What is STARR?
The STARR study is a prospective, multi-site natural history study for STXBP1-Related Disorders (STXBP1-RD). The purpose of this study is to gain a better understanding of how STXBP1-RD progresses over time and develop a standardized set of clinic assessments and biomarkers for STXBP1-RD patients. This study is an important step in preparing our community for upcoming clinical trials for new drugs and therapeutics. The initial year of funding for the STARR study was funded through a transformational campaign by the STXBP1 patient community, FLOURISH, led by the Finn Beaubien family, and by the ENDD Center at Penn Medicine and Children's Hospital of Philadelphia.
As a natural history study, this is an observational study and not an interventional study. That means that patients will be followed over a period of time during which they will continue to receive their current medications and supportive care such as physical and occupational therapies; however, they will not be treated with any new investigational drugs or therapeutics (that is, drugs that are not yet approved by the FDA).
Watch Informational Webinar Recording on STARR
Listen to the recorded information webinar on STARR with James Goss, PhD, Foundation Scientific Director, and Jackie Steinberg, MPH, BSN, from our board who is leading our natural history study support team.
How is STARR Different than Other Studies?
You may have been, or currently are, taking part in other STXBP1 studies such as Ciitizens, Simons Searchlight, or RARE-X and wondering what’s different about the STARR study and why you should enroll. Those studies collect STXBP1-related data mostly through past medical records or by surveys. They have been, and continue to be, very important as first steps in collecting natural history data for STXBP1, and you should participate in these studies if you can. However, because of the type of data they collect, there are limitations on how it can be used to advance new drug approvals by the FDA. The STARR study is a prospective study, meaning that it is designed to follow participants forward in time over the next 2-5 years. Prospective studies allow for a greater degree of control over the data that is collected and are less prone to scientific bias, thus they can more easily be used to support clinical drug trials for new therapeutics.
Importantly, the STARR study represents an opportunity to collect more information about STXBP1-RD in more patients than ever before. This will provide a more complex and full understanding of the various presentations, symptoms, and outcomes experienced by people with STXBP1-RD leading to improved counseling, prognosis, and healthcare.
How will the STARR Study Work?
Participants (the person with STXBP1-RD) and parents/caregivers will need to enroll in the study at one of four clinic sites: 1) Children’s Hospital of Philadelphia (CHOP); 2) Children’s Hospital Colorado (CHCO); 3) Weill Cornell Medicine (WCM); or 4) Texas Children’s Hospital (TCH). See next section on how to enroll.
At your first visit to the clinic of your choice (the baseline visit), you will be given a verbal explanation of how the study will work, the procedures that are involved, and have any questions you have addressed. You will also be asked to review, sign, and date a consent form agreeing to take part in the study.
A clinical assessment will then be performed on the participant. This may include a physical examination, blood tests, a medical history review, an EEG, and several behavioral assessments such as the Bayleys and Gross Motor Function Measure, which will be overseen by a healthcare professional. Either during this first clinic visit or shortly thereafter, the parent/caregiver will be asked to fill out several parent/caregiver reported outcome measures, such as the Vineland and Quality of Life Inventory. These may be completed before the first visit if directed to do so by clinic personnel. In follow-up clinic visits, the parent/caregiver reported outcome measures can be filled out in the week or two prior to the clinic visit.
Clinic visits and assessments and parent/caregiver reported outcome measures will be repeated every 6 months. Currently the study is planned to continue for 2 to 5 years. Figure 1 illustrates the timing of visits and the tests that will be performed.
Figure 1. Study Timeline and Assessments
Current Clinic Sites for the STARR Study
Children’s Hospital of Philadelphia / Center for Epilepsy and Neurodevelopmental Disorders
Baylor / Texas Children’s Hospital
Houston, Texas
Dr. Hsiao-Tuan Chao
STARR Study Flyer for Baylor / Texas Children’s
CONTACT: CHAO-LAB@BCM.EDU
Children’s Hospital Colorado
Weill Cornell Medicine
New York, New York
Dr. Zachary Grinspan
STARR Study Flyer for Weill Cornell Medicine
CONTACT: NAB2781@MED.CORNELL.EDU
Will participating in the STARR study cost any money?
Since clinic visits are associated with medical care, the visit itself should be covered by insurance and other funding available to each site. However, travel costs, to and from the clinic site, are not directly covered. The STXBP1 Foundation is providing a Patient Assistance Program for enrolled patients in the study to cover travel expenses up to $2000 / year. To apply for reimbursement for travel costs related to the STARR study, fill out the travel reimbursement form and follow the directions found here in the travel reimbursement policy. Please email finance@stxbp1disorders.org with any questions.
STARR Study Leadership
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, Strategic Director of the ENDD Center at Penn Medicine and Children’s Hospital of Philadelphia, serve as the administrative core leads.
If you have any questions concerning the STARR study please email RESEARCH@STXBP1DISORDERS.ORG.