Clinical Trial: A Study in Adults With Angelman Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 Summary: Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
Sponsor: Ovid Therapeutics Inc.

Current Primary Outcome: Incidence of adverse events in placebo and active treatment groups [ Time Frame: Change from baseline to week 12 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Modified Performance Oriented Mobility Assessment-Gait (mPOMA-G)
• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Bayley Scales of Infant and Toddler Development (BSID-III)
• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Pediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Sleep onset latency (SOL)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Total sleep time (TST)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Wake after sleep onset (WASO)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Nocturnal awakenings
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Sedentary behavior
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Daily activity level
• Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
  Assessed by Aberrant Behavior Checklist (ABC)
• Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
  Assessed by Anxiety, Depression, and Mood Scales (ADAMS)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by EuroQol 5 Dimension 5 Level (EQ5D5L)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Child Health Assessment Questionnaire (CHAQ)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Parent Global Impression (PGI)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Short Form Health Survey 36 Items (SF-36)
• Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
  Assessed by Clinical Global Impression - Improvement (CGI-I)
• Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
  Assessed by Clinical Global Impression - Severity (CGI-S)

Original Secondary Outcome:

• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Modified Performance Oriented Mobility Assessment-Gait (mPOMA-G)
• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Bayley Scales of Infant and Toddler Development (BSID-III)
• Exploratory efficacy measurement for motor function [ Time Frame: Change from baseline to week 12 ]
  Assessed by Pediatric Evaluation of Disability Inventory - Computer Adaptive Test (PEDI-CAT)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Sleep onset latency (SOL)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Total sleep time (TST)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Wake after sleep onset (WASO)
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Nocturnal awakenings
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Sedentary behavior
• Exploratory efficacy measurement for sleep [ Time Frame: Change from baseline to week 12 ]
  Assessed by Daily activity level
• Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
  Assessed by Aberrant Behavior Checklist (ABC)
• Exploratory efficacy measurement for behavior [ Time Frame: Change from baseline to week 12 ]
  Assessed by Anxiety, Depression, and Mood Scales (ADAMS)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by EuroQol 5 Dimension 5 Level (EQ5D5L)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Child Health Assessment Questionnaire (CHAQ)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Child Health Assessment Questionnaire (PGI)
• Exploratory efficacy measurement for health related quality of life [ Time Frame: Change from baseline to week 12 ]
  Assessed by Short Form Health Survey 36 Items (SF-36)
• Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
  Assessed by Clinical Global Impression - Improvement (CGI-I)
• Exploratory efficacy measurement for overall clinical change [ Time Frame: Change from baseline to week 12 ]
  Assessed by Clinical Global Impression - Severity (CGI-S)

Information By: Ovid Therapeutics Inc.

Dates:
Date Received: December 2, 2016
Date Started: January 2016
Date Completion: November 2017
Last Updated: March 23, 2017
Last Verified: March 2017