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WiTNNess - TNNT1 Myopathy Natural History Study
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
Who can join
All ages · All sexes
Full eligibility criteria
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.
About the study
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).
Sponsor: Clinic for Special Children · Participants: 40 · Started: Sep 23, 2018
Contact the study team
- Justin Hersh · Phone: 7176879407
- Joelle Williamson, MPH · Phone: 7176879407
Official record on ClinicalTrials.gov — NCT06374719
Locations in the U.S.
| Pennsylvania | Clinic for Special Children, Gordonville |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.