Status:
RECRUITING
WiTNNess - TNNT1 Myopathy Natural History Study
Lead Sponsor:
Clinic for Special Children
Conditions:
TNNT1-associated Myopathy
Infantile-onset Nemaline Rod Myopathy
Eligibility:
All Genders
Brief 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 out...
Detailed Description
WiTNNess is an observational study that includes prospective and cross-sectional arms, both of which include people diagnosed with autosomal recessive TNNT1-associated muscle disease, commonly describ...
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.
Key Trial Info
Start Date :
September 23 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
June 1 2027
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT06374719
Start Date
September 23 2018
End Date
June 1 2027
Last Update
February 12 2025
Active Locations (1)
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1
Clinic for Special Children
Gordonville, Pennsylvania, United States, 17529