NCT05224778 DMCRN-02-001: Assessing Pediatric Endpoints in DM1
| NCT ID | NCT05224778 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Virginia Commonwealth University |
| Condition | Congenital Myotonic Dystrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 50 participants |
| Start Date | 2022-08-24 |
| Primary Completion | 2026-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 50 participants in total. It began in 2022-08-24 with a primary completion date of 2026-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 (\<30 days), such as hypotonia, feeding or respiratory difficulty, requiring hospitalization to a ward or to the neonatal intensive care unit for more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. An expanded CTG repeat size in the child is considered greater than 200 repeats or E1-E4 classification (E1= 200-500, E2=500-1,000, E3=1,000-1,500, E4\>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 \<50,000 * History of reaction to local anesthetic
Contact & Investigator
Nicholas E. Johnson, MD
PRINCIPAL INVESTIGATOR
Virginia Commonwealth University
Frequently Asked Questions
Who can join the NCT05224778 clinical trial?
This trial is open to participants of all sexes, up to 59 Months, studying Congenital Myotonic Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05224778 currently recruiting?
Yes, NCT05224778 is actively recruiting participants. Contact the research team at ruby.langeslay@vcuhealth.org for enrollment information.
Where is the NCT05224778 trial being conducted?
This trial is being conducted at Los Angeles, United States, Fairway, United States, Rochester, United States, Richmond, United States and 1 additional location.
Who is sponsoring the NCT05224778 clinical trial?
NCT05224778 is sponsored by Virginia Commonwealth University. The principal investigator is Nicholas E. Johnson, MD at Virginia Commonwealth University. The trial plans to enroll 50 participants.