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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04106557




Registration number
NCT04106557
Ethics application status
Date submitted
25/09/2019
Date registered
27/09/2019
Date last updated
17/11/2020

Titles & IDs
Public title
A Study of OV101 in Individuals With Angelman Syndrome (AS)
Scientific title
A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome
Secondary ID [1] 0 0
OV101-19-001
Universal Trial Number (UTN)
Trial acronym
NEPTUNE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Disease or Condition Being Studied: Angelman Syndrome (AS) 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Gaboxadol
Treatment: Drugs - Placebo

Experimental: OV101 once daily (weight-based dosing) Other Name:Gaboxadol - OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration

Placebo Comparator: Placebo once daily - Matching placebo,oral, provided once daily at bedtime for 12 week duration


Treatment: Drugs: Gaboxadol
OV101 versus placebo once daily at bedtime for 12 weeks

Treatment: Drugs: Placebo
Matching placebo capsules to OV101 capsules.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) - Clinical Global Impressions-Improvement-Angelman Syndrome (CGI-I-AS) - Scale of 1-7 reflects a spectrum where 1 is most improved, 4 is no change, and 7 is most worsened.
Timepoint [1] 0 0
12 weeks

Eligibility
Key inclusion criteria
- Male or female and 2 to 12 years old (inclusive) at the time of informed consent

- Confirmed molecular diagnosis of AS

- Has a CGI-S-AS score of 3 or more at baseline.

- Meets the following age-appropriate body weight criterion:

1. Subjects 2 to 3 years old must have a minimum body weight of 9 kg.

2. Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).

- Stable concomitant mediations for at least 4 weeks before study start
Minimum age
2 Years
Maximum age
12 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Any condition that would limit study participation

- Clinically significant lab or vital sign abnormalities at the time of screening

- Poorly controlled seizures (weekly seizures of any frequency with a duration more than
3 minutes, weekly seizures occurring more than 3 times per week, each with a duration
of less than 3 minutes, or as defined by investigator assessment)

- Use of prescription medications for sleep, minocycline, or levodopa within the 4 weeks
prior to Day 1 or during the study. Benzodiazepines chronically administered for
seizure control are permitted.

- Cannot comply with protocol study assessments during screening or caregiver unable to
comply with study requirements.

- Enrolled in any clinical trial or used any investigational agent within the 30 days
before screening or concurrently with this study.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Ovid Therapeutics Investigative Site - Brisbane
Recruitment hospital [2] 0 0
Ovid Therapeutics Investigative Site - Heidelberg
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
3084 - Heidelberg
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Tennessee
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Germany
State/province [9] 0 0
Munich
Country [10] 0 0
Israel
State/province [10] 0 0
Ramat Gan
Country [11] 0 0
Netherlands
State/province [11] 0 0
Rotterdam

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ovid Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in
pediatric subjects with Angelman syndrome.
Trial website
https://clinicaltrials.gov/show/NCT04106557
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications