Team Leader:

Ingo Helbig, MD

Ingo Helbig, MD

Biomarker Leadership Clinical Outcomes Assessment Leadership NHS Leadership Leadership Biomarker NHS

What are clinical outcomes for clinical trial studies?

Clinical outcomes are the predefined and measurable endpoints or results of a clinical trial that researchers use to assess whether a new intervention (a drug, medical device, vaccine, or therapy) is safe, effective, has limited side effects, and provides meaningful benefits to patients. Clinical trial outcomes are carefully selected to answer specific research questions and to guide regulatory decisions.

For the STXBP1 and SYNGAP1 natural history studies, we selected and standardized tests to cover as many aspects of these disorders as possible, thereby providing us with a highly accurate clinical picture of each disorder. The clinical outcomes our NHS measures include seizure, cognitive and social development, behavior, adaptive functioning, fine and gross motor function, language and communication, autism features, gastrointestinal function, and sleep patterns. Knowing the most difficult symptoms experienced in these conditions can help scientists know where the most help is needed for people with genetic conditions, and which are the most pertinent measures to focus on in clinical trials.