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NCT03059264 · ClinicalTrials.gov registry record
Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy
A clinical trial, sponsored by Virginia Commonwealth University.
- Completed
- Registry status
- 100
- Enrollment target
NCT03059264 is a clinical trial that has completed, run by Virginia Commonwealth University. The registered enrollment target is 100 participants.
The verdict
NCT03059264 has completed, sponsored by Virginia Commonwealth University.
- COMPLETED
- Registry status
- 100 participants
- Enrollment target
Study Summary
Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene. CDM occurs when the CTGn increases between the adult myotonic dystrophy type-1 (DM1) parent and the child. Children with CDM present at birth with respiratory insufficiency, talipes equinovarus, feeding difficulties and hypotonia. There is a 30% mortality rate in the first year of life. As children grow, they are at risk for intellectual impairment, autistic features, gastrointestinal symptoms, and motor delay. The investigators will enroll children with CDM between ages 0-15 with visits at baseline and one year to evaluate appropriate physical functional outcomes, cognitive function and quality of life over time. Functional outcome measures will be correlated with potential biomarkers in the children. Completion of these specific aims will extend the understanding of disease progression in CDM and will provide the requisite information for successful therapeutic trials in children with DM.
Interventions
- OTHER Natural history
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 100 participants |
| Start Date | 2016-12-14 |
| Est. Completion | 2025-01-27 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03059264
The ClinicalTrials.gov registry entry for NCT03059264 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 100 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Virginia Commonwealth University, which has 558 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Natural history is the first listed.
NCT03059264 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03059264 about?
NCT03059264 is a clinical study titled "Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy". Congenital Myotonic Dystrophy (CDM) is a multi-systemic, dominantly inherited disorder caused by a trinucleotide repeat expansion (CTGn) in the DMPK gene. CDM occurs when the CTGn increases between the adult myotonic dystrophy type-1 (DM1) parent and the child. Children with CDM present at birth wit...
What is the current status of trial NCT03059264?
This trial is currently completed. The enrollment target is 100 participants. The study started on 2016-12-14. Estimated completion is 2025-01-27.
What interventions are being tested in trial NCT03059264?
The interventions under investigation include: Natural history (OTHER).
Who is sponsoring clinical trial NCT03059264?
This trial is sponsored by Virginia Commonwealth University, which has 558 total clinical trials registered on ClinicalTrials.gov.
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