NMD Natural History Studies

Through approved access to completed and ongoing natural histories in Duchenne, Becker, and facioscapulohumeral muscular dystrophy 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.

TRiNDS personnel 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 has 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 study chair 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

CINRG and TRiNDS have two other ongoing neuromuscular natural history studies: The Becker natural history study which was modeled of the CINRG DNHS. It has successfully enrolled over 80 Becker patients from around the world. It continues to collect annual data to prospectively collect longitudinal data in the following domains:

  • Medical events
  • Anthropometrics
  • Joint range of motion
  • Strength and function
  • Pulmonary function
  • Cardiac function
  • Patient reported outcomes.

The infantile facioscapulohumeral (FSHD) natural history study which also closed enrollment with over 50 FSHD patients and continues to follow many of them in longitudinal follow-up visits. Its focus was to learn more about patients with early onset FSHD and establish standardized functional strength testing protocol. It also plans to describe the longitudinal changes in the clinical phenotypes of infantile FSHD.

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