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NCT05224778 · ClinicalTrials.gov registry record
DMCRN-02-001: Assessing Pediatric Endpoints in DM1
A clinical trial of Congenital Myotonic Dystrophy and CDM, sponsored by Virginia Commonwealth University.
- Recruiting
- Registry status
- 50
- Enrollment target
- 5
- Study locations
NCT05224778: Recruiting study of Congenital Myotonic Dystrophy and CDM, sponsored by Virginia Commonwealth University.
NCT05224778 is a study of Congenital Myotonic Dystrophy and CDM that is actively recruiting participants, run by Virginia Commonwealth University. The registered enrollment target is 50 participants, below the 1,694-participant average among 4 other Congenital Myotonic Dystrophy trials with a reported enrollment target (97% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05224778, a study of Congenital Myotonic Dystrophy and CDM, is actively recruiting participants, sponsored by Virginia Commonwealth University.
- RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 5
- Study locations
Study 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.
Primary Outcome
Milestone Assessment using Peabody definitions: This survey would ask parents to assess the age of motor milestones in days, months of infant age. Birth history, including prematurity, ventilatory status, and feeding problems would also be collected on the CRF. Feeding and ventilatory support, as well as height and weight will be collected at each study visit.
Conditions Studied
Study Locations (5)
California
- University of California, Los Angeles - Los Angeles
Kansas
- University of Kansas Medical Center - Fairway
New York
- University of Rochester Medical Center - Rochester
Virginia
- Virginia Commonwealth University - Richmond
Other
- Centro Clinico NeMO - Milan
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2022-08-24 |
| Est. Completion | 2026-12 |
What NCT05224778 shows while recruiting
NCT05224778 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target, below the 1,694-participant average among 4 other Congenital Myotonic Dystrophy trials with a reported enrollment target (97% lower).
The record links to 2 conditions, with Congenital Myotonic Dystrophy appearing as the primary indexed condition, and to 0 interventions.
NCT05224778 lists 5 locations in 5 states (California, Kansas, New York).
Frequently Asked Questions
What is clinical trial NCT05224778 about?
NCT05224778 is a clinical study titled "DMCRN-02-001: Assessing Pediatric Endpoints in DM1". 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.
What is the current status of trial NCT05224778?
This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2022-08-24. Estimated completion is 2026-12.
What conditions does trial NCT05224778 study?
This clinical trial studies the following conditions: Congenital Myotonic Dystrophy, CDM.
Who is sponsoring clinical trial NCT05224778?
This trial is sponsored by Virginia Commonwealth University, which has 558 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05224778 being conducted?
This trial has 5 study locations across California, Kansas, New York, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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