The STARS study will enroll approximately 75 people at least 18 years old who are diagnosed with Angelman syndrome. The primary caregiver for the individual with Angelman syndrome also will need to be able to complete study activities and answer some questions from the study doctor at prescheduled times.
If you think STARS is an option for the individual with Angelman syndrome under your care and this person qualifies for the study, participation will last approximately four months and include phone appointments and four in-person visits to the medical center or study site conducting the study. These appointments will last most of the day and include examinations and assessments, plus a series of questions for you to answer.
The investigational medicine will be provided at no charge by Ovid Therapeutics, the study sponsor. Ovid will also provide travel assistance and help with other study-related costs. Please talk to your study team for details.
Angelman syndrome does not have a cure, and current treatments help manage specific symptoms. For example, people living with Angelman syndrome may receive physical, occupational and speech therapies for delays in gross and fine motor skills, behavior and speech. They may also receive medications for seizures and sleep or behavior issues.
By participating in STARS, the individual with Angelman syndrome may help medical researchers learn more about Angelman syndrome and possible future treatment options.
To join the STARS study, the individual with Angelman syndrome must meet these initial criteria:
STARS also has other requirements that help ensure the safety of all participants. If the individual with Angelman syndrome qualifies to participate in the study, these additional conditions will be reviewed with you by the team at your study site. This prescreener can help determine if the individual with Angelman syndrome under your care may qualify for STARS.
Angelman syndrome is a rare, genetic disorder that causes developmental delay and specific neurological characteristics, such as difficulty speaking, balancing and walking, as well as other symptoms like anxiety, sleep issues and seizures. Angelman syndrome is caused by a loss of functional Ubiquitin protein ligase E3A (UBE3A) in neurons. The UBE3A gene is located on chromosome 15.
The first signs of the disorder tend to be developmental delays, such as a lack of crawling or walking, that are typically seen between the ages of six to 12 months.
STARS is a Phase 2 double-blind placebo-controlled randomized clinical research study that is seeking adults at least 18 years old with confirmed Angelman syndrome to better understand the safety and potential effectiveness of the investigational medicine.
In a Phase 2 study, doctors further evaluate an investigational medicine for safety and effectiveness in a larger group of people or in a targeted population living with the disease or disorder being studied, which is the case for STARS.
In a double-blind study, neither the study team nor the caregivers or participants know who is receiving the investigational medicine and who is receiving placebo.
Randomized means the individual with Angelman syndrome will be assigned to receive either the investigational medicine or placebo by chance.
What is the investigational medication?
The investigational medication in the STARS study is OV101 (gaboxadol). To learn more about OV101, click here.
How can I find out if an individual with Angelman syndrome under my care is eligible to participate in the STARS study?
You can determine if the individual in your care is eligible by answering this short prescreener.
What is a placebo, and why is it important?
A placebo is a pill, liquid, spray or injection that contains the inactive ingredients of a study medication. Placebos are used in clinical studies when no specific medicines for that condition exist. Using a placebo helps doctors determine if the investigational medicine is effective and, if so, how effective. The placebo in the STARS study will be a capsule.
How will the privacy of an individual with Angelman syndrome be protected during the study?
The study sponsor, Ovid Therapeutics, respects the privacy of families and those who participate in its clinical studies and the personal healthcare data generated throughout the study. In accordance with HIPAA regulations, only the medical personnel associated with the study will have access to any personally identifiable medical information.
Who do I contact with questions?
For study-related questions, please email email@example.com.
Will I have to pay for the treatment in the STARS study?
No. You will not be charged for taking part in the clinical study. The investigational medicine and care required by the study will be provided at no expense by the study sponsor, Ovid Therapeutics.
Do I need to pay to travel to the study site?
You will be reimbursed for travel-related expenses. Please talk with your local study team for more information.
What if I have doubts about the individual with Angelman syndrome participating?
The study team is available to answer any questions you may have. Please remember that the healthcare professionals on your study team are a resource for you. Participating in STARS is completely voluntary, and you may withdraw the individual from the study at any time and without consequences.
You can learn more about STARS by downloading STARS materials. Additional materials may be available in the future.
The following organizations are dedicated to Angelman syndrome research and support for patients and families:
Information about clinical research studies and what it means to be a study participant can be found on the following websites:
Includes important information to consider before joining a clinical trial and informational videos on how to be a good clinical trial participant.
A comprehensive list of all clinical studies currently underway in the United States.
Information for parents and children who are interested in joining a clinical trial.
Clinical research studies, like STARS, help doctors and medical researchers learn how investigational medicines work and if they are safe. Studies are monitored by the U.S. Food and Drug Administration. In addition, independent ethics committees review research studies to safeguard the rights and welfare of research participants.
Clinical research studies depend on volunteers to participate. People agree to participate in studies for many reasons, including the chance to help others who may be or become affected by certain conditions, to give back to the scientific community, to play an active role in their health, and to gain access to the new potential medicines.
Deciding to volunteer in a clinical research study is an important choice that also comes with responsibilities. Talk to your study team to make sure you understand STARS, as well as the possible risks and benefits associated with the study. If you enroll the individual with Angelman syndrome under your care in the study, he or she may withdraw at any time and without consequences.
Ovid Therapeutics, the sponsor of the STARS study, is different. Ovid takes the time to understand its patient communities and weave that knowledge with its scientific insights to develop transformative therapies for patients and their families. The larger Ovid team, which includes patients, healthcare providers and the entire rare disease community, works together to develop medicines that can create meaningful change.