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A Study in Adults With Angelman Syndrome

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Disease Information

Descriptive Information
Brief Title † A Study in Adults With Angelman Syndrome
Official Title † A Phase 2 Adult Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
Brief Summary The purpose of the study is to assess the safety and tolerability of oral OV101 (gaboxadol) in adult subjects with Angelman syndrome. In addition, several exploratory efficacy outcome measures will be investigated.
Detailed Description Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
Study Phase Phase 2
Study Type † Interventional
Study Design †
Primary Outcome Measure † Incidence of adverse events in placebo and active treatment groups
Secondary Outcome Measure † Exploratory efficacy measurement for motor function
Condition † Angelman Syndrome
Intervention † DrugOV101 Regimen 1
Study Arms / Comparison Groups OV101 regimen 1 OV101 once daily OV101 regimen 2 OV101 twice daily Placebo Twice daily
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 75
Start Date † January 2016
Completion Date November 2017
Primary Completion Date October 2017
Eligibility Criteria † Inclusion Criteria 1. Age 18- 49 years 2. Diagnosis of Angelman syndrome 3. Receiving a stable regimen of concomitant medications for at least 4 weeks prior to Baseline, and able to maintain these throughout the duration of the study 4. Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits 5. Able to ingest study medication 6. Caregivers must agree not to post any subject or study information on social media Exclusion Criteria 1. Unable to perform the study related safety and exploratory efficacy assessments, such as motor function 2. Poorly controlled seizure activity 3. Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study 4. Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception 5. Concomitant use of minocycline, levodopa, zolpidem, zaleplon, eszopiclone, ramelteon, and cannabinoid derivatives, or any other use of any investigational agent, device, and/or investigational procedure 4 weeks prior to Baseline and during the study 6. Allergy to OV101 or any excipients 7. At increased risk of harming self and/or others based on investigator assessment 8. Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment 9. Inability of subject or caregiver to comply with study requirements Other protocol-defined inclusion/exclusion criteria may apply.
Gender All
Ages 18 Years - 49 Years
Accepts Healthy Volunteers No
Contacts †† Amit Rakhit, MD, MBA, +1 (646) 661-7661, info@ovidrx.com
Location Countries † United States
Administrative Information
NCT ID † NCT02996305
Organization ID OV101-15-001
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Ovid Therapeutics Inc.
Collaborators ††
Investigators † Study Director: Amit Rakhit, MD, MBA, Ovid Therapeutics Inc.
Information Provided By
Verification Date May 2017
First Received Date † December 2, 2016
Last Updated Date May 24, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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