WiTNNess - TNNT1 Myopathy Natural History Study

Part of paid clinical trials in Gordonville, Pennsylvania.

Sponsor
Clinic for Special Children
Study ID
NCT06374719
Status
Recruiting
Apply to this trial

Conditions

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

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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).

Key Dates

Start date
Sep 23, 2018
Status verified
Feb 2025
Primary completion
Jan 1, 2027
Completion
Jun 1, 2027

Study Design

Enrollment
40 participants (estimated)

Arms

  • Arm: Prospective
    Participants in the prospective cohort are enrolled during early infancy, shortly after diagnosis, and followed longitudinally with repeated assessments until reaching the primary study outcome.
  • Arm: Cross-Sectional
    Participants in the cross-sectional cohort enroll at any time after diagnosis and all study data are collected at a single time point.

Primary Outcome Measure

Event-free survival [ Time Frame: Day 1 up to 15 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Clinic for Special ChildrenGordonvillePennsylvania17529
Erin Sweigert
[email protected]
717-687-9407
Joelle Wiliamson, MPH
[email protected]
7176879407
Kevin Strauss, MD (PRINCIPAL_INVESTIGATOR)

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