Disease · MONDO_0005258

autism spectrum disorder

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. autism spectrum disorder. Diseases, record MONDO_0005258. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0005258

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: autism spectrum disorder; Open Targets
Open Targets ID: MONDO_0005258; Open Targets
Other names: Autism Spectrum Disorders, CHILD DEVELOPMENT DIS PERVASIVE, Child Development Disorders, Pervasive, Development Disorder, Pervasive, Development Disorders, Pervasive, Disorder, Autism Spectrum, Disorder, Pervasive Development, Disorders, Autism Spectrum, Disorders, Pervasive Development, PERVASIVE CHILD DEVELOPMENT DIS, Pervasive Child Development Disorders, Pervasive Development Disorder, Pervasive Development Disorders, Spectrum Disorder, Autism, Spectrum Disorders, Autism, autism spectrum disease, autistic spectrum disorder, pervasive developmental disorder - not otherwise specified, pervasive developmental disorders; Open Targets
Disease Ontology: DOID:0060041; Disease Ontology
NCI Thesaurus: C88412; NCI Thesaurus
Orphanet: ORPHA:106; Orphanet
Highest stage of its drugs: Approval, for 2 of its 123 drugs, from PMDA and TTD; Open Targets

Drugs123

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
OXYTOCIN	CHEMBL395429	Protein	Approval	TTD	35	34	34	Drugs and targets
ARIPIPRAZOLE	CHEMBL1112	Small molecule	Approval	PMDA	9	8	8	Drugs and targets
CANNABIDIOL	CHEMBL190461	Small molecule	Phase 3	ClinicalTrials.gov	12	12	10	Drugs and targets
ARBACLOFEN	CHEMBL301742	Small molecule	Phase 3	ClinicalTrials.gov	8	8	8	Drugs and targets
BUMETANIDE	CHEMBL1072	Small molecule	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
MEMANTINE	CHEMBL807	Small molecule	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
BALOVAPTAN	CHEMBL4297183	Small molecule	Phase 3	ClinicalTrials.gov and TTD	6	4	4	Drugs and targets
LUMATEPERONE	CHEMBL3306803	Small molecule	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets
METHYLPHENIDATE	CHEMBL796	Small molecule	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets
CARIPRAZINE	CHEMBL2028019	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets

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Targets139

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
SLC6A4	Sodium-dependent serotonin transporter	9	Serotonin transporter inhibitor; Sodium-dependent serotonin transporter releasing agent	Phase 3	https://conductscience.org/library/genes/6532	Drugs and targets
SLC6A2	Sodium-dependent noradrenaline transporter	7	Norepinephrine transporter inhibitor; Norepinephrine transporter releasing agent; Sodium-dependent noradrenaline transporter releasing agent	Phase 3	https://conductscience.org/library/genes/6530	Drugs and targets
DRD2	D(2) dopamine receptor	6	D2-like dopamine receptor antagonist; Dopamine D2 receptor antagonist; Dopamine D2 receptor modulator; Dopamine D2 receptor partial agonist	Approval	https://conductscience.org/library/genes/1813	Drugs and targets
GRIN2A	Glutamate [NMDA] receptor	6	Glutamate [NMDA] receptor antagonist; Glutamate [NMDA] receptor negative allosteric modulator; Glutamate [NMDA] receptor subunit epsilon 1 antagonist	Phase 3	https://conductscience.org/library/genes/2903	Drugs and targets
HTR2A	5-hydroxytryptamine receptor 2A	6	Serotonin 2a (5-HT2a) receptor antagonist; Serotonin 2a (5-HT2a) receptor inverse agonist	Approval	https://conductscience.org/library/genes/3356	Drugs and targets
GABRA1	GABA A receptor alpha-1/beta-1/gamma-2	5	GABA A receptor alpha-1/beta-1/gamma-2 positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator	Phase 2	https://conductscience.org/library/genes/2554	Drugs and targets
GABRA2	GABA-A receptor; anion channel	5	GABA receptor alpha-2 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator	Phase 2	https://conductscience.org/library/genes/2555	Drugs and targets
GABRA3	GABA-A receptor; anion channel	5	GABA receptor alpha-3 subunit positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator	Phase 2	https://conductscience.org/library/genes/2556	Drugs and targets
GABRA4	GABA-A receptor; anion channel	5	GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator; Gamma-aminobutyric acid receptor subunit alpha-4/beta3/delta agonist	Phase 2	https://conductscience.org/library/genes/2557	Drugs and targets
GABRB1	GABA A receptor alpha-1/beta-1/gamma-2	5	GABA A receptor alpha-1/beta-1/gamma-2 positive allosteric modulator; GABA-A receptor; anion channel positive allosteric modulator; GABA-A receptor; anion channel positive modulator	Phase 2	https://conductscience.org/library/genes/2560	Drugs and targets

1 to 10 of 139

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Trials218

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 11 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT03179787	Post-Marketing Surveillance Study of Aripiprazole in Patients With Autism		528	2021-09-16	Autism	ARIPIPRAZOLE	Its record names the disease	ClinicalTrials.gov
NCT02901431	A Study to Investigate the Efficacy and Safety of Balovaptan (RO5285119) in Participants With Autism Spectrum Disorder (ASD)	Phase 2	339	2021-02-08	Autism Spectrum Disorder	BALOVAPTAN	Its record names the disease	ClinicalTrials.gov
NCT03504917	A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension	Phase 3	322	2021-05-07	Autism Spectrum Disorder	BALOVAPTAN	Its record names the disease	ClinicalTrials.gov
NCT01944046	Study of Oxytocin in Autism to Improve Reciprocal Social Behaviors	Phase 2	290	2021-01-05	Autism Spectrum Disorders	OXYTOCIN	Its record names the disease	ClinicalTrials.gov
NCT05523895	Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder	Phase 2/3	237	2025-08-24	Irritability Associated With Autism Spectrum Disorder	PIMAVANSERIN	Its record names the disease	ClinicalTrials.gov
NCT03715153	Efficacy and Safety of Bumetanide Oral Liquid Formulation in Children Aged From 2 to Less Than 7 Years Old With Autism Spectrum Disorder.	Phase 3	211	2023-06-05	Autism Spectrum Disorder (ASD)	BUMETANIDE	Its record names the disease	ClinicalTrials.gov
NCT00872898	Study of Pharmacokinetics, Safety, Efficacy, and Tolerability of Memantine in Children With Autism	Phase 2	124	2014-01-14	Autism	MEMANTINE	Its record names the disease	ClinicalTrials.gov
NCT03197922	Treatment of Encopresis in Children With Autism Spectrum Disorders	Phase 3	117	2024-01-31	Encopresis, Autism Spectrum Disorder	GLYCERIN	Its record names the disease	ClinicalTrials.gov
NCT01620060	Lurasidone Pediatric Pharmacokinetics Study	Phase 1	105	2014-07-31	Schizophrenia, Autism	LURASIDONE	Its record names the disease	ClinicalTrials.gov
NCT04299464	A 12-Week Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of RO7017773 in Participants Aged 15-45 Years With Autism Spectrum Disorder (ASD)	Phase 2	104	2025-01-28	Autism Spectrum Disorder (ASD)	ALOGABAT	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials73

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
BUMETANIDE (S95008) vs PLACEBO: Number of Patients With Abnormalities in 12-leads Electrocardiogram (ECG) Parameters	0 of 78	0 of 77	155	NCT03715153	Trial comparisons
GWP42003-P vs Placebo: deaths from any cause	0 of 49	0 of 54	103	NCT04745026	Trial comparisons
GWP42003-P vs Placebo: serious adverse events	2 of 49	1 of 54	103	NCT04745026	Trial comparisons
D-cycloserine vs Placebo: serious adverse events	0 of 34	1 of 33	67	NCT01086475	Trial comparisons
Intranasal Oxytocin (Syntocinon) vs Placebo: deaths from any cause	0 of 30	0 of 30	60	NCT01908205	Trial comparisons
Intranasal Oxytocin (Syntocinon) vs Placebo: serious adverse events	0 of 30	0 of 30	60	NCT01908205	Trial comparisons
Sertraline Active Medication vs Sertraline Liquid Placebo: deaths from any cause	0 of 32	0 of 26	58	NCT02385799	Trial comparisons
Sertraline Active Medication vs Sertraline Liquid Placebo: serious adverse events	1 of 32	0 of 26	58	NCT02385799	Trial comparisons
Syntocinon vs Placebo: deaths from any cause	0 of 28	0 of 28	56	NCT01183221	Trial comparisons
Syntocinon vs Placebo: serious adverse events	0 of 28	0 of 28	56	NCT01183221	Trial comparisons

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Papers from its trials88

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
37445385	RESULT	1	PubMed
35804408	RESULT	1	PubMed
35734582	RESULT	1	PubMed
34593045	RESULT	1	PubMed
33823927	RESULT	1	PubMed
33658971	RESULT	1	PubMed
33578648	RESULT	1	PubMed
32242086	RESULT	1	PubMed
32206584	RESULT	1	PubMed
29625151	RESULT	1	PubMed

1 to 10 of 88

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

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
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

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

For software

Read this disease from software.

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

GET https://conductscience.org/api/diseases/v1/MONDO_0005258

GET https://conductscience.org/api/diseases/v1/MONDO_0005258/drugs?page=2

API reference For AI assistants