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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.
Two dosing schedules of OV101 (gaboxadol) giving as once daily or twice daily dose will be assessed against placebo.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OV101 regimen 1 | Experimental | OV101 once daily |
|
| OV101 regimen 2 | Experimental | OV101 twice daily |
|
| Placebo | Placebo Comparator | Twice daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OV101 Regimen 1 | Drug |
|
| |
| OV101 regimen 2 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. | 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. | Baseline and Week 12 |
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Inclusion Criteria
Exclusion Criteria
Other protocol-defined inclusion/exclusion criteria may apply.
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| Name | Affiliation | Role |
|---|---|---|
| Amit Rakhit, MD, MBA | Healx AI | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ovid Therapeutics Investigative Site | Phoenix | Arizona | United States | |||
| Ovid Therapeutics Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33443117 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Giving twice daily |
| FG001 | OV101 QD | OV101, once daily (15mg night) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan: Study Protocol | Oct 24, 2017 |
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| Drug |
|
|
| Placebo | Other |
|
| San Diego |
| California |
| United States |
| Ovid Therapeutics Investigative Site | Gainesville | Florida | United States |
| Ovid Therapeutics Investigative Site | Tampa | Florida | United States |
| Ovid Therapeutics Investigative Site | Atlanta | Georgia | United States |
| Ovid Therapeutics Investigative Site | Chicago | Illinois | United States |
| Ovid Therapeutics Investigative Site | Boston | Massachusetts | United States |
| Ovid Therapeutics Investigative Site | Lexington | Massachusetts | United States |
| Ovid Therapeutics Investigative Site | Cincinnati | Ohio | United States |
| Ovid Therapeutics Investigative Site | Media | Pennsylvania | United States |
| Ovid Therapeutics Investigative Site | Greenwood | South Carolina | United States |
| Ovid Therapeutics Investigative Site | Nashville | Tennessee | United States |
| Ovid Therapeutics Investigative Site | Ramat Gan | Israel |
| FG002 |
| OV101 BID |
OV101, twice daily (10mg morning, 15mg night) |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo BID Evening and Morning |
| BG001 | OV101 QD | OV101 15 mg Evening Placebo in the Morning |
| BG002 | OV101 BID | OV101 15 mg Evening OV101 10 mg Morning |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Gender | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Posted | Count of Participants | Participants | Baseline and Week 12 |
|
|
| ||||||||||||||||||||||||||||||||||||
| Primary | 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. | Posted | Count of Participants | Participants | Baseline and Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Primary | 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. | Posted | Count of Participants | Participants | Baseline and Week 12 |
|
|
12 Weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo Morning and Evening | 0 | 29 | 0 | 29 | 13 | 29 |
| EG001 | OV101 QD | OV101 15mg Evening | 0 | 29 | 1 | 29 | 18 | 29 |
| EG002 | OV101 BID | OV101 15 mg Evening OV101 10 mg Morning | 0 | 29 | 1 | 29 | 19 | 29 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seizure | Nervous system disorders | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Non-systematic Assessment |
| ||
| General disorders and administration site conditions | Nervous system disorders | Non-systematic Assessment |
| ||
| Fatigue | General disorders | Non-systematic Assessment |
| ||
| Decreased appetite | Metabolism and nutrition disorders | Non-systematic Assessment |
| ||
| Somnolence | Nervous system disorders | Non-systematic Assessment |
| ||
| Myoclonus | Nervous system disorders | Non-systematic Assessment |
| ||
| Seizure | Nervous system disorders | Non-systematic Assessment | Seizures include both Seizures and Myclonic jerks, both of which were balanced across all treatment groups. |
| |
| Lethargy | Nervous system disorders | Non-systematic Assessment |
| ||
| Irritability | Psychiatric disorders | Non-systematic Assessment |
| ||
| Aggression | Psychiatric disorders | Non-systematic Assessment |
| ||
| Insomnia | Psychiatric disorders | Non-systematic Assessment |
| ||
| Agitation | Psychiatric disorders | Non-systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Non-systematic Assessment |
| ||
| Sleep disorder | Psychiatric disorders | Non-systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
| ||
| Myoclonic epilepsy | Nervous system disorders | Non-systematic Assessment |
|
Publication
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Adera Matthews, MD | Ovid Therapeutics | 570 620 6290 | madera@ovidrx.com |
| Mar 3, 2022 |
| Prot_SAP_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan: Statistical Analysis Plan document | Jun 27, 2018 | Mar 3, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D017204 | Angelman Syndrome |
| ID | Term |
|---|---|
| D009069 | Movement Disorders |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D025063 | Chromosome Disorders |
| D030342 | Genetic Diseases, Inborn |
| D000096803 | Imprinting Disorders |
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| ID | Term |
|---|---|
| C015542 | gaboxadol |
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| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Famale |
|
| Title | Measurements |
|---|---|
|
| Any Moderate TEAE |
|
| Any Severe TEAE |
|
| Any Life-Threatening TEAE |
|
| Drug-related TEAE |
|
| Any Serious TEAE |
|
| Any TEAE leading to Dose Change or Interruption |
|
| Any TEAE Leading to Study Withdrawal |
|
|
|