A Study in Adults and Adolescents With Angelman Syndrome

Brief Title

A Study in Adults and Adolescents With Angelman Syndrome

Official Title

A Phase 2 Adult and Adolescent 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 and adolescent 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


Primary Outcome

Incidence of adverse events in placebo and active treatment groups

Secondary Outcome

 Exploratory efficacy measurement for motor function

Condition

Angelman Syndrome

Intervention

OV101 Regimen 1

Study Arms / Comparison Groups

 OV101 regimen 1
Description:  OV101 once 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

88

Start Date

January 2016

Completion Date

August 6, 2018

Primary Completion Date

June 7, 2018

Eligibility Criteria

        Inclusion Criteria

          1. Age 13- 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

13 Years - 49 Years

Accepts Healthy Volunteers

No

Contacts

Amit Rakhit, MD, MBA, , 

Location Countries

Israel

Location Countries

Israel

Administrative Informations


NCT ID

NCT02996305

Organization ID

OV101-15-001


Responsible Party

Sponsor

Study Sponsor

Ovid Therapeutics Inc.


Study Sponsor

Amit Rakhit, MD, MBA, Study Director, Ovid Therapeutics Inc.


Verification Date

August 2018