STXBP1 Foundation Announces STARR Study for Clinical Trial Readiness
Study Focuses on Clinical Trial Readiness for STXBP1-Related Disorders
Westminster, CO — July 22, 2023 — At the STXBP1 Summit+ conference this weekend, the STXBP1 Foundation is announcing STARR, a new multi-year study focused on developing clinical trial readiness for STXBP1-related disorders. STXBP1 disorder is a rare epileptic and neurodevelopmental condition caused by changes in the STXBP1 gene. STXBP1 disorder is one of the most common causes of genetic epilepsies, with an estimated incidence rate of 1:30,000.
With a growing focus on therapeutic development for STXBP1 disorder in multiple academic research groups and biopharma companies, the STXBP1 Foundation prioritized development of the study to prepare for future clinical trials. The STXBP1 Foundation has provided funding to each of the sites for the initial year of the study. This initial year funding was raised by the STXBP1 patient community, through a transformational campaign, FLOURISH, led by the Finn Beaubien family.
“The STARR study will generate important insights to inform clinical trial design,” commented Ingo Helbig, MD, Co-Director of the Center for Epilepsy and Neurodevelopmental Disorders (ENDD) at Penn Medicine and Children’s Hospital of Philadelphia. “The study will follow participants over time to get a comprehensive understanding of their development, and evaluate measures that could be used in clinical trials for STXBP1-related disorders.”
The STARR study will be conducted initially at four clinical centers in the United States: Children’s Hospital of Philadelphia, Children’s Hospital Colorado, Baylor College of Medicine / Texas Children’s Hospital, and Weill-Cornell in New York. Ingo Helbig, MD is the multi-site Principal Investigator for the study. James Goss, PhD, Scientific Director of the STXBP1 Foundation, and Michael Boland, PhD from University of Pennsylvania serve as the administrative core leads.
“We are elated to launch this important study which will help our community prepare for future clinical trials,” said Charlene Son Rigby, President and Co-Founder of the STXBP1 Foundation. “We are also grateful to be able to partner with our long-time research collaborators at Children’s Hospital of Philadelphia on the STARR study.”
The STARR study has launched this month, and is currently recruiting participants across all age groups. Contact research@stxbp1disorders.org for more information.
The STARR study complements and is harmonized with the protocol of the STXBP1 natural history study being launched by the European STXBP1 Consortium (ESCO) in Europe.
The STARR study is being announced at the 5th annual STXBP1 Summit+ Family Meeting, being held on July 22-23 at the Westin Westminster in Westminster, CO. The STXBP1 Summit+ conference will bring researchers, clinicians, and families together to review and understand the latest work on STXBP1-related disorders. Families will also have networking and social opportunities.
PDF version of news release here.
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STXBP1 Foundation is dedicated to finding a cure for STXBP1-Related Disorders while improving the lives of our patients and families. Founded in 2017, STXBP1 Foundation is a parent-led advocacy organization. STXBP1 Disorder is a rare epileptic and neurodevelopmental disorder caused by changes in the STXBP1 gene. With an incidence of approximately 1 in 30,000 live births, STXBP1 Disorder is one of the most common genetic causes of epilepsy. For more information, contact info@stxbp1disorders.org and visit https://www.stxbp1disorders.org/
