WiTNNess - TNNT1 Myopathy Natural History Study
Recruiting
Observational Study
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
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 40 (estimated)
- Sponsor
- Clinic for Special Children · Other
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About This Trial
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 (pro…
Trial Locations
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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 …
Contacts