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Recruiting
NCT05224778
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
Conditions: Congenital Myotonic Dystrophy, CDM
Sex: All
Ages: N/A – 59 Months
Healthy volunteers: No
Enrollment: 50
Sponsor: Virginia Commonwealth University
Location: University of California, Los Angeles Los Angeles California
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 Criteria
Inclusion Criteria:
* Age neonate to 3 years 11 months at enrollment.
* A diagnosis of CDM, which is defined as children having symptoms of myotonic dystrophy in the newborn period (\1,500).
* Guardian is willing and able to sign consent and follow study procedures
Exclusion Criteria:
* Any other non-DM1 illness that would interfere with the ability or results of the study in the opinion of the site investigator
* Significant trauma within one month
* Internal metal or devices (exclusion for DEXA component)
* History of bleeding disorder or platelet count \
Source: ClinicalTrials.gov (NCT05224778). StuddyBuddy aggregates publicly available trial information.
