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A Study in Adults and Adolescents With Angelman Syndrome (STARS)

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
Results First Posted : March 31, 2022
Last Update Posted : March 31, 2022
Sponsor:
Information provided by (Responsible Party):
Ovid Therapeutics Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Angelman Syndrome Drug: OV101 Regimen 1 Drug: OV101 regimen 2 Other: Placebo Phase 2

Detailed Description:
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
Study Design
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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

Resource links provided by the National Library of Medicine


Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Adverse Events in Placebo and Active Treatment Groups [ Time Frame: Baseline and Week 12 ]
    Summary of Subjects Reporting at least one Treatment Emergent Adverse Event (TEAEs), Safety Set. The table below summarizes the subjects who experienced TEAEs in the study.

  2. Incidence of Adverse Events in Placebo and Active Treatment Groups [ Time Frame: Baseline and Week 12 ]
    The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.

  3. Incidence of Adverse Events in Placebo and Active Treatment Groups [ Time Frame: Baseline and Week 12 ]
    Treatment-related TEAEs (Treatment Emergent Adverse Event) in ≥ 2 Subjects in OV101 Combined, Safety Set. The incidence of TEAEs assessed as treatment-related (at least possibly related to study drug, by the Investigator). Preferred Term in the table below.


Eligibility Criteria
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Ages Eligible for Study:   13 Years to 49 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Ovid Therapeutics Inc.
Investigators
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Study Director: Amit Rakhit, MD, MBA Ovid Therapeutics Inc.
  Study Documents (Full-Text)

Documents provided by Ovid Therapeutics Inc.:
Study Protocol and Statistical Analysis Plan: Study Protocol  [PDF] October 24, 2017
Statistical Analysis Plan: Statistical Analysis Plan document  [PDF] June 27, 2018

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Ovid Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT02996305    
Other Study ID Numbers: OV101-15-001
First Posted: December 19, 2016    Key Record Dates
Results First Posted: March 31, 2022
Last Update Posted: March 31, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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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