gaboxadol has been researched along with Angelman Syndrome in 4 studies
gaboxadol: GABA agonist; inhibitor of GABA uptake systems; structure
Angelman Syndrome: A syndrome characterized by multiple abnormalities, MENTAL RETARDATION, and movement disorders. Present usually are skull and other abnormalities, frequent infantile spasms (SPASMS, INFANTILE); easily provoked and prolonged paroxysms of laughter (hence happy); jerky puppetlike movements (hence puppet); continuous tongue protrusion; motor retardation; ATAXIA; MUSCLE HYPOTONIA; and a peculiar facies. It is associated with maternal deletions of chromosome 15q11-13 and other genetic abnormalities. (From Am J Med Genet 1998 Dec 4;80(4):385-90; Hum Mol Genet 1999 Jan;8(1):129-35)
| Excerpt | Relevance | Reference |
|---|---|---|
| "To evaluate safety and tolerability and exploratory efficacy end points for gaboxadol (OV101) compared with placebo in individuals with Angelman syndrome (AS)." | 9.41 | The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome. ( Bird, LM; Burdine, RD; During, MJ; Heimer, G; Holcroft, C; Kolevzon, A; Melmed, RD; Ochoa-Lubinoff, C; Rakhit, A; Tan, WH; Thibert, RL; Visootsak, J, 2021) |
| "To evaluate safety and tolerability and exploratory efficacy end points for gaboxadol (OV101) compared with placebo in individuals with Angelman syndrome (AS)." | 5.41 | The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome. ( Bird, LM; Burdine, RD; During, MJ; Heimer, G; Holcroft, C; Kolevzon, A; Melmed, RD; Ochoa-Lubinoff, C; Rakhit, A; Tan, WH; Thibert, RL; Visootsak, J, 2021) |
| "Angelman syndrome is a neurodevelopmental disorder caused by loss of function of the UBE3A gene encoding a ubiquitin E3 ligase." | 1.38 | Decreased tonic inhibition in cerebellar granule cells causes motor dysfunction in a mouse model of Angelman syndrome. ( Egawa, K; Fukuda, A; Inoue, K; Kishino, T; Kitagawa, K; Kitagawa, M; Saitoh, S; Takayama, C; Takayama, M, 2012) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 0 (0.00) | 29.6817 |
| 2010's | 1 (25.00) | 24.3611 |
| 2020's | 3 (75.00) | 2.80 |
| Authors | Studies |
|---|---|
| Copping, NA | 1 |
| McTighe, SM | 1 |
| Fink, KD | 1 |
| Silverman, JL | 1 |
| Eisold, A | 1 |
| Bird, LM | 1 |
| Ochoa-Lubinoff, C | 1 |
| Tan, WH | 1 |
| Heimer, G | 1 |
| Melmed, RD | 1 |
| Rakhit, A | 1 |
| Visootsak, J | 1 |
| During, MJ | 1 |
| Holcroft, C | 1 |
| Burdine, RD | 1 |
| Kolevzon, A | 1 |
| Thibert, RL | 1 |
| Egawa, K | 1 |
| Kitagawa, K | 1 |
| Inoue, K | 1 |
| Takayama, M | 1 |
| Takayama, C | 1 |
| Saitoh, S | 1 |
| Kishino, T | 1 |
| Kitagawa, M | 1 |
| Fukuda, A | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol[NCT02996305] | Phase 2 | 88 participants (Actual) | Interventional | 2016-01-31 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
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. (NCT02996305)
Timeframe: Baseline and Week 12
| Intervention | Participants (Count of Participants) | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Any TEAE | Any Mild TEAE | 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 | |
| OV101 BID | 25 | 23 | 9 | 4 | 0 | 19 | 1 | 8 | 3 |
| OV101 QD | 27 | 23 | 15 | 1 | 0 | 18 | 1 | 5 | 0 |
| Placebo | 25 | 23 | 9 | 0 | 0 | 13 | 0 | 5 | 1 |
The Treatment Emergent Adverse Event (TEAEs) Reported by ≥10% of Subjects in Any Treatment Group by Preferred Term, Safety Set. (NCT02996305)
Timeframe: Baseline and Week 12
| Intervention | Participants (Count of Participants) | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| At least 1 TEAE | Vomiting | Somnolence | Irritability | Pyrexia | Upper respiratory tract infection | Aggression | Rash | Seizure | Decreased appetite | Diarrhoea | Nausea | Nasopharyngitis | |
| OV101 BID | 25 | 5 | 3 | 5 | 1 | 1 | 1 | 2 | 3 | 2 | 3 | 4 | 1 |
| OV101 QD | 27 | 5 | 5 | 3 | 7 | 5 | 4 | 3 | 2 | 2 | 1 | 0 | 2 |
| Placebo | 25 | 9 | 5 | 4 | 2 | 4 | 5 | 1 | 0 | 4 | 3 | 3 | 5 |
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. (NCT02996305)
Timeframe: Baseline and Week 12
| Intervention | Participants (Count of Participants) | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| At least 1 Treatment-related TEAE | Nausea | Fatigue | Decreased appetite | Somnolence | Myoclonic epilepsy | Myoclonus | Seizure | Lethargy | Sedation | Tremor | Irritability | Aggression | Insomnia | Agitation | Anxiety | Enuresis | Sleep disorder | Rash | |
| OV101 BID | 19 | 2 | 2 | 2 | 2 | 2 | 2 | 2 | 2 | 1 | 1 | 4 | 1 | 1 | 2 | 2 | 1 | 2 | 0 |
| OV101 QD | 18 | 0 | 1 | 0 | 5 | 1 | 1 | 1 | 0 | 1 | 1 | 2 | 4 | 2 | 1 | 0 | 1 | 0 | 2 |
| Placebo | 13 | 2 | 2 | 2 | 5 | 0 | 0 | 0 | 0 | 0 | 0 | 3 | 2 | 1 | 0 | 0 | 0 | 0 | 0 |
1 review available for gaboxadol and Angelman Syndrome
| Article | Year |
|---|---|
Emerging Gene and Small Molecule Therapies for the Neurodevelopmental Disorder Angelman Syndrome.
Topics: Angelman Syndrome; Animals; Anticonvulsants; Biological Products; Genetic Therapy; Humans; Isoxazole | 2021 |
1 trial available for gaboxadol and Angelman Syndrome
| Article | Year |
|---|---|
The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome.
Topics: Adolescent; Adult; Angelman Syndrome; Dose-Response Relationship, Drug; Double-Blind Method; Drug Ad | 2021 |
2 other studies available for gaboxadol and Angelman Syndrome
| Article | Year |
|---|---|
Upcoming market catalysts in Q3 2020.
Topics: Angelman Syndrome; Azo Compounds; Drug Approval; Fatty Acids; Humans; Isoxazoles; Muscular Atrophy, | 2020 |
Decreased tonic inhibition in cerebellar granule cells causes motor dysfunction in a mouse model of Angelman syndrome.
Topics: Angelman Syndrome; Animals; Cerebellum; Disease Models, Animal; GABA Agonists; GABA Plasma Membrane | 2012 |