Trial · NCT04106557

A Study of OV101 in Individuals With Angelman Syndrome (AS)

NCT04106557, Phase 3, Completed, Ovid Therapeutics Inc.; results first posted 2024-01-08

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of OV101 in Individuals With Angelman Syndrome (AS). Trials, record NCT04106557. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04106557

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of OV101 in Individuals With Angelman Syndrome (AS)
NCT number: NCT04106557
Status: Completed
Phases: Phase 3
Enrollment: 104 participants, actual
Lead sponsor: Ovid Therapeutics Inc.
Conditions: Primary Disease or Condition Being Studied: Angelman Syndrome (AS)
Interventions: Gaboxadol, Placebo
First posted: 2019-09-27
Last updated: 2024-01-08
Results first posted: 2024-01-08

Source ClinicalTrials.gov

Registered outcomes1

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	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).

Source ClinicalTrials.gov

Outcomes1

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	12 weeks	Total of 97 subjects, ages 4-12 years, inclusive. Participants aged 2 to 3 (n=7) did not participate in the scoring.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group14

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	OV101 (Ages 4-12 Years, Inclusive)	1-Very much improved	1	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	Placebo	1-Very much improved	2	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	OV101 (Ages 4-12 Years, Inclusive)	2-Much improved	11	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	Placebo	2-Much improved	11	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	OV101 (Ages 4-12 Years, Inclusive)	3-Minimally improved	11	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	Placebo	3-Minimally improved	11	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	OV101 (Ages 4-12 Years, Inclusive)	4-No change	22	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	Placebo	4-No change	21	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	OV101 (Ages 4-12 Years, Inclusive)	5-Minimally worse	1	Participants
Clinical Global Impressions- Improvement in Angelman Syndrome (CGI-I-AS) Rating at 12 Weeks	Placebo	5-Minimally worse	5	Participants

1 to 10 of 14Source ClinicalTrials.gov

See all 14 results by group

Participant flow9

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	OV101 Once Daily (Weight-based Dosing) Age 4 to 12	47
Overall Study	STARTED	Placebo Once Daily	50
Overall Study	STARTED	OV101 Once Daily (Weight-based Dosing) Age 2 to 3	7
Overall Study	COMPLETED	OV101 Once Daily (Weight-based Dosing) Age 4 to 12	47
Overall Study	COMPLETED	Placebo Once Daily	48
Overall Study	COMPLETED	OV101 Once Daily (Weight-based Dosing) Age 2 to 3	7
Overall Study	NOT COMPLETED	OV101 Once Daily (Weight-based Dosing) Age 4 to 12	0
Overall Study	NOT COMPLETED	Placebo Once Daily	2
Overall Study	NOT COMPLETED	OV101 Once Daily (Weight-based Dosing) Age 2 to 3	0

Source ClinicalTrials.gov

Adverse events51

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Fatigue	General disorders	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	3	47
other	Fatigue	General disorders	Placebo Once Daily	1	50
other	Fatigue	General disorders	OV101 Once Daily (Weight-based Dosing) Age 2-3	0	7
other	Generalized tonic-clonic seizure	General disorders	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	3	47
other	Generalized tonic-clonic seizure	General disorders	Placebo Once Daily	0	50
other	Generalized tonic-clonic seizure	General disorders	OV101 Once Daily (Weight-based Dosing) Age 2-3	0	7
other	Irritability	General disorders	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	3	47
other	Irritability	General disorders	Placebo Once Daily	2	50
other	Irritability	General disorders	OV101 Once Daily (Weight-based Dosing) Age 2-3	0	7
other	Somnolence	General disorders	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	3	47

1 to 10 of 51Source ClinicalTrials.gov

See all 51 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	0	47
Total, all-cause mortality	Placebo Once Daily	0	50
Total, all-cause mortality	OV101 Once Daily (Weight-based Dosing) Age 2-3	0	7
Total, other adverse events	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	31	47
Total, other adverse events	Placebo Once Daily	22	50
Total, other adverse events	OV101 Once Daily (Weight-based Dosing) Age 2-3	4	7
Total, serious adverse events	OV101 Once Daily (Weight-based Dosing) Other Name:Gaboxadol	1	47
Total, serious adverse events	Placebo Once Daily	0	50
Total, serious adverse events	OV101 Once Daily (Weight-based Dosing) Age 2-3	0	7

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37639777	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04106557

GET https://conductscience.org/api/trials/v1/NCT04106557/events?page=2

API reference For AI assistants