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Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study

NCT03550300 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 130

Last updated 2018-10-04

No results posted yet for this study

Summary

This cohort study (participants with CMT and control participants) has two parts (Part 1: CMT1A cohort; Part2: CMT1B, CMT2A and CMTX1 cohort) and is proposed to take place over 3 years across three sites. Participants with CMT aged 5-60 for potential enrolment in the trial will be identified through the existing inherited neuropathy clinics at each site and control participants will be identified among the unaffected relatives and carers of the participants with CMT. If they show interest in participating, they will be given the relevant Patient Information Sheets, Written Consent forms and/or Assent forms. Half of the participants will be recruited at the UK sites (NHNN and GOSH) and the other half at the US collaborating site. Each participant will be invited to two separate research visits (12 months apart) for which travel expenses (return journey) will be reimbursed. Each research visit is expected to last approximately 3 hours and during it, relevant detailed clinical data will be collected (CMTPedS for participants with CMT aged 5-20, CMTESv2-R for participants with CMT over the age of 10, CMT-HI for participants with CMT over the age of 16) and the participant will also undergo an MRI scan (up to 45 minutes) of the lower limbs (feet and calves or calves and thighs). Two separate neuromuscular MRI protocols with specific sequences will be used for the scans of foot and calf muscles and scans of calf and thigh muscles. Blood samples for plasma NEFL levels will be optional at both research visits for the participants at the UK trial sites; plasma NEFL levels will be processed according to our previously published protocol

Conditions

  • Charcot-Marie-Tooth Disease, Type IA
  • Charcot-Marie-Tooth Disease Type 2A
  • Charcot-Marie-Tooth Disease, Type IB
  • Charcot-Marie-Tooth Disease, Type X

Sponsors & Collaborators

  • University of Iowa

    collaborator OTHER

  • University College, London

    lead OTHER

Eligibility

Min Age
5 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-09-11
Primary Completion
2021-07-01
Completion
2021-10-01

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03550300 on ClinicalTrials.gov