Primary Disease or Condition Being Studied: Angelman Syndrome (AS)Phase 3NEPTUNENCT04106557
A Study of OV101 in Individuals With Angelman Syndrome (AS)
A Phase 3 study testing Gaboxadol in people with Primary Disease or Condition Being Studied: Angelman Syndrome (AS), run by Ovid Therapeutics Inc. at 14 sites in 5 countries.
Trial summary
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome. Read more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
- What is being tested?
- Gaboxadol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 104 participants
- Where
- 14 sitesin 5 countries
- Study dates
- 9 Sep 2019to 2 Nov 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
- Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
- 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
Who cannot join
- 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.
Trial locations and contacts
14 sites in 5 countries.
14 sites in 5 countries
United States9 sites
Ovid Therapeutics Investigative Site
Boston, Massachusetts, 02115
Ovid Therapeutics Investigative Site
Chicago, Illinois, 60612
Ovid Therapeutics Investigative Site
Cincinnati, Ohio, 45229
Ovid Therapeutics Investigative Site
Lexington, Massachusetts, 02421
Ovid Therapeutics Investigative Site
Media, Pennsylvania, 19063
Ovid Therapeutics Investigative Site
Nashville, Tennessee, 37232
Ovid Therapeutics Investigative Site
Phoenix, Arizona, 85006
Ovid Therapeutics Investigative Site
San Diego, California, 92024
Ovid Therapeutics Investigative Site
Tacoma, Washington, 85006
Australia2 sites
Ovid Therapeutics Investigative Site
Brisbane, Queensland, 4101
Ovid Therapeutics Investigative Site
Heidelberg, Victoria, 3084
About this study
Official 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
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.
- Being tested
- Gaboxadol, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GaboxadolDrug
OV101 versus placebo once daily at bedtime for 12 weeks
PlaceboDrug
Matching placebo capsules to OV101 capsules.
| Study group | Role | What they receive |
|---|---|---|
| OV101 once daily (weight-based dosing) Other Name:Gaboxadol | Experimental | OV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration Receives: Drug: Gaboxadol |
| Placebo once daily | Placebo comparator | Matching placebo,oral, provided once daily at bedtime for 12 week duration Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2019
- Study start9 Sep 2019
- Primary completion2 Nov 2020
- Study completion2 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Disease or Condition Being Studied: Angelman Syndrome (AS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 104 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ovid Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04106557
- Sponsor's protocol ID
- OV101-19-001
- Acronym
- NEPTUNE
- Status last verified
- Jan 2024
- First submitted
- 25 Sep 2019
- Last updated
- 8 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Keary C, Bird LM, de Wit MC, Hatti S, Heimer G, Heussler H, Kolevzon A, Mathews A, Ochoa-Lubinoff C, Tan WH, Yan Y, Adams M. Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study. Eur J Paediatr Neurol. 2023 Nov;47:6-12. doi: 10.1016/j.ejpn.2023.07.008. Epub 2023 Aug 1.
PubMed 37639777
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT04106557
- Acronym / sponsor ID
- NEPTUNE / OV101-19-001
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 104 participants (actual)
- Lead sponsor
- Ovid Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Sep 2019 (actual)
- Primary completion
- 2 Nov 2020 (actual)
- Study completion
- 2 Nov 2020 (actual)
- First posted
- 27 Sep 2019 (actual)
- Results
- Posted 8 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to assess the efficacy and safety of oral OV101 (gaboxadol) in pediatric subjects with Angelman syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OV101 once daily (weight-based dosing) Other Name:Gaboxadol | Experimental | Drug: GaboxadolOV101 (gaboxadol), oral, provided once daily at bedtime for 12 week duration |
| Placebo once daily | Placebo comparator | Drug: PlaceboMatching placebo,oral, provided once daily at bedtime for 12 week duration |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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:
- Stable concomitant mediations for at least 4 weeks before study start
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GaboxadolOV101 versus placebo once daily at bedtime for 12 weeks | Drug | OV101 once daily (weight-based dosing) Other Name:Gaboxadol |
| PlaceboMatching placebo capsules to OV101 capsules. | Drug | Placebo once daily |
Primary outcome
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks
Time frame: 12 weeks
To evaluate the efficacy of OV101 versus placebo as assessed by the Clinical Global Impressions-Improvement-Angelman syndrome (CGI-I-AS) score at Week 12. CGIA-I-AS scores range from 1 (very much improved), to 4 (no change), to 7 (very much worse).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37639777Keary C, Bird LM, de Wit MC, Hatti S, Heimer G, Heussler H, Kolevzon A, Mathews A, Ochoa-Lubinoff C, Tan WH, Yan Y, Adams M. Gaboxadol in angelman syndrome: A double-blind, parallel-group, randomized placebo-controlled phase 3 study. Eur J Paediatr Neurol. 2023 Nov;47:6-12. doi: 10.1016/j.ejpn.2023.07.008. Epub 2023 Aug 1.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Gaboxadol in people with Primary Disease or Condition Being Studied: Angelman Syndrome (AS), run by Ovid Therapeutics Inc. at 14 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Australia, Germany, Israel, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04106557, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2024.