NMD Natural History Studies

Through approved access to completed natural histories in Duchenne, Becker, and facioscapulohumeral muscular dystrophy (FSHD) TRiNDS can works with customers on their clinical trial programs. Summary data can assist with trial planning and sample size calculations, study endpoint selection, individual patient matching analyses, and more.

Several members of the TRiNDS team and board have played central roles in the design and management of the CINRG Duchenne Natural History Study (DNHS). This study is the largest and longest Duchenne natural history study to date and is providing critical information to the Duchenne community. The study enrolled over 400 Duchenne boys and young men and followed their disease course for over 5 years and in some cases, close to 10 years. Analyses of the CINRG DNHS study cohort have generated data that responds to many of the outstanding questions that have been raised by the field in general. Key publications have provided information on variation in disease progression due to underlying genetic characteristics, contemporary use of steroids, adherence to recommended care standards, and identification of biomarker panels to evaluate disease progress and therapeutic efficacy in the clinic and research settings.

By obtaining approval from the respective study chairs of our completed natural history studies in Duchenne, Becker, or FSHD and from the CINRG executive committee, TRiNDS can help you with your clinical trial needs. We have worked with clients on:

  • Study planning consulting
  • Sample size calculation support
  • Assistance with endpoint selection
  • Control data matching analyses
  • And many other projects

Learn more about these completed observational studies by visiting the CINRG website or by visiting clinicaltrials.gov.