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WiTNNess - TNNT1 Myopathy Natural History Study

NCT06374719 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 40

Last updated 2025-02-12

No results posted yet for this study

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).

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

Sponsors & Collaborators

  • Clinic for Special Children

    lead OTHER

Principal Investigators

  • Kevin Strauss, MD · Clinic for Special Children

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-09-23
Primary Completion
2027-01-01
Completion
2027-06-01

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06374719 on ClinicalTrials.gov