RecruitingNCT05224778

DMCRN-02-001: Assessing Pediatric Endpoints in DM1

Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)


Sponsor

Virginia Commonwealth University

Enrollment

50 participants

Start Date

Aug 24, 2022

Study Type

OBSERVATIONAL

Conditions

Summary

The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.


Eligibility

Max Age: 59 Months

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a new treatment for people with cdm and congenital myotonic dystrophy. The study is currently recruiting participants at 5 locations. People eligible for this study include up to age 59 Months.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(5)

University of California, Los Angeles

Los Angeles, California, United States

University of Kansas Medical Center

Fairway, Kansas, United States

University of Rochester Medical Center

Rochester, New York, United States

Virginia Commonwealth University

Richmond, Virginia, United States

Centro Clinico NeMO

Milan, Italy

View Full Details on ClinicalTrials.gov

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NCT05224778


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