The Charcot-Marie-Tooth Association (CMTA), through its strategic alliance with the Inherited Neuropathy Consortium (INC), is proud to share this important opportunity for families living with Charcot-Marie-Tooth disease (CMT). This longitudinal study, led by INC, aims to refine the Charcot-Marie-Tooth Pediatric Scale (CMTPedS).
Information about how different forms of CMT affect children is not readily available because there are not good methods to measure impairment of children with CMT. The purpose of this project is to develop and test such a method and to then test this scale to ensure that it is an effective measurement of impairment of CMT in children, that the children tolerate it well, that different investigators using this method obtain similar results, and that changes over time with the scale allow measurement of progression of CMT in the children.
About the Study
This is a longitudinal study of 500 children (<21 yrs of age) with various forms of CMT, divided into CMT1, CMT2, and CMT4. Patients will be seen for a total of 4 times at six-month intervals. The evaluations will take place at one of the INC Rare Disease Clinical Research Centers (RDCRC) sites that are each a CMTA Center of Excellence and have specialized pediatric neurology expertise.
Who Can Participate?
This study is open to individuals who meet the following criteria:
Age: Children and teens under 21 years old.
Diagnosis: A known or probable diagnosis of CMT1, CMT2, or CMT4.
Participation is voluntary and non-invasive, offering families the opportunity to contribute to research that directly benefits the pediatric CMT community.
Participating Locations
Assessments will be conducted at the following CMTA Centers of Excellence:
- Cedars Sinai Medical Center
- Children’s Hospital at Westmead
- Children’s Hospital of Philadelphia
- Connecticut Children’s Medical Center
- National Hospital for Neurology and Neurosurgery
- Nemours Children’s Health System Orlando
- Stanford University
- University of Iowa
- University of Rochester
How to Get Involved
To learn more about this study and how to participate, visit INC’s Website.
CMTA-INC Alliance’s Role in Advancing Research
As the global leader in driving transformative progress in CMT research, CMTA, through its alliance with INC, is committed to advancing patient-centered research that delivers meaningful outcomes for individuals and families living with CMT. This study underscores the power of collaboration between researchers, clinicians, and the patient community.
Register with Patients as Partners in Research Today
Not yet registered with Patients as Partners in Research but wish to be informed about more opportunities like the INC Study 6603? Click the button below to register today!
Registering today ensures you will be the first to hear when INC announces new study locations and that you are updated on all the latest news and developments in CMT research. Registration is free and secure, and all who have CMT are invited to register.
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Published on: December 19, 2024