Trial · NCT03882918
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
NCT03882918, Phase 3, Terminated, Healx AI; results first posted 2025-07-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome. Trials, record NCT03882918. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03882918
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
- NCT number
- NCT03882918
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 141 participants, actual
- Lead sponsor
- Healx AI
- Conditions
- Angelman Syndrome
- Interventions
- OV101
- First posted
- 2019-03-20
- Last updated
- 2025-07-18
- Results first posted
- 2025-07-18
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence of Participants Experiencing Adverse Events in Active Treatment Group | 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. |
| Secondary | 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. | change from baseline to 12 wks and baseline to early termination of the study at 39 weeks | 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. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence of Participants Experiencing Adverse Events in Active Treatment Group | Change from baseline to Week 39 | One participant completed the study; all other participants did not complete the study due to the early termination of the study by the Sponsor. | participants | NUMBER |
| SECONDARY | 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. | change from baseline to 12 wks and baseline to early termination of the study at 39 weeks | 132 participants were evaluated using the ABC-I. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence of Participants Experiencing Adverse Events in Active Treatment Group | OV101 | 1 | participants | ||
| 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. | OV101 | Week 12 | -0.7 | Standard Deviation 1.5 | score on a scale |
| 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. | OV101 | Week 39 | -0.5 | Standard Deviation 1.3 | score on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | OV101 | 141 | |
| Overall Study | COMPLETED | OV101 | 1 | |
| Overall Study | NOT COMPLETED | OV101 | 140 |
Source ClinicalTrials.gov
Adverse events16
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 | Events |
|---|---|---|---|---|---|---|
| serious | Cardiac arrest | Cardiac disorders | OV101 | 1 | 141 | 1 |
| serious | Gastritis | Gastrointestinal disorders | OV101 | 1 | 141 | 1 |
| serious | Hematemesis | Gastrointestinal disorders | OV101 | 1 | 141 | 1 |
| serious | Corona virus infection | Infections and infestations | OV101 | 2 | 141 | 2 |
| serious | Device related infection | Infections and infestations | OV101 | 1 | 141 | 1 |
| serious | Gastroenteritis | Gastrointestinal disorders | OV101 | 1 | 141 | 1 |
| serious | Lung infection | Infections and infestations | OV101 | 1 | 141 | 1 |
| serious | Dehydration | Respiratory, thoracic and mediastinal disorders | OV101 | 1 | 141 | 1 |
| serious | Partial Seizures | Nervous system disorders | OV101 | 2 | 141 | 2 |
| serious | Generalized tonic-clonic seizure | Nervous system disorders | OV101 | 1 | 141 | 1 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | OV101 | 0 | 141 |
| Total, other adverse events | OV101 | 0 | 141 |
| Total, serious adverse events | OV101 | 18 | 141 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33395098 | 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.
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/NCT03882918
GET https://conductscience.org/api/trials/v1/NCT03882918/registered