WiTNNess - TNNT1 Myopathy Natural History Study - NCT06374719

RECRUITING

NCT06374719

WiTNNess - TNNT1 Myopathy Natural History Study

Sponsor: Clinic for Special Children

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).

Official title: WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy

Key Details

Gender

All

Age Range

Any - Any

Study Type

OBSERVATIONAL

Enrollment

40

Start Date

2018-09-23

Completion Date

2027-06-01

Last Updated

2025-02-12

Healthy Volunteers

No

Conditions

TNNT1-associated Myopathy Infantile-onset Nemaline Rod Myopathy Myopathies, Nemaline Myopathy Myopathy, Rod Myopathy; Hereditary Amish Nemaline Myopathy Nemaline Myopathy 5 NEM5 Genetic Muscle Disease Recessive Hereditary Disorder (Autosomal)ANM

Locations (1)

Clinic for Special Children

Gordonville, Pennsylvania, United States

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