What is STARR?
STARR — which stands for STXBP1 Clinical Trial Ready) is a natural history study for STXBP1-Related Disorders (STXBP1-RD) to better understand how the disorder affects someone over time. This is done through multiple clinic visits over several years. Though this is an observational study, meaning it will not test any new medicines or therapies, it is a very important step towards preparing our community for upcoming clinical trials for new drugs and treatments, and improving clinical care. During the study, patients will continue to receive their current medications and supportive care such as physical and occupational therapies.
The STARR study is funded by the STXBP1 Foundation through a transformational campaign, FLOURISH, led by the Finn Beaubien family, and by the ENDD Center at Penn Medicine and Children's Hospital of Philadelphia. Also, the STARR site at Cincinnati Children’s Hospital is being funded by the Glazer family.
The purpose of the STARR study is to identify a set of measurements that can be used to describe how STXBP1-RD changes over time in individuals. These measurements could then be used to demonstrate to drug-approval agencies, like the FDA, that a new therapy is effective enough to allow it to be used in the STXBP1-RD population.
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 in other studies about STXBP1 such as Citizen Health, Simons Searchlight, or RARE-X, and wonder what’s special about the STARR study and why you should join. The other studies mostly get information from past medical records or through surveys, and the studies were designed for multiple conditions. 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. STARR focuses specifically on aspects of development and progression of STXBP1 over time, using tools and measurements that collect information that is important for STXBP1. Collecting data over time is called a prospective study, and this approach can be helpful to support development of clinical trials.
Importantly, the STARR study is a big chance to learn more about STXBP1 in more people than ever before. We hope to get a better understanding of how STXBP1 affects people, which can help improve how doctors give advice, predict what might happen, and take care of people with STXBP1.
How Can I Join STARR and What Should I Expect?
The STARR study is being conducted at 6 clinical sites:
Children’s Hospital of Philadelphia; Philadelphia, PA
Children’s Hospital Colorado; Denver, CO
Cincinnati Children’s Hospital; Cincinnati, OH (opening in future)
Stanford Children’s Hospital; San Francisco, CA
Texas Children’s Hospital; Houston, TX
Weill Cornell Medicine; New York City, NY
Currently, 5 sites are enrolling participants with STXBP1-RD: Children’s Hospital of Philadelphia, Children’s Hospital of Colorado, Texas Children’s Hospital, Stanford Children’s Hospital, and Weill Cornell Medicine. The site at Cincinnati Children’s Hospital is still being set up.
Enrollment for the study is done at the individual clinics and contact details for each clinic are in the section below. Families are asked to enroll in the clinic site that is geographically closest to them unless they already have an established relationship with one of the other clinic sites.
The STARR study involves visiting your chosen clinic every 6 months. Visiting the clinic every 6 months will make it easier to properly describe how STXBP1-RD changes over time. Likewise, it is important to visit the same clinic every 6 months to ensure that the measurements and tests being conducted in an individual with STXBP1-RD are being conducted by the same healthcare team over the entire course of the study.
At the first visit to the clinic (“baseline visit”), they'll explain how the study works, what will happen during the visits, and answer any questions you have. You'll also need to sign a form saying you agree to be in the study. They will then do an evaluation of the participant including a physical exam, blood tests, and a review of their medical history. They will then conduct several clinical assessments that involve a series of questionnaires, surveys, and testing to see how the participant moves and acts. Some sites may also perform an EEG. All assessments and testing will be carried out by trained healthcare professionals. The evaluation of the participant and clinical assessments will be repeated at every 6 month visit.
Additionally, the parent or caregiver will also need to fill out a set of parent/caregiver reported outcome measures about how the participant is doing, like the Vineland and Quality of Life Inventory. These measures are in the form of questionnaires and surveys and will be done online through the RARE-X platform. We request that these measures be filled out, at your own convenience, within 7 days of visiting the clinic.
Figure 1 shows when the visits are and what tests will be done.
Figure 1. Study Timeline and Assessments. Clinic visits are scheduled every six months for participants.
The STARR Study is now also listed on clinicaltrials.gov (NCT06555965).
Current Clinic Sites for the STARR Study
Children’s Hospital of Philadelphia / Center for Epilepsy and Neurodevelopmental Disorders
Stanford Children’s Health
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?
Most of the clinic visit medical costs are typically covered by insurance and other funding the clinic has, but some of our families may have uncovered medical expenses. In addition, families may incur travel expenses to and from the clinic. The STXBP1 Foundation has expanded its Patient Assistance Program for enrolled patients in the study to help cover both medical and travel expenses — now up to $3000 / year. If you need help with incurred costs for the STARR study, you can apply for reimbursement by filling out the travel reimbursement form and follow the instructions in the travel reimbursement policy and the accompanying medical expenses 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.