Texas Children’s and Baylor College participate in a multicenter study to evaluate readiness for future STXBP1 disorders clinical trials

Texas Children’s Hospital and Baylor College of Medicine clinicians and research investigators have recently launched the STXBP1 Clinical Trial Ready (STARR), a new study focused on developing clinical trial readiness for STXBP1-related disorders, an epileptic and neurodevelopmental condition caused by changes in the STXBP1 gene. STXBP1-related disorders are one of the most common genetic causes of childhood epilepsies.

Child neurologist and neuroscientist, Dr. Hsiao Tuan Chao, an assistant professor at Baylor College, a faculty member with the Cain Pediatric Neurology Research Foundation Laboratories at the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital, and an investigator at the McNair Medical Institute with The Robert and Janice McNair Foundation is the lead investigator at this site, which is one of the four inaugural sites in the US currently conducting this study. The multi-center STARR study is funded by the STXBP1 Foundation and led by Dr. Ingo Helbig at Children’s Hospital of Philadelphia. Dr. Scott Demarest at Children’s Hospital Colorado and Dr. Zachary Grinspan at Weill Cornell Medicine are lead investigators for the STARR study at their respective sites.

Like any disease, clinical trials are the key to developing and evaluating new treatments for rare diseases. However, often there is not enough information about the symptoms and biology of rare diseases to design clinical trials. Clinical readiness trials like STARR are undertaken to address some of the obstacles, including gaps in the knowledge about a disease’s natural history, suitable biomarkers, and clinical outcome measures.

In 2020, researchers led by Baylor College and Duncan NRI investigator, Dr. Mingshan Xue, who holds the title of Caroline DeLuca scholar, developed new rodent models of STXBP1 encephalopathy to better understand this disorder, found new therapeutic strategies, and lay the groundwork for clinical studies. It is indeed fitting that Texas Children’s Hospital is now one of the sites partnering with the STXBP1 Foundation for STARR.

“Our team is excited to be a part of this important study with our colleagues at Children’s Hospital of Philadelphia, Children’s Hospital Colorado, and Weill Cornell Medicine. This study will provide critical information needed for future clinical trials on STXBP1-related disorders,” Dr. Chao said. “We are thankful to the STXBP1 Foundation for providing funding to each of the sites for the initial year of the study, which was raised by the STXBP1 family community, through a transformational campaign, FLOURISH, led by the Finn Beaubien family.”

The STARR study at Texas Children’s Hospital is currently recruiting participants across all age groups. Families interested in participating are invited to complete the STARR study eligibility survey, https://redcap.link/stxbp1survey. For more information, contact chao-lab@bcm.edu.

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Rajalaxmi Natarajan, PhD