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PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study)

NCT02831504 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 213

Last updated 2021-04-13

No results posted yet for this study

Summary

PhenoDM1 will use patient reported outcomes to assess levels of pain, fatigue and quality of life in this cohort. Clinical and functional outcomes will look at muscle wasting and levels of myotonia. DNA, RNA, serum and CSF samples will be taken from all patients so that additional genetic and molecular biomarker analysis can be carried out. A subset of patients will undergo detailed sleep studies along with skeletal muscle MRI of the lower limbs. This study will complement the work of other groups currently looking at myotonic dystrophy type 1 using the same outcomes and measures where possible.

Conditions

Sponsors & Collaborators

  • Newcastle-upon-Tyne Hospitals NHS Trust

    lead OTHER

Principal Investigators

  • Hanns Lochmuller, MD, FAAN · University of Newcastle Upon-Tyne

  • Chris Turner, FRCP, PhD · National Hospital for Neurology and Neurosurgery

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-08-31
Primary Completion
2018-10-31
Completion
2018-10-31

Countries

  • United Kingdom

Study Locations

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Entities

Diseases

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 NCT02831504 on ClinicalTrials.gov