Muscular Dystrophy Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Congenital Myotonic Dystrophy (CDM) Group:

• Children aged 5 to 17 years and 11 months at the time of joining (the lower age limit may not apply for children who completed a previous study called ASPIRE-DM1, and the upper age limit may not apply for those who took part in the TREAT-CDM study)
• Children diagnosed with CDM, meaning they had symptoms of myotonic dystrophy as a newborn (within the first 30 days of life) — such as poor muscle tone, feeding difficulties, or breathing problems — that required a hospital stay of more than 72 hours, confirmed by a genetic test showing an expanded CTG repeat in the DMPK gene in the child or their mother
• Signed, voluntary consent must be provided before any study activities begin

Childhood Myotonic Dystrophy (ChDM) Group:

• Children aged 3 to 17 years and 11 months at the time of joining (the upper age limit may not apply for those who previously took part in the TREAT-CDM study)
• Children diagnosed with ChDM, meaning they developed thinking or learning difficulties, muscle weakness, or muscle stiffness (myotonia) after age 1 and before age 10, confirmed by a genetic test showing an expanded CTG repeat in the DMPK gene in the child or their mother
• Signed, voluntary consent must be provided before any study activities begin

Who may not be able to join:

• Children with a serious illness unrelated to myotonic dystrophy that could affect the safety of testing or make results difficult to interpret, as assessed by the trial doctor
• Children who have experienced a significant injury in the past month
• Children who have metal implants or internal medical devices (this applies specifically to the bone density scan component of the trial)
• Children taking blood-thinning medications such as warfarin or direct oral anticoagulants (for example, dabigatran), due to an increased bleeding risk associated with the muscle biopsy procedure
• Children with a platelet count below 50,000 (platelets are blood cells that help with clotting — a low level increases bleeding risk)
• Children with a known history of a bleeding disorder
• Children currently participating in another clinical trial that involves an investigational (unapproved) treatment
• Children with a known history of a bad reaction to lidocaine, a local anaesthetic (this applies to those taking part in the muscle biopsy portion of the study)

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