Planning for Genetic Variability

February 5, 2020

Planning for Genetic Variability

At TRiNDS, we focus on the unique needs for companies conducting clinical trials in rare disease populations. Clinical trials are always challenging, but rare neuromuscular disorders add complexity to study design and execution.

Many neuromuscular disorders have genetic causes, such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). Unlike other disorders, an individual’s diagnostic genotype can vary widely, affecting the severity of symptoms, standards of clinical care, and natural history of disease progression.

It is important to design your clinical trial protocols to minimize variability in your study population. For many studies, that means confirming participants’ genotypes during study screening. Whether you’re testing an investigational product that acts at the DNA or RNA level, or you’re excluding certain diagnostic subtypes, having expert help is critical.

TRiNDS offers central diagnostic confirmation services to research projects and all phases of clinical trials. Our licensed genetic counselors combine their clinical training and research expertise to provide consistent review and analysis of test results. After informed consent, sites submit de-identified reports to our team for review. Our genetic counselors interpret the results and inform the site and the medical monitoring team of the participant’s eligibility for the study.

Contact us to request a quote for central diagnostic confirmation services.