Muscular Dystrophy Trial, Recruiting NCT05224778 Sponsor: Virginia Commonwealth University Condition: Muscular Dystrophy
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Muscular Dystrophy Trial, Recruiting

NCT05224778
Recruiting Not Applicable

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • The child must be between newborn age and 3 years and 11 months old at the time of enrollment.
  • The child must have been diagnosed with Congenital Myotonic Dystrophy (CDM), meaning they showed symptoms of myotonic dystrophy as a newborn (within the first 30 days of life) — such as poor muscle tone, difficulty feeding, or breathing problems — that required a hospital stay of more than 72 hours.
  • The diagnosis must be confirmed by a genetic test showing a specific gene change (an expanded CTG repeat in the DMPK gene) in either the child or the mother, with the child having more than 200 repeats if tested.
  • The child's parent or legal guardian must be willing and able to sign a consent form and follow the study procedures.

Who may not be able to join:

  • The child has another serious illness (not related to myotonic dystrophy) that could affect their ability to participate or affect the study results, in the opinion of the study doctor.
  • The child has experienced a significant injury within the past month.
  • The child has metal implants or internal medical devices in their body (this specifically affects one part of the study involving a bone density scan called a DEXA scan — confirm with trial site).
  • The child has a history of a bleeding disorder or has a very low platelet count (below 50,000), which affects blood clotting.
  • The child has previously had a reaction to local anesthetic (numbing medication used on the skin or a small area of the body).

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 27 July 2026
This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Nicholas E. Johnson, MD, Virginia Commonwealth University

Phone: 804-828-8481

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Not Applicable
Registry
ClinicalTrials.gov
Start date
24 August 2022
Est. completion
1 October 2026

Where this trial is recruiting

🇮🇹 Italy 🇺🇸 United States

Primary endpoints

To evaluate motor milestone attainment in individuals with CDM and ChDM and compare to typically developing children

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov