RecruitingNot Applicable

WiTNNess - TNNT1 Myopathy Natural History Study

United States40 participantsStarted 2018-09-23

Plain-language summary

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).

Who can participate

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Event-free survival

Timeframe: Day 1 up to 15 years

Trial details

NCT IDNCT06374719

SponsorClinic for Special Children

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2027-01-01

Contact for this trial

Erin Sweigert

7176879407 esweigert@clinicforspecialchildren.org

View on ClinicalTrials.gov More TNNT1-associated Myopathy trials