Trial reportNeurology2021
The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome.
Registry-linked trialAbstract readRandomized Controlled Trial
In one paragraph
Trial report in Neurology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02996305 (A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial), which is not on this map. Cited by 13 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed, 2 pooled it
–field-weighted citation impact
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Phase 2 Adult and Adolescent Angelman Syndrome Clinical Trial: A Randomized, Double-Blind, Safety and Efficacy Study of Gaboxadol
Who cites it
13 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Neural excitation/inhibition imbalance and neurodevelopmental pathology in human copy number variant syndromes: a systematic review.Journal of neurodevelopmental disorders · 2025Pooled it
- Evidence Synthesis of Gene Therapy and Gene Editing from Different Disorders-Implications for Individuals with Rett Syndrome: A Systematic Review.International journal of molecular sciences · 2023Pooled it
- Health-related quality of life and medication use among individuals with Angelman syndrome.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2023Trial
- Molecular Basis of GABA Aminotransferase Inhibition in Epilepsy: Structure, Mechanisms, and Drug Development.Current issues in molecular biology · 2025Review
- Development of the Angelman syndrome video assessment: quantifying meaningful change.Journal of neurodevelopmental disorders · 2025Article
- Methodological challenges in outcomes research for early-trials for implementation of new therapies in neuropediatric rare diseases.Orphanet journal of rare diseases · 2025Article
- Courtship and distress ultrasonic vocalizations are altered in a mouse model of Angelman syndrome.Journal of neurodevelopmental disorders · 2025Article
- The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial.Nature medicine · 2025Article
- Prader-Willi and Angelman Syndromes: Mechanisms and Management.The application of clinical genetics · 2023Review
- Promising therapeutic aspects in human genetic imprinting disorders.Clinical epigenetics · 2022Review
- A multidisciplinary approach and consensus statement to establish standards of care for Angelman syndrome.Molecular genetics & genomic medicine · 2022Review
- Emerging Gene and Small Molecule Therapies for the Neurodevelopmental Disorder Angelman Syndrome.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021Review
- Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study.Frontiers in pharmacology · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
12 authors.
Lynne M BirdFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Cesar Ochoa-LubinoffFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Wen-Hann TanFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Gali HeimerFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Raun D MelmedFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Amit RakhitFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Jeannie VisootsakFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Matthew J DuringFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Christina HolcroftFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Rebecca D BurdineFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Alexander KolevzonFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston. alexander.kolevzon@mssm.edu.
Ronald L ThibertFrom the University of California, San Diego (L.M.B.); Rady Children's Hospital (L.M.B.), San Diego, CA; Division of Developmental-Behavioral Pediatrics (C.O.-L.), Rush University Medical Center, Chicago, IL; Division of Genetics and Genomics (W.-H.T.), Boston Children's Hospital, Harvard Medical School, MA; Pediatric Neurology Unit (G.H.), Safra Children's Hospital, the Sheba Medical Center, Ramat Gan; The Sackler School of Medicine (G.H.), Tel Aviv University, Israel; Southwest Autism Research and Resource Center (R.D.M.), Phoenix, AZ; Ovid Therapeutics Inc. (A.R., M.J.D.); Neurogene (J.V.), New York, NY; Prometrika, LLC (C.H.), Cambridge, MA; Department of Molecular Biology (R.D.B.), Princeton University, NJ; Seaver Autism Center for Research and Treatment, Department of Psychiatry (A.K.), Icahn School of Medicine at Mount Sinai, New York, NY; and Angelman Syndrome Clinic, Department of Neurology (R.L.T.), Massachusetts General Hospital, Boston.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectiveTo evaluate safety and tolerability and exploratory efficacy end points for gaboxadol (OV101) compared with placebo in individuals with Angelman syndrome (AS).
methodsGaboxadol is a highly selective orthosteric agonist that activates δ-subunit-containing extrasynaptic γ-aminobutyric acid type A (GABA
resultsEighty-eight individuals were randomized. Of 87 individuals (aged 13-45 years) who received at least 1 dose of study drug, 78 (90%) completed the study. Most adverse events (AEs) were mild to moderate, and no life-threatening AEs were reported. Efficacy of gaboxadol, as measured by CGI-I improvement in an exploratory analysis, was observed in gaboxadol qd vs placebo (
conclusionAfter 12 weeks of treatment, gaboxadol was found to be generally well-tolerated with a favorable safety profile. The efficacy as measured by the AS-adapted CGI-I scale warrants further studies. CLINICALTRIALSGOV IDENTIFIER: NCT02996305. CLASSIFICATION OF EVIDENCE: This study provides Class I evidence that, for individuals with AS, gaboxadol is generally safe and well-tolerated.
Indexed as
AdolescentAdultAngelman SyndromeDose-Response Relationship, DrugDouble-Blind MethodDrug Administration ScheduleFemaleGABA AgonistsHumansIsoxazolesMaleMiddle AgedTreatment OutcomeYoung AdultGABA AgonistsgaboxadolIsoxazoles
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390
Registered trials
NCT02996305ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.