Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
NCT07700225
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DM1
Richmond, Virginia, United States
View Trial DetailsNCT Number: NCT05020002
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample.
Interested in participating?
Request Info5 year and older
All sexes
Observational
Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States
Healthy volunteers accepted: Yes
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Time frame: 4 years
The extracellular RNA biomarkers in the muscular dystrophy groups will be evaluated and compared with the extracellular RNA content in control groups. Statistical analysis will be used to evaluate the sensitivity and specificity of these markers as measurements of disease activity and severity.
Contact information is provided by the study sponsor or research team.
Massachusetts General Hospital
Other
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