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WiTNNess - TNNT1 Myopathy Natural History Study

Recruitingn/aAges any - anyAllClinic for Special Children
NCT06374719 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim of the study is to specify meaningful outcome measures for future clinical trials. WiTNNess is open to children and adults worldwide. Participants can choose to include their information once (cross-sectional cohort) or every few months (prospective cohort).

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📋 Eligibility rules (as published)
Inclusion Criteria: * Diagnosed with biallelic pathogenic variants of TNNT1 * Infantile-onset or childhood-onset proximal weakness without confounding medical conditions that could effect muscle health. Exclusion Criteria: * Another known or suspected medical condition (genetic or acquired) that could potentially alter the natural disease course or otherwise interfere with completion of study procedures.
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What it tests

  • Not listed
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Where

1 site
Clinic for Special Children
Gordonville, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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