New Research Project - RDCRN and CMTA - Recruiting

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Working together with the Rare Diseases Clinical Research Network (RDCRN), the

CMTA invites you (or your child) to participate in a research project that will

develop a nation-wide registry for patients.

Why Join?

The RDCRN Patient Contact Registry is a method by which patients with rare

diseases can register themselves with the RDCRN in order to be contacted in the

future about clinical research opportunities and updates on the progress of the

research projects. The contact registry is anonymous and free of charge.

Information contained within this registry will be used for recruitment to

research studies directed at improving our knowledge and treatment of people

with CMT.

Patients who participate in this research will make it possible for scientists

to find new treatments, create new studies, and work for the improvement of all

our lives.

What do you have to do to join?

Register. After you have read and agreed to the Authorization, the Registry form

will appear on your screen. You will be asked for information such as your (or

your child's) type of CMT, name, address, birth date, place of birth, email

address, and other items relevant to your (or your child's) disorder.

https://rarediseasesnetwork.epi.usf.edu/rdnwebapp/registry/RDCRNRegistryConsent.\

aspx?OwnerId=3583 & DiseaseType=242

Concerned about your privacy?

Once you have entered and submitted this information online, the data will be

stored in a secure, computerized database. No personal identifying information

(such as your name, address, or telephone number) will be given to anyone

without your expressed approval.

How will I be notified of research studies and updates?

You will be notified via email when a study is recruiting or when there are

updates to research being conducted.

To find out about studies currently recruiting, please see below section.

RDCRN Studies Currently Recruiting

The following studies are now enrolling patients

6601: Natural History Evaluation of Charcot Marie Tooth Disease

This is a longitudinal study of individuals with CMT. Those participating in

this study will be invited to be re-evaluated every year. There is no cap on the

number of participants who can enroll - all people who meet the inclusion

criteria are encouraged to participate.

6602: Genetics of CMT - There are two aims of this study: finding genetic

modifiers for CMT1A, and finding new genes that cause CMT2.

6603: Development of CMT Peds Scale for Children with CMT

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.