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Autism Spectrum Disorder

35
Pipeline Programs
30
Companies
50
Clinical Trials
12 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
10
4
14
4
2
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

AbbVie
VRAYLARApproved
cariprazine
AbbVie
Atypical Antipsychotic [EPC]oral2015
936M Part D

Competitive Landscape

39 companies ranked by most advanced pipeline stage

AbbVie
AbbVieNORTH CHICAGO, IL
2 programs
1
1
1
CariprazinePhase 31 trial
CariprazinePhase 11 trial
Active Trials
NCT04382885Completed24Est. Dec 2021
NCT05439616Completed161Est. Oct 2024
Neurim Pharmaceuticals
Neurim PharmaceuticalsIsrael - Tel Aviv
1 program
1
MelatoninPhase 31 trial
Active Trials
NCT04233502Withdrawn0Est. Dec 2022
Human BioSciences
Human BioSciencesWV - Martinsburg
23 programs
1
1
3
2
N acetyl cysteinePhase 2/31 trial
N-acetylcysteinePhase 2/31 trial
AmphetaminePhase 21 trial
Folinic AcidPhase 21 trial
N-AcetylcysteinePhase 21 trial
+18 more programs
Active Trials
NCT03873831Completed72Est. Apr 2021
NCT00947700Completed100Est. Jun 2013
NCT02725619Completed70Est. Oct 2022
+18 more trials
Innovation Pharmaceuticals
3 programs
1
1
NTI164Phase 2/31 trial
FEN164Phase 1/21 trial
Visual perspective taking trainingN/A1 trial
Active Trials
NCT06196060Unknown100Est. Sep 2024
NCT05516407Unknown18Est. Sep 2022
NCT05626959Unknown54Est. Nov 2023
Neurotech Pharmaceuticals
2 programs
1
1
NTI164Phase 2/3
FEN164Phase 1/2
Angeles Therapeutics
1
2
AZD7325Phase 21 trial
Behavioral TherapyPhase 21 trial
cognitive behavioral therapyPhase 1/21 trial
ABC-CT Pre-School Feasibility StudyN/A1 trial
Autism Biomarker Consortium for Clinical TrialsN/A1 trial
+15 more programs
Active Trials
NCT06413316Completed50Est. Jun 2025
NCT02996669Completed399Est. May 2019
NCT04841525Completed265Est. Aug 2022
+17 more trials
Colorado Therapeutics
Colorado TherapeuticsCO - Louisville
14 programs
2
Ferrous sulfatePhase 21 trial
Oral cannabidiol 100mg/mLPhase 21 trial
Behavioral skills trainingN/A1 trial
Equine-assisted Occupational TherapyN/A1 trial
Facing Your Fears - School BasedN/A1 trial
+9 more programs
Active Trials
NCT03079765Completed12Est. May 2019
NCT03493347Completed8Est. Dec 2018
NCT03685474Completed389Est. Jun 2020
+11 more trials
Young BioPharma
Young BioPharmaMA - Lowell
3 programs
1
ReconnectPhase 21 trial
Focused Acceptance and Commitment TherapyN/A1 trial
Pai.ACT GroupN/A1 trial
Active Trials
NCT06262646Completed254Est. Jun 2025
NCT06086951Recruiting60Est. Jun 2025
NCT02543203Completed28Est. Mar 2018
Biocodex
BiocodexFrance - Gentilly
2 programs
2
FMT ProtocolPhase 21 trial
S. boulardii CNCMPhase 21 trial
Active Trials
NCT07105267Not Yet Recruiting5Est. Apr 2027
NCT06568588Active Not Recruiting66Est. Aug 2026
Finch Therapeutics
1 program
1
CP101Phase 21 trial
Active Trials
NCT03829878Withdrawn0Est. Dec 2021
Yamo Pharmaceuticals
1 program
1
L1-79Phase 21 trial
Active Trials
NCT05067582Completed58Est. Jun 2024
Aardvark Therapeutics
1
Low Dose ARD-501Phase 21 trial
Active Trials
NCT06126653Completed5Est. Jan 2025
Essential Pharmaceuticals
1
ReconnectPhase 2
Genentech
4 programs
4
BalovaptanPhase 11 trial
BalovaptanPhase 11 trial
BalovaptanPhase 11 trial
RO7017773Phase 11 trial
Active Trials
NCT04049578Terminated2Est. May 2020
NCT04156646Completed16Est. Jan 2020
NCT03912350Withdrawn0Est. Oct 2022
+1 more trials
STALICLA
STALICLASwitzerland - Geneva
3 programs
1
STP1Phase 11 trial
Characterization of a Clinical Subpopulation in Children With Autism Spectrum DisorderN/A1 trial
ObservationN/A1 trial
Active Trials
NCT04600882Withdrawn0Est. Apr 2021
NCT04273087Completed236Est. Oct 2022
NCT04644003Completed12Est. Jan 2022
Intra-Cellular Therapies
1 program
1
Lumateperone 10.5 mg capsulePhase 11 trial
Active Trials
NCT06557902Recruiting26Est. Jul 2026
Sandoz
SandozAustria - Kundl
1 program
1
Omalizumab Injection [Xolair]Phase 1
Jiuzhitang Maker Cell Technology
1 program
1
hBMMSCPhase 11 trial
Active Trials
NCT07177677Not Yet Recruiting42Est. Aug 2028
Oregon Therapeutics
AASPIRE Healthcare ToolkitN/A1 trial
Autism ALERTN/A
Autism ALERTN/A
ONLINEN/A1 trial
Opt-In Early Latinx interventionN/A1 trial
+2 more programs
Active Trials
NCT03234608Completed244Est. Dec 2019
NCT05582655Unknown80Est. Dec 2024
NCT06908187Not Yet Recruiting120Est. Dec 2028
+1 more trials
Jerusalem Pharmaceuticals
Jerusalem PharmaceuticalsIsrael - Ramallah
3 programs
"Parent training for disruptive behaviors" manualN/A1 trial
"Parentship"- a multi dimensional intervention program to improve parental resilienceN/A1 trial
Parent interventionN/A1 trial
Active Trials
NCT04097457Unknown100Est. Jan 2021
NCT03361787Completed10Est. Sep 2019
NCT05879705Unknown50Est. Sep 2023
Jazz Pharmaceuticals
Jazz PharmaceuticalsCA - Palo Alto
3 programs
CannabidivarinPHASE_21 trial
GWP42003-PPHASE_21 trial
GWP42006PHASE_21 trial
Active Trials
NCT03202303Recruiting100Est. Feb 2027
NCT04745026Completed103Est. Dec 2023
NCT03849456Terminated1Est. May 2020
Genomics
GenomicsUK - Oxford
2 programs
14-Day Device MonitoringN/A1 trial
FlorePHASE_11 trial
Active Trials
NCT07504224Completed40Est. Jan 2026
NCT04655326Completed296Est. Jul 2025
Providence Therapeutics
2 programs
Autism ALERTN/A1 trial
Autism ALERTN/A1 trial
Active Trials
NCT04861740Completed148Est. Jan 2024
NCT06246890Active Not Recruiting500Est. Jan 2028
Shield Therapeutics
Shield TherapeuticsMA - Wellesley
2 programs
MINDful TIMEN/A1 trial
Play-based assessmentN/A1 trial
Active Trials
NCT05685589Completed42Est. Apr 2024
NCT05153551Completed60Est. Oct 2022
Roche
RocheSTAVANGER NORWAY, Norway
2 programs
BalovaptanPHASE_1
RO7017773PHASE_1
MapLight Therapeutics
MapLight TherapeuticsMA - Burlington
2 programs
ML-004PHASE_21 trial
ML-004PHASE_21 trial
Active Trials
NCT05081245Active Not Recruiting150Est. Nov 2026
NCT05889273Enrolling By Invitation120Est. Dec 2027
ProgenaBiome
1 program
Fecal Microbiota Transplant; VancomycinN/A1 trial
Active Trials
NCT04878718No Longer Available
UNION therapeutics
ImPACTN/A3 trials
Active Trials
NCT07528066Recruiting200Est. Mar 2027
NCT07541924Recruiting200Est. May 2026
NCT03323918Completed56Est. Nov 2021
Seed Therapeutics
Seed TherapeuticsNY - New York
1 program
Launching! to AdulthoodN/A1 trial
Active Trials
NCT06107972Active Not RecruitingEst. Dec 2025
Carrick Therapeutics
Mente Autism™N/A1 trial
Active Trials
NCT02773303Completed83Est. Dec 2017

+9 more companies

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
City Therapeuticsamitriptyline
AbbVieCariprazine
Vanda PharmaceuticalsTasimelteon Oral Capsule, Tasimelteon Liquid Suspension
Neurim PharmaceuticalsMelatonin
Human BioSciencesN-acetylcysteine
Human BioSciencesN acetyl cysteine
Innovation PharmaceuticalsNTI164
BiocodexFMT Protocol
Aardvark TherapeuticsLow Dose ARD-501
BiocodexS. boulardii CNCM
MapLight TherapeuticsML-004
MapLight TherapeuticsML-004
Yamo PharmaceuticalsL1-79
Colorado TherapeuticsOral cannabidiol 100mg/mL
Jazz PharmaceuticalsGWP42003-P

Showing 15 of 49 trials with date data

Clinical Trials (50)

Total enrollment: 3,784 patients across 50 trials

Amitriptyline for Repetitive Behaviors in Autism Spectrum Disorders

Start: Nov 2023Est. completion: May 2025
Phase 3Terminated

Study of Cariprazine Oral Capsules or Solution to Assess Adverse Events and Change in Irritability Due to Autism Spectrum Disorder (ASD) in Participants Aged 5-17 Years With ASD

Start: Jul 2022Est. completion: Oct 2024161 patients
Phase 3Completed
NCT05361707Vanda PharmaceuticalsTasimelteon Oral Capsule, Tasimelteon Liquid Suspension

Evaluating the Effects of Tasimelteon in Individuals With Autism Spectrum Disorder (ASD) and Sleep Disturbances

Start: Jul 2021Est. completion: Jul 2025100 patients
Phase 3Unknown

Efficacy and Safety of Slenyto for Insomnia in Children With ASD

Start: Sep 2020Est. completion: Dec 20220
Phase 3Withdrawn

Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Open Label

Start: Mar 2024Est. completion: Jan 2029144 patients
Phase 2/3Enrolling By Invitation

Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Trial

Start: Apr 2023Est. completion: Jul 2028144 patients
Phase 2/3Recruiting

Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder

Start: Nov 2022Est. completion: Nov 202354 patients
Phase 2/3Unknown

Open Label Phase IIa Trial Evaluating the Effect and Safety of Transplantation of Fecal Microbiota in Children With Autism Spectrum Disorders and Gastrointestinal Symptoms

Start: Sep 2025Est. completion: Apr 20275 patients
Phase 2Not Yet Recruiting

A Study to Evaluate ARD-501 in Patients With Autism Spectrum Disorder

Start: Aug 2024Est. completion: Jan 20255 patients
Phase 2Completed
NCT06568588BiocodexS. boulardii CNCM

Effects of Probiotic Consumption in Children With Autism Spectrum Disorder (ASD)

Start: Jan 2024Est. completion: Aug 202666 patients
Phase 2Active Not Recruiting

ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)

Start: Jun 2023Est. completion: Dec 2027120 patients
Phase 2Enrolling By Invitation

ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)

Start: Sep 2022Est. completion: Nov 2026150 patients
Phase 2Active Not Recruiting

A 12-Week Crossover Study to Assess the Efficacy, Safety and Tolerability of L1-79 in Subjects Aged 12-21 Years With Autism Spectrum Disorder

Start: Jan 2022Est. completion: Jun 202458 patients
Phase 2Completed
NCT04520685Colorado TherapeuticsOral cannabidiol 100mg/mL

CASCADE: CAnnabidiol Study in Children With Autism Spectrum DisordEr

Start: Dec 2021Est. completion: Dec 202486 patients
Phase 2Active Not Recruiting

Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder

Start: May 2021Est. completion: Dec 2023103 patients
Phase 2Completed

Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Single-dose

Start: Feb 2021Est. completion: Jan 202924 patients
Phase 2Recruiting

Leucovorin for the Treatment of Language Impairment in Children With Autism Spectrum Disorder

Start: Aug 2020Est. completion: Jul 202656 patients
Phase 2Active Not Recruiting

Efficacy, Safety, and Tolerability Study of Oral Full-Spectrum MicrobiotaTM (CP101) in Subjects With Autism Spectrum Disorder and Associated GI Symptoms (SPROUT)

Start: May 2020Est. completion: Dec 20210
Phase 2Withdrawn

Safety and Tolerability of Cannabidivarin (CBDV) in Children and Young Adults With Autism Spectrum Disorder

Start: Jan 2020Est. completion: May 20201 patients
Phase 2Terminated

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

Start: Apr 2019Est. completion: Feb 2027100 patients
Phase 2Recruiting

Impact of Combined Medication and Behavioral Treatment for ASD & ADHD

Start: Dec 2018Est. completion: Apr 202118 patients
Phase 2Terminated

Essential Oils for Enhancing QOL in ASD

Start: Jul 2015Est. completion: Mar 201828 patients
Phase 2Completed

Targeting GABA-A for the Treatment of Social Disability in Young Adults With Autism Spectrum Disorders: A Phase II Proof of Mechanism Trial

Start: Oct 2013Est. completion: Jul 201540 patients
Phase 2Completed

Iron Treatment of Sleep Disorders in Children With Autism Spectrum Disorder

Start: Dec 2012Est. completion: Aug 201524 patients
Phase 2Completed

Combination Treatment for Augmenting Language in Children With ASD

Start: Jun 2012Est. completion: Dec 201958 patients
Phase 2Completed

Safety and Efficacy of Oral Full-Spectrum Medicinal Cannabis Plant Extract in Children With Autism Spectrum Disorder.

Start: Apr 2021Est. completion: Sep 202218 patients
Phase 1/2Unknown

Combination Probiotic: BB-12 With LGG (Different Doses) in Treating Children With Autism Spectrum Disorder

Start: May 2016Est. completion: Dec 202570 patients
Phase 1/2Recruiting
NCT00789932Angeles Therapeuticscognitive behavioral therapy

Cognitive Behavioral Therapy for Core Autism Symptoms in School-Aged Children: A Randomized, Controlled Trial

Start: Sep 2008Est. completion: Dec 201120 patients
Phase 1/2Completed

A Study of Mesenchymal Stem Cell in the Treatment of Autism Spectrum Disorder

Start: Sep 2025Est. completion: Aug 202842 patients
Phase 1Not Yet Recruiting
NCT06557902Intra-Cellular TherapiesLumateperone 10.5 mg capsule

Safety, Tolerability and Pharmacokinetics of Lumateperone in Pediatric Patients With Autism Spectrum Disorder

Start: May 2024Est. completion: Jul 202626 patients
Phase 1Recruiting

The Effect of Hepatic Impairment on the Pharmacokinetics of Balovaptan

Start: Jun 2022Est. completion: Oct 20220
Phase 1Withdrawn

Safety, Tolerability and Pharmacokinetics Study of STP1 in a Subgroup of Patients With Autism Spectrum Disorder (ASD)

Start: Dec 2020Est. completion: Jan 202212 patients
Phase 1Completed
NCT05491720Human BioSciencesRisperiDONE 1 MG/ML

Electrical Brain Stimulation and Pharmacological Treatments in Autism Spectrum Disorder

Start: Aug 2020Est. completion: Aug 202245 patients
Phase 1Completed

Cariprazine Pediatric ASD PK Study

Start: Jun 2020Est. completion: Dec 202124 patients
Phase 1Completed

A Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Balovaptan in Children With Autism Spectrum Disorder

Start: Dec 2019Est. completion: May 20202 patients
Phase 1Terminated

A Study to Investigate the Effect of Esomeprazole and the Effect of Food on the Pharmacokinetics of Balovaptan in Healthy Volunteers

Start: Nov 2019Est. completion: Jan 202016 patients
Phase 1Completed

Open Label, Adaptive, Parallel Group PET Study Using RO7017773 And [11C] RO15-4513

Start: Apr 2018Est. completion: Jun 20186 patients
Phase 1Completed
NCT04878718ProgenaBiomeFecal Microbiota Transplant; Vancomycin

Vancomycin and Fecal Microbiota Transplant in a Single Patient With Autism Spectrum Disorder

N/ANo Longer Available

Downstaging and RATS After Neo-Chemo-IO: Impact on Surgical Outcomes in NSCLC

Start: Mar 2026Est. completion: Mar 2027200 patients
N/ARecruiting

Fecal Microbiota Transplantation in Children With Autism Spectrum Disorder and Gastrointestinal Symptoms

Start: Jan 2026Est. completion: Mar 202720 patients
N/ANot Yet Recruiting

The Impact of Image-Assisted Colonoscope on Patient Experience, Physician Workload, and Examination Quality

Start: Dec 2025Est. completion: May 2026200 patients
N/ARecruiting
NCT06908187Oregon TherapeuticsOpt-In Early Latinx intervention

Opt-In Early Pilot Study

Start: Dec 2025Est. completion: Dec 2028120 patients
N/ANot Yet Recruiting

pBFS Guided aiTBS Over Language Network for ASD Child

Start: Apr 2025Est. completion: Jun 2027150 patients
N/ARecruiting
NCT06772324ShionogiNORSPAN Buprenorphine Transdermal Patchs for Very-low dose group

An Exploratory Clinical Study of a Low-dose Buprenorphine Patch for the Treatment of Autism Spectrum Disorders

Start: Jan 2025Est. completion: Sep 202660 patients
N/ANot Yet Recruiting

Hybrid Effectiveness-Implementation Trial of an Executive Functioning Intervention for Children's Mental Health Services

Start: Dec 2024Est. completion: Aug 2029672 patients
N/ARecruiting
NCT06502054EppendorfParenting with an Autism Spectrum Disorder

Parenting with an Autism Spectrum Disorder

Start: Sep 2024Est. completion: Oct 2025184 patients
N/ARecruiting
NCT07504224Genomics14-Day Device Monitoring

ASD Wearables Feasibility Study

Start: Jul 2024Est. completion: Jan 202640 patients
N/ACompleted

STRW-T Intervention for Autistic Adolescents in 11th and 12th Grade

Start: Jul 2024Est. completion: Dec 2029192 patients
N/ARecruiting
NCT06413316Angeles TherapeuticsABC-CT Pre-School Feasibility Study

ABC-CT Pre-School Feasibility Study

Start: Jul 2024Est. completion: Jun 202550 patients
N/ACompleted
NCT06506097Human BioSciencesFamily-school Partnership Intervention

Family-school Partnership Intervention for Early Childhood Education

Start: Jul 2024Est. completion: Jul 202670 patients
N/ARecruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
12 actively recruiting trials targeting 3,784 patients
30 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.