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A Study in Adults and Adolescents With Angelman Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02996305
Recruitment Status : Completed
First Posted : December 19, 2016
Last Update Posted : August 8, 2018
Sponsor:
Information provided by (Responsible Party):
Ovid Therapeutics Inc.

Tracking Information
First Submitted Date  ICMJE December 2, 2016
First Posted Date  ICMJE December 19, 2016
Last Update Posted Date August 8, 2018
Study Start Date  ICMJE January 2016
Actual Primary Completion Date June 7, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 14, 2016)
Incidence of adverse events in placebo and active treatment groups [ Time Frame: Change from baseline to week 12 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 4, 2017)
  • 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 Measures  ICMJE
 (submitted: December 14, 2016)
  • 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)
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study in Adults and Adolescents With Angelman Syndrome
Official Title  ICMJE 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Angelman Syndrome
Intervention  ICMJE
  • Drug: OV101 Regimen 1
    Other Name: Gaboxadol
  • Drug: OV101 regimen 2
    Other Name: Gaboxadol
  • Other: Placebo
Study Arms  ICMJE
  • Experimental: OV101 regimen 1
    OV101 once daily
    Intervention: Drug: OV101 Regimen 1
  • Experimental: OV101 regimen 2
    OV101 twice daily
    Intervention: Drug: OV101 regimen 2
  • Placebo Comparator: Placebo
    Twice daily
    Intervention: Other: Placebo
Publications * Bird LM, Ochoa-Lubinoff C, Tan WH, Heimer G, Melmed RD, Rakhit A, Visootsak J, During MJ, Holcroft C, Burdine RD, Kolevzon A, Thibert RL. The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome. Neurology. 2021 Feb 16;96(7):e1024-e1035. doi: 10.1212/WNL.0000000000011409. Epub 2020 Dec 21.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 15, 2018)
88
Original Estimated Enrollment  ICMJE
 (submitted: December 14, 2016)
75
Actual Study Completion Date  ICMJE August 6, 2018
Actual Primary Completion Date June 7, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 13 Years to 49 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Israel,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02996305
Other Study ID Numbers  ICMJE OV101-15-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ovid Therapeutics Inc.
Study Sponsor  ICMJE Ovid Therapeutics Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Amit Rakhit, MD, MBA Ovid Therapeutics Inc.
PRS Account Ovid Therapeutics Inc.
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP