Actively Recruiting
WiTNNess - TNNT1 Myopathy Natural History Study
Led by Clinic for Special Children · Updated on 2025-02-12
40
Participants Needed
1
Research Sites
453 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
WiTNNess - TNNT1 Myopathy Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with biallelic pathogenic variants of TNNT1
- Infantile-onset or childhood-onset proximal weakness without confounding medical conditions that could affect muscle health
You will not qualify if you...
- Presence of another known or suspected medical condition (genetic or acquired) that could alter the natural disease course or interfere with study procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Clinic for Special Children
Gordonville, Pennsylvania, United States, 17529
Actively Recruiting
Research Team
E
Erin Sweigert
CONTACT
J
Joelle Williamson, MPH
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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