Angelman SyndromePhase 2NCT02996305
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
A Phase 2 study testing OV101 Regimen 1 and OV101 regimen 2 in people with Angelman Syndrome, run by Healx AI at 13 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 13 to 49 years
- Conditions the study is for
- Angelman Syndrome
- What is being tested?
- OV101 Regimen 1 and OV101 regimen 2
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 88 participants
- Where
- 13 sitesin 2 countries
- Study dates
- Jan 2016to 6 Aug 2018
- Phase
- Phase 2
- 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 13 to 49 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 10Show fewer
- 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.
Trial locations and contacts
13 sites in 2 countries.
13 sites in 2 countries
United States12 sites
Ovid Therapeutics Investigative Site
Atlanta, Georgia
Ovid Therapeutics Investigative Site
Boston, Massachusetts
Ovid Therapeutics Investigative Site
Chicago, Illinois
Ovid Therapeutics Investigative Site
Cincinnati, Ohio
Ovid Therapeutics Investigative Site
Gainesville, Florida
Ovid Therapeutics Investigative Site
Greenwood, South Carolina
Ovid Therapeutics Investigative Site
Lexington, Massachusetts
Ovid Therapeutics Investigative Site
Media, Pennsylvania
Ovid Therapeutics Investigative Site
Nashville, Tennessee
Ovid Therapeutics Investigative Site
Phoenix, Arizona
Ovid Therapeutics Investigative Site
San Diego, California
Ovid Therapeutics Investigative Site
Tampa, Florida
About this study
Official title: A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
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.
Read the detailed description
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
- Conditions
- Angelman Syndrome
- Being tested
- OV101 Regimen 1, OV101 regimen 2, Placebo
- Medical subject headings
- Angelman Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OV101 Regimen 1Drug
Also called Gaboxadol
OV101 regimen 2Drug
Also called Gaboxadol
PlaceboOther
| Study group | Role | What they receive |
|---|---|---|
| OV101 regimen 1 | Experimental | OV101 once daily Receives: Drug: OV101 Regimen 1 |
| OV101 regimen 2 | Experimental | OV101 twice daily Receives: Drug: OV101 regimen 2 |
| Placebo | Placebo comparator | Twice daily Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Events in Placebo and Active Treatment Groups
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.
Measured: Baseline and Week 12
Incidence of Adverse Events in Placebo and Active Treatment Groups
The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set.
Measured: Baseline and Week 12
Incidence of Adverse Events in Placebo and Active Treatment Groups
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.
Measured: Baseline and Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2016 (estimated)
- Study startJan 2016
- Primary completion7 Jun 2018
- Study completion6 Aug 2018
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 Angelman SyndromeStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 88 participants (actual)
Sponsor and investigators
- Lead sponsor
- Healx AI
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amit Rakhit, MD, MBA, Study director, Healx AI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02996305
- Sponsor's protocol ID
- OV101-15-001
- Status last verified
- Jul 2025
- First submitted
- 2 Dec 2016
- Last updated
- 3 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
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.
PubMed 33443117
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
- ClinicalTrials.gov ID
- NCT02996305
- Acronym / sponsor ID
- OV101-15-001
- Conditions
- Angelman Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 88 participants (actual)
- Lead sponsor
- Healx AIIndustry (pharma, biotech, devices)
- Study start
- Jan 2016
- Primary completion
- 7 Jun 2018 (actual)
- Study completion
- 6 Aug 2018 (actual)
- First posted
- 19 Dec 2016 (estimated)
- Results
- Posted 31 Mar 2022
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 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| OV101 regimen 1 | Experimental | Drug: OV101 Regimen 1OV101 once daily |
| OV101 regimen 2 | Experimental | Drug: OV101 regimen 2OV101 twice daily |
| Placebo | Placebo comparator | Other: PlaceboTwice daily |
Who may qualify
- Ages
- 13 to 49 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (10 listed)
- 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
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 |
|---|---|---|
| OV101 Regimen 1Also: Gaboxadol | Drug | OV101 regimen 1 |
| OV101 regimen 2Also: Gaboxadol | Drug | OV101 regimen 2 |
| Placebo | Other | Placebo |
Primary outcome
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.
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.
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33443117Bird 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.
- Individual participant dataSharing planned: No
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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.
Amit Rakhit, MD, MBA study director
Healx AI
13 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing OV101 Regimen 1 and OV101 regimen 2 in people with Angelman Syndrome, run by Healx AI at 13 sites in 2 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 6 inclusion and 10 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 3 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 31 Mar 2022). 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?
Incidence of Adverse Events in Placebo and Active Treatment Groups (time frame: Baseline and Week 12). The study lists 3 primary outcomes in all. 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 NCT02996305, 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 3 Aug 2025.