S.T.A.R.S.

A Randomized, Double-Blind, Placebo Controlled Study of Safety and Efficacy of Gaboxadol in Adult Subjects With Angelman Syndrome.

STARS

Presented by:

USF Health andusfhealth

In conjunction with:

FASTangel

We Are Currently Looking for New Options for Angelman Syndrome Patients!

An upcoming Phase 2 clinical research study will investigate a potential new treatment that may improve conditions for people with Angelman syndrome and their caregivers.

We are investigating potential new options for people with Angelman syndrome

Managing the disease can be an ongoing struggle for people living with Angelman syndrome and their families. A clinical study will soon begin enrolling patients to investigate a potential treatment that may improve the health and conditions of people with Angelman syndrome and their caregivers. “We don’t have much data on adult Angelman syndrome patients,” said Eileen Braun, Executive Director of Angelman Syndrome Foundation Inc., citing that this study could be hugely beneficial in expanding what we know about this population. The study will be sponsored by Ovid Therapeutics, which has partnered with two national Angelman syndrome foundations to discuss and address the needs of the patient community.

A clinical research study, called the STARS study, is now enrolling participants with a confirmed diagnosis of Angelman syndrome who are at least 18 years old. The STARS study will evaluate the safety and potential effectiveness of an investigational medicine for Angelman syndrome. The STARS Study will enroll approximately 75 people at 15 U.S. study sites. Participation in STARS will last approximately four months and include phone appointments and four in-person visits to the medical center conducting the study, or study site. These appointments will last most of the day and include examinations and assessments, plus a series of questions for you to answer. There are no costs for taking part in the STARS study. The investigational medicine and care required by the study will be provided at no expense and you will be reimbursed for travel-related expenses by the study sponsor, Ovid Therapeutics™.

Goal of the STARS Study

“Number one, we want to make sure [study medication OV101] is safe for patients,” said Dr. Lynne Bird, Professor of Clinical Pediatrics at University of California, San Diego.

After safety, the secondary goal of the study is exploratory, testing OV101 for efficacy in improving several disease symptoms.

“The STARS study will look at various aspects of Angelman syndrome, such as motor function by assessing gait and walking, seizures by measuring at brain waves by an EEG, sleep metrics, behavior improvements, and overall healthcare-related quality of life by using questionnaires,” said Dr. Amit Rakhit, Chief Medical and Portfolio Management Officer at Ovid Therapeutics.

The STARS clinical study is now open for adult Angelman syndrome patients across the country. Find more information on Angelman syndrome and the upcoming trial at www.angelmanstudy.com.

Disclaimers:

This clinical research trial will be performed at the USF Health Byrd Institute on the USF Campus, as well as at Florida Hospital Tampa, both on Fowler Avenue in Tampa, FL

There may be an improved quality of life for AS patients as a result of this study. It is possible that OV101 may alleviate the motor dysfunction observed in individuals with AS.

Accepted participants will be enrolled in the study for a period of about four months.

Travel may be arranged and paid for participants, and should be discussed with the study coordinator. Additionally, a small stipend of 81$ per day may be provided by the sponsor to help ease the financial burden.

In order to be eligible for this study, the participant must meet the following requirements:

Participant must be between 18 and 49 years at the time of informed consent  |  Participant must have a molecular confirmation of AS  | Receiving a stable dose of concomitant medications, including anti-epileptic medication, gabapentin, clonidine, melatonin, trazadone, supplements, and special diets, for at least 4weeks prior to Baseline, and able to maintain these throughout the duration of the study  |  Has a parent or caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits to participate in all assessments described in the protocol  |  Able to attend scheduled study visits and be willing and capable to perform the required clinical evaluations, including using the wearable device for actigraphy.A single caregiver should be identified to wear the ActiWatch for the duration of the study and attend scheduled study visits  |  Able to ingestthe study drug  |  Caregivers must agree not to post any subject’s personal medical data related to the study or information related to the study on any website or social media site (e.g. Facebook, Twitter, etc.) until the study is completed.

The following conditions preclude entry into this study:

Non-ambulatory subjects (e.g. requiring a wheelchair) not able to perform the assessments of Motor Ability/Function  |  Poorly controlled seizures defined as >3seizures lasting less than 1minute (atonic, atypical, absence, myoclonic, focal) per week or > 1 seizure episode lasting more than 1minute (focal,
tonic-clonic) per weekor as per investigator judgement  |  Concomitant cardiovascular or respiratory diseases of a degree that would limit participation in the study  |  Concomitant liver disease with alanine aminotransferase or aspartate aminotransferase > 2.5 × upper limit of normal (ULN)  |  Concomitant renal disease with creatinine above ULN  |  Concomitant hematologic disease with absolute neutrophil count <2 × 109/L, platelets < 50 × 109/L or hemoglobin <80 g/L | Pregnancy  |  Women of child-bearing potential who are not using a double-barrier method of contraception (e.g. condoms plus oral contraceptives), with abstinence being an accepted method  |  Concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, and cannabinoid derivatives, and any other use of any investigational agent, device, and/or investigational procedure 4 weeks prior to Baseline and during the study  |  Allergy to OV101 or any excipients  |  At increasedrisk of harming self and/or others based on investigator assessment  |  Any condition or reasonthat in the opinion of the investigator makes the subject unsuitable for enrollment  |  Inability of subject or caregiver to comply with study requirements

Protocol #: 00023755

Edwin Weeber, Ph.D.

University of South Florida

4001 E Fowler Ave

Tampa, FL 33613

Office: (813) 396-9995