RECRUITING

WiTNNess - TNNT1 Myopathy Natural History Study

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy

Quick Facts

Study Start:2018-09-23
Study Completion:2027-06-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06374719

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Diagnosed with biallelic pathogenic variants of TNNT1
  2. * Infantile-onset or childhood-onset proximal weakness without confounding medical conditions that could effect muscle health.
  1. * 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.

Contacts and Locations

Study Contact

Joelle Williamson, MPH
CONTACT
7176879407
jwilliamson@clinicforspecialchildren.org

Principal Investigator

Kevin Strauss, MD
PRINCIPAL_INVESTIGATOR
Clinic for Special Children

Study Locations (Sites)

Clinic for Special Children
Strasburg, Pennsylvania, 17579
United States

Collaborators and Investigators

Sponsor: Clinic for Special Children

  • Kevin Strauss, MD, PRINCIPAL_INVESTIGATOR, Clinic for Special Children

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-09-23
Study Completion Date2027-06-01

Study Record Updates

Study Start Date2018-09-23
Study Completion Date2027-06-01

Terms related to this study

Additional Relevant MeSH Terms

  • 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