A Study in Adults and Adolescents With Angelman Syndrome
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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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Angelman Syndrome | Drug: OV101 Regimen 1 Drug: OV101 regimen 2 Other: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 88 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol |
Study Start Date : | January 2016 |
Actual Primary Completion Date : | June 7, 2018 |
Actual Study Completion Date : | August 6, 2018 |

Arm | Intervention/treatment |
---|---|
Experimental: OV101 regimen 1
OV101 once daily
|
Drug: OV101 Regimen 1
Other Name: Gaboxadol |
Experimental: OV101 regimen 2
OV101 twice daily
|
Drug: OV101 regimen 2
Other Name: Gaboxadol |
Placebo Comparator: Placebo
Twice daily
|
Other: Placebo |
- Incidence of adverse events in placebo and active treatment groups [ Time Frame: Change from baseline to week 12 ]
- 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)

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Ages Eligible for Study: | 13 Years to 49 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria
- Age 13- 49 years
- Diagnosis of Angelman syndrome
- 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
- Has a caregiver capable of providing informed consent on behalf of the subject and able to attend scheduled study visits
- Able to ingest study medication
- Caregivers must agree not to post any subject or study information on social media
Exclusion Criteria
- Unable to perform the study related safety and exploratory efficacy assessments, such as motor function
- Poorly controlled seizure activity
- Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
- Pregnancy or women of child-bearing potential who are not using and acceptable method of contraception
- 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
- Allergy to OV101 or any excipients
- At increased risk of harming self and/or others based on investigator assessment
- Any condition or reason that in the opinion of the investigator makes the subject unsuitable for enrollment
- Inability of subject or caregiver to comply with study requirements
Other protocol-defined inclusion/exclusion criteria may apply.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02996305
United States, Arizona | |
Ovid Therapeutics Investigative Site | |
Phoenix, Arizona, United States | |
United States, California | |
Ovid Therapeutics Investigative Site | |
San Diego, California, United States | |
United States, Florida | |
Ovid Therapeutics Investigative Site | |
Gainesville, Florida, United States | |
Ovid Therapeutics Investigative Site | |
Tampa, Florida, United States | |
United States, Georgia | |
Ovid Therapeutics Investigative Site | |
Atlanta, Georgia, United States | |
United States, Illinois | |
Ovid Therapeutics Investigative Site | |
Chicago, Illinois, United States | |
United States, Massachusetts | |
Ovid Therapeutics Investigative Site | |
Boston, Massachusetts, United States | |
Ovid Therapeutics Investigative Site | |
Lexington, Massachusetts, United States | |
United States, Ohio | |
Ovid Therapeutics Investigative Site | |
Cincinnati, Ohio, United States | |
United States, Pennsylvania | |
Ovid Therapeutics Investigative Site | |
Media, Pennsylvania, United States | |
United States, South Carolina | |
Ovid Therapeutics Investigative Site | |
Greenwood, South Carolina, United States | |
United States, Tennessee | |
Ovid Therapeutics Investigative Site | |
Nashville, Tennessee, United States | |
Israel | |
Ovid Therapeutics Investigative Site | |
Ramat Gan, Israel |
Study Director: | Amit Rakhit, MD, MBA | Ovid Therapeutics Inc. |
Responsible Party: | Ovid Therapeutics Inc. |
ClinicalTrials.gov Identifier: | NCT02996305 |
Other Study ID Numbers: |
OV101-15-001 |
First Posted: | December 19, 2016 Key Record Dates |
Last Update Posted: | August 8, 2018 |
Last Verified: | August 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Angelman Syndrome Syndrome Disease Pathologic Processes Movement Disorders Central Nervous System Diseases Nervous System Diseases Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders Genetic Diseases, Inborn |
Gaboxadol Analgesics Sensory System Agents Peripheral Nervous System Agents Physiological Effects of Drugs Anticonvulsants GABA Agonists GABA Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action |