WiTNNess - TNNT1 Myopathy Natural History Study

Launched by CLINIC FOR SPECIAL CHILDREN · Apr 17, 2024

Keywords

ClinConnect Summary

The WiTNNess study is a clinical trial looking at a specific muscle disease caused by changes in the TNNT1 gene, which can lead to various types of myopathy, including conditions that affect infants and children. The main goal of this study is to understand how this muscle disease progresses over time and to identify important measures that can help in future clinical trials. This study is open to both children and adults from around the world, and participants can choose to share their health information just once or continue to provide updates every few months.

To be eligible for this study, participants need to have been diagnosed with specific genetic changes in the TNNT1 gene that cause muscle weakness starting in infancy or childhood. However, they should not have other medical conditions that could affect their muscle health or interfere with the study. If you or someone you know meets these criteria, participating in the WiTNNess study could help advance our understanding of this condition and contribute to future treatments.

Gender

ALL

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.