Angelman SyndromePhase 3ELARANCT03882918
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
A Phase 3 study testing OV101 in people with Angelman Syndrome, run by Healx AI at 11 sites in 2 countries.
Trial summary
This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). 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
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 141 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 31 Jan 2019to 30 Jun 2021
- 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 13 to 49 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Each subject must meet all the following criteria to be enrolled in this study:
- Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
- Is male or female and 13 to 49 years old (inclusive) at the time of inclusion in the OV101-15-001 or OV101-16-001 study.
- Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
- Has an LAR/caregiver capable of providing informed consent and able to attend all scheduled study visits, oversee the administration of study drug, and provide feedback regarding the subject's symptoms and performance as described in the protocol.
- Provides assent to the protocol (to the extent possible and in accordance with local institutional review board (IRB) and regulatory requirements) and has an LAR/caregiver who will provide written informed consent. Subjects providing assent must do so at the same visit as LAR/caregiver written informed consent is provided.
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- Can swallow study drug capsules or ingest the contents of study drug capsules after sprinkling the capsule contents onto 1 spoon of applesauce or low-fat yogurt.
- Is currently receiving a stable dose of concomitant medications such as anti-epileptic medication, gabapentin, clonidine, trazadone, melatonin, and special diets for at least 4 weeks prior to Baseline.
- Agrees to remain sexually abstinent from the first day of screening until 30 days after the last dose of study treatment.
- Has LAR/caregiver(s) who agree not to post any of the subject's personal medical data or information related to the study on any website or social media site (eg, Facebook, Instagram, Twitter) until notified that the study is completed.
Who cannot join
- Subjects meeting any of the following criteria will be excluded from the study:
- Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
- Has a concomitant disease (eg, gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or any clinically significant finding at Screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in this study.
- Has poorly controlled seizures defined as > 3 seizures lasting < 3 minutes per week or > 1 seizure episode lasting more than 3 minutes per week or as per medical monitor judgment.
- Has clinically significant clinical laboratory abnormalities or vital signs at the time of screening (eg, alanine aminotransferase or aspartate aminotransferase > 2.5 × upper limit of normal; total bilirubin or creatinine > 1.5 × upper limit of normal). Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
- Current use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep within the 4 weeks prior to Day 1. Benzodiazepines administered for situational anxiety related to occasional procedures or events are permitted.
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- Has a history of suicidal behavior or is considered by the investigator to be at increased risk of suicide.
- Has any condition or circumstance that, in the opinion of the investigator, makes the subject unsuitable for enrollment.
- Has enrolled in any clinical trial or used any investigational agent or device, or has participated in any investigational procedure, within the 30 days before screening or does so concurrently with this study.
- Is a family member of the investigator or of study site staff.
Trial locations and contacts
11 sites in 2 countries.
11 sites in 2 countries
United States10 sites
Ovid Therapeutics Investigative Site
Atlanta, Georgia, 30322
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, 37212
Ovid Therapeutics Investigative Site
Phoenix, Arizona, 85006
Ovid Therapeutics Investigative Site
San Diego, California, 32123
Ovid Therapeutics Investigative Site
Tacoma, Washington, 98405
About this study
This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.
Read the detailed description
This will be an open-label, long-term safety study for evaluation of further treatment with OV101 in subjects with AS who have completed previous Ovid studies (OV101-15-001 or OV101-16-001). There will be no placebo treatment. As this study will enroll subjects who have completed previous AS studies for different periods of time before entering this study, subjects will be required to complete screening and baseline visits before receiving OV101 under this protocol.The secondary objective of this study is to evaluate the long-term efficacy of OV101 treatment assessed by changes in behavior, sleep, and functioning in individuals with AS.
- Conditions
- Angelman Syndrome
- Being tested
- OV101
- Medical subject headings
- Angelman Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OV101Drug
Also called gaboxadol
Each subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime.
| Study group | Role | What they receive |
|---|---|---|
| OV101 | Experimental | once daily at bedtime (gaboxadol) Receives: Drug: OV101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Participants Experiencing Adverse Events in Active Treatment Group
Safety assessments related to the primary study objective of evaluating the safety and tolerability of OV101, including serious adverse events (SAEs) and adverse events (AEs) leading to study discontinuation, as assessed by the number of participants who experienced at least one adverse event.
Measured: Change from baseline to Week 39
Secondary outcomes (1)
To Evaluate the Long-term Efficacy of OV101 Treatment as Assessed by Changes in Behavior in Study Participants With AS Who Were at Least 4 Years Old.
The long-term efficacy of OV101 was assessed by changes in irritability using the Aberrant Behavior Checklist - Irritability subscale (ABC-I), part of the broader ABC-Community (ABC-C). The final value reflects change from baseline to early termination at 39 weeks. The ABC-I consists of 15 items measuring behaviors such as aggression, self-injury, temper tantrums, and mood instability. Each item is rated by a caregiver on a 4-point scale: 0 = not at all a problem; 1 = slight problem; 2 = moderately serious; 3 = severe. Scores range from 0 (no symptoms) to 45 (maximum severity). A higher total score indicates greater behavioral disturbance and worse clinical status. The scale provides a standardized means to track symptom changes over time, making it suitable for evaluating treatment efficacy in neurodevelopmental and neuropsychiatric disorders.
Measured: change from baseline to 12 wks and baseline to early termination of the study at 39 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2019
- Study start31 Jan 2019
- Primary completion4 Jun 2021
- Study completion30 Jun 2021
Why it stopped: Company decision
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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 141 participants (actual)
Sponsor and investigators
- Lead sponsor
- Healx AI
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03882918
- Sponsor's protocol ID
- OV101-18-002
- Acronym
- ELARA
- Status last verified
- Jul 2025
- First submitted
- 18 Mar 2019
- Last updated
- 18 Jul 2025
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Heussler HS. Emerging Therapies and challenges for individuals with Angelman syndrome. Curr Opin Psychiatry. 2021 Mar 1;34(2):123-128. doi: 10.1097/YCO.0000000000000674.
PubMed 33395098
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
- ClinicalTrials.gov ID
- NCT03882918
- Acronym / sponsor ID
- ELARA / OV101-18-002
- Conditions
- Angelman Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Company decision
- Enrolment
- 141 participants (actual)
- Lead sponsor
- Healx AIIndustry (pharma, biotech, devices)
- Study start
- 31 Jan 2019 (actual)
- Primary completion
- 4 Jun 2021 (actual)
- Study completion
- 30 Jun 2021 (actual)
- First posted
- 20 Mar 2019 (actual)
- Results
- Posted 18 Jul 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.
Detailed description
This will be an open-label, long-term safety study for evaluation of further treatment with OV101 in subjects with AS who have completed previous Ovid studies (OV101-15-001 or OV101-16-001). There will be no placebo treatment. As this study will enroll subjects who have completed previous AS studies for different periods of time before entering this study, subjects will be required to complete screening and baseline visits before receiving OV101 under this protocol.The secondary objective of this study is to evaluate the long-term efficacy of OV101 treatment assessed by changes in behavior, sleep, and functioning in individuals with AS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OV101 | Experimental | Drug: OV101once daily at bedtime (gaboxadol) |
Who may qualify
- Ages
- 13 to 49 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
- Is male or female and 13 to 49 years old (inclusive) at the time of inclusion in the OV101-15-001 or OV101-16-001 study.
- Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
- Has an LAR/caregiver capable of providing informed consent and able to attend all scheduled study visits, oversee the administration of study drug, and provide feedback regarding the subject's symptoms and performance as described in the protocol.
- Provides assent to the protocol (to the extent possible and in accordance with local institutional review board (IRB) and regulatory requirements) and has an LAR/caregiver who will provide written informed consent. Subjects providing assent must do so at the same visit as LAR/caregiver written informed consent is provided.
Key exclusion criteria (10 listed)
- Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
- Has a concomitant disease (eg, gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or any clinically significant finding at Screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in this study.
- Has poorly controlled seizures defined as > 3 seizures lasting < 3 minutes per week or > 1 seizure episode lasting more than 3 minutes per week or as per medical monitor judgment.
- Has clinically significant clinical laboratory abnormalities or vital signs at the time of screening (eg, alanine aminotransferase or aspartate aminotransferase > 2.5 × upper limit of normal; total bilirubin or creatinine > 1.5 × upper limit of normal). Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
- Current use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep within the 4 weeks prior to Day 1. Benzodiazepines administered for situational anxiety related to occasional procedures or events are permitted.
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 |
|---|---|---|
| OV101Also: gaboxadolEach subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime. | Drug | OV101 |
Primary outcome
Incidence of Participants Experiencing Adverse Events in Active Treatment Group
Time frame: Change from baseline to Week 39
Safety assessments related to the primary study objective of evaluating the safety and tolerability of OV101, including serious adverse events (SAEs) and adverse events (AEs) leading to study discontinuation, as assessed by the number of participants who experienced at least one adverse event.
1 secondary outcome
To Evaluate the Long-term Efficacy of OV101 Treatment as Assessed by Changes in Behavior in Study Participants With AS Who Were at Least 4 Years Old.
Time frame: change from baseline to 12 wks and baseline to early termination of the study at 39 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33395098Heussler HS. Emerging Therapies and challenges for individuals with Angelman syndrome. Curr Opin Psychiatry. 2021 Mar 1;34(2):123-128. doi: 10.1097/YCO.0000000000000674.
- Individual participant dataSharing planned: No
Related trials for Angelman Syndrome
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REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
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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.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing OV101 in people with Angelman Syndrome, run by Healx AI at 11 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 10 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: stopped early, as of the sponsor's last update on 18 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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. 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 18 Jul 2025). 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 Participants Experiencing Adverse Events in Active Treatment Group (time frame: Change from baseline to Week 39). 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 NCT03882918, 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 18 Jul 2025.