetic analysis
Genetic Analysis
Blood samples are processed through exome sequencing to identify genetic modifiers and new causes of CMT
Follow-up
Participants are informed of the genetic analysis results and any potential findings
What Are the Treatments Tested in This Trial?
Interventions
- None
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of Iowa
Lead Sponsor
Children's National Research Institute
Collaborator
Seattle Children's Hospital
Collaborator
Massachusetts General Hospital
Collaborator
University of Michigan
Collaborator
St. Jude Children's Research Hospital
Collaborator
Connecticut Children's Medical Center
Collaborator
The Hospital for Sick Children
Collaborator
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Muscular Dystrophy Association
Collaborator
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