Autism

Pipeline Programs

Companies

Clinical Trials

7 recruiting

Approved Products

Pipeline by Development Stage

Preclinical

Phase 1

Phase 1/2

Phase 2

Phase 2/3

Phase 3

On Market

Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule

3100%

+ 59 programs with unclassified modality

On Market (5)

Approved therapies currently available

ABILIFY ASIMTUFIIApproved

aripiprazole

Otsuka

Atypical Antipsychotic [EPC]intramuscular2023

13M Part D

ABILIFYApproved

aripiprazole

Otsuka

Atypical Antipsychotic [EPC]oral2002

12M Part D

MEZOFYApproved

aripiprazole

Otsuka

oral2025

OPIPZAApproved

aripiprazole

Otsuka

Atypical Antipsychotic [EPC]oral2024

ABILIFY MAINTENA KITApproved

aripiprazole

Otsuka

intramuscular2013

Competitive Landscape

24 companies ranked by most advanced pipeline stage

Pre

P1/2

P2/3

Mkt

OtsukaJapan - Tokushima

2 programs

AripiprazolePhase 4Small Molecule1 trial

Aripiprazole oral productN/A1 trial

Active Trials

NCT03179787Completed528Est. Jul 2020

NCT02069977Unknown79Est. Feb 2016

BiocorpFrance - Issoire

2 programs

Gammaplex 5%Phase 41 trial

human cord blood mononuclear cellsPhase 1/21 trial

Active Trials

NCT01343511Completed37Est. May 2011

NCT02199925Unknown30Est. Jul 2015

Human BioSciencesWV - Martinsburg

16 programs

ValproatePhase 31 trial

secretin, synthetic porcinePhase 31 trial

VasopressinPhase 2/31 trial

RisperidonePhase 2

Caregiver joint attention interventionPhase 1/21 trial

+11 more programs

Active Trials

NCT06705907Recruiting224Est. Aug 2029

NCT02998684Completed25Est. Jul 2017

NCT06703125Recruiting850Est. Feb 2029

+11 more trials

Alliance PharmaceuticalsUK - Wiltshire

4 programs

GalantaminePhase 31 trial

AripiprazolePhase 2Small Molecule

EEG Studies of Sensory Processing in Autistic ChildrenN/A1 trial

OxcarbazepineN/A1 trial

Active Trials

NCT00319722Unknown130Est. Mar 2011

NCT00467753Completed5Est. Dec 2011

NCT00252603Completed20Est. Apr 2007

CuremarkNY - Rye Brook

4 programs

CM-ATPhase 31 trial

Active Trials

NCT00881452Completed182Est. Sep 2011

NCT00912691Completed165Est. Feb 2016

NCT02410902Completed190Est. Dec 2017

+1 more trials

RepligenWALTHAM, MA

2 programs

RG1068Phase 31 trial

Active Trials

NCT00036244CompletedEst. Jan 2004

NCT00036231Terminated

Innovation PharmaceuticalsMA - Wakefield

1 program

NTI164Phase 31 trial

Active Trials

NCT07257939Not Yet Recruiting98Est. Jul 2030

Neurotech PharmaceuticalsRI - Cumberland

1 program

NTI164Phase 3

Oregon TherapeuticsFrance - Paris

2 programs

Parent massage tx groupPhase 2/31 trial

Parent massage tx groupN/A1 trial

Active Trials

NCT02222662Completed35Est. Feb 2016

NCT01801696Completed103Est. Mar 2016

Angeles TherapeuticsCA - Los Angeles

10 programs

RisperidonePhase 21 trial

Behavioral Interventions for Anxiety in Children with AutismN/A1 trial

Childhood Autism Risks From Genetics and the Environment (The CHARGE Study)N/A1 trial

Discrete Trial TrainingN/A1 trial

Early Start Denver ModelN/A1 trial

+5 more programs

Active Trials

NCT05031364Recruiting100Est. Jun 2026

NCT00106652Completed2,000Est. Sep 2017

NCT01018407Completed192Est. Jun 2015

+7 more trials

SPARK BiopharmaKorea - Seoul

1 program

EsomeprazolePhase 2Small Molecule1 trial

Active Trials

NCT03866668Recruiting25Est. Dec 2028

Yamo PharmaceuticalsOH - Columbus

1 program

L1-79Phase 21 trial

Active Trials

NCT02947048Completed42Est. Feb 2018

Allergy TherapeuticsUK - West Sussex

2 programs

Acupressure treatments and NAET treatmentsPhase 1/21 trial

NAET with acupressure and/or chiropractic proceduresN/A1 trial

Active Trials

NCT00277407Completed48Est. May 2007

NCT00247156Completed60Est. Nov 2005

Beike Bio-TechnologyChina - Shenzhen

1 program

human cord blood mononuclear cellsPhase 1/2

Colorado TherapeuticsCO - Louisville

3 programs

DMXB-A 150 mgPhase 11 trial

Facing Your FearsN/A1 trial

Unstuck and On TargetN/A1 trial

Active Trials

NCT01727466Completed50Est. Dec 2009

NCT07084324Recruiting40Est. Mar 2030

NCT02111551Terminated2Est. May 2016

Purdue PharmaCT - Stamford

2 programs

Eyelink Portable DuoN/A1 trial

Active Trials

NCT06685822Completed27Est. Aug 2025

NCT06586788Completed154Est. Sep 2022

Temple TherapeuticsNetherlands - Geleen

1 program

CAPSN/A1 trial

Active Trials

NCT05702658Active Not Recruiting40Est. Aug 2025

GSKLONDON, United Kingdom

1 program

Developmental Delay in Children Exposed During Pregnancy to Either Lamotrigine,Sodium Valproate, or N/A1 trial

Active Trials

NCT01097720Unknown298Est. Sep 2010

ViiV HealthcareNC - Durham

1 program

Developmental Delay in Children Exposed During Pregnancy to Either Lamotrigine,Sodium Valproate, or N/A

Genome & CompanyKorea - Suwon

1 program

Predictors of Caregiver Adaptation to Pervasive Developmental DisordersN/A

Sunshine BiopharmaQC - Varennes

1 program

Rehearsal strategyN/A1 trial

Active Trials

NCT04209452Terminated15Est. Nov 2020

Jerusalem PharmaceuticalsIsrael - Ramallah

1 program

The social ABCsN/A1 trial

Active Trials

NCT07025603Completed20Est. Dec 2023

ProgenaBiomeCA - Ventura

1 program

Fecal Microbiota TransplantPHASE_11 trial

Active Trials

NCT04630847Not Yet Recruiting10Est. Jul 2027

BrainsWayJerusalem, Israel

1 program

Transcranial Magnetic StimulationPHASE_21 trial

Active Trials

NCT01388179Terminated20Est. Dec 2016

Trial Timeline

Clinical trial activity over time

2021

2022

2023

2024

2025

2026

2027

2028

2029

2030

OtsukaAripiprazole

BiocorpGammaplex 5%

Innovation PharmaceuticalsNTI164

CuremarkCM-AT

Alliance PharmaceuticalsGalantamine

RepligenRG1068

Human BioSciencessecretin, synthetic porcine

Human BioSciencesVasopressin

Oregon TherapeuticsParent massage tx group

SPARK BiopharmaEsomeprazole

Yamo PharmaceuticalsL1-79

Showing 15 of 49 trials with date data

Clinical Trials (50)

Total enrollment: 5,848 patients across 50 trials

NCT02069977 OtsukaAripiprazole

Study to Evaluate the Efficacy and Safety of Aripiprazole

Start: Feb 2014Est. completion: Feb 201679 patients

Phase 4Unknown

NCT02199925 BiocorpGammaplex 5%

An Open-Label Study to Evaluate the Efficacy of High-Dose Gammaplex in Children on the Autism Spectrum

Start: Jun 2013Est. completion: Jul 201530 patients

Phase 4Unknown

NCT00065884 Human BioSciencesValproate

Valproate Response in Aggressive Autistic Adolescents

30 patients

Phase 3Unknown

NCT07257939 Innovation PharmaceuticalsNTI164

NTI164 in Autism Spectrum Disorder

Start: Jul 2026Est. completion: Jul 203098 patients

Phase 3Not Yet Recruiting

NCT02649959 CuremarkCM-AT

An Open Label Study of CM-AT for the Treatment of Children With Autism

Start: Oct 2015Est. completion: Jan 2028405 patients

Phase 3Active Not Recruiting

NCT02410902 CuremarkCM-AT

A Trial of CM-AT in Children With Autism With All Levels of FCT (The Blum Study)

Start: May 2015Est. completion: Dec 2017190 patients

Phase 3Completed

NCT00912691 CuremarkCM-AT

A Trial of CM-AT in Children With Autism- Open Label Extension Study

Start: Jan 2010Est. completion: Feb 2016165 patients

Phase 3Completed

NCT00881452 CuremarkCM-AT

A Trial of CM-AT in Children With Autism

Start: May 2009Est. completion: Sep 2011182 patients

Phase 3Completed

NCT00252603 Alliance PharmaceuticalsGalantamine

Galantamine Versus Placebo in Childhood Autism

Start: Apr 2004Est. completion: Apr 200720 patients

Phase 3Completed

NCT00036231 RepligenRG1068

Synthetic Human Secretin in Children With Autism and Gastrointestinal Dysfunction

Start: Apr 2002

Phase 3Terminated

NCT00036244 RepligenRG1068

Synthetic Human Secretin in Children With Autism

Start: Apr 2002Est. completion: Jan 2004

Phase 3Completed

NCT00065962 Human BioSciencessecretin, synthetic porcine

Secretin for the Treatment of Autism

Start: Jun 1999Est. completion: May 200085 patients

Phase 3Completed

NCT03204786 Human BioSciencesVasopressin

Intranasal Vasopressin Treatment in Children With Autism

Start: Feb 2018Est. completion: Mar 2024157 patients

Phase 2/3Completed

NCT01801696 Oregon TherapeuticsParent massage tx group

Parent-delivered Massage for Children With Autism

Start: Sep 2012Est. completion: Mar 2016103 patients

Phase 2/3Completed

NCT03866668 SPARK BiopharmaEsomeprazole

A Study of Esomeprazole in Children With Autism

Start: May 2019Est. completion: Dec 202825 patients

Phase 2Recruiting

NCT02947048 Yamo PharmaceuticalsL1-79

Safety of L1-79 in Adolescent and Adult Males With Autism

Start: Oct 2016Est. completion: Feb 201842 patients

Phase 2Completed

NCT01388179 BrainsWayTranscranial Magnetic Stimulation

Deep rTMS (Repetitive Transcranial Magnetic Stimulation)for Treatment of Autism Symptoms in Children.

Start: Jan 2013Est. completion: Dec 201620 patients

Phase 2Terminated

NCT01171937 Angeles TherapeuticsRisperidone

Risperidone Treatment In Children With Autism Spectrum Disorder And High Levels Of Repetitive Behavior

Start: Sep 2008Est. completion: Dec 201641 patients

Phase 2Completed

NCT00965068 Human BioSciencesCholesterol

Cholesterol in ASD: Characterization and Treatment

Start: Jul 2009Est. completion: Nov 201344 patients

Phase 1/2Completed

NCT01343511 Biocorphuman cord blood mononuclear cells

Safety and Efficacy of Stem Cell Therapy in Patients With Autism

Start: Mar 2009Est. completion: May 201137 patients

Phase 1/2Completed

NCT00247156 Allergy TherapeuticsAcupressure treatments and NAET treatments

An Autism Study Using Nambudripad's Food Allergy Elimination Treatments

Start: Nov 2004Est. completion: Nov 200560 patients

Phase 1/2Completed

NCT00065910 Human BioSciencesCaregiver joint attention intervention

Improving Attention Skills of Children With Autism

Start: Sep 2001Est. completion: Jan 200630 patients

Phase 1/2Completed

NCT04630847 ProgenaBiomeFecal Microbiota Transplant

Familial Fecal Microbiota Transplant for the Treatment of Subjects With Autism Spectrum Disorders

Start: Dec 2025Est. completion: Jul 202710 patients

Phase 1Not Yet Recruiting

NCT02111551 Colorado TherapeuticsDMXB-A 150 mg

Phase I Nicotinic Agonist Treatment Trial for Autism

Start: Jun 2015Est. completion: May 20162 patients

Phase 1Terminated

NCT07508436 Angeles TherapeuticsSafety Planning Intervention

Crisis Interventions for Pediatric Providers - Autism Version

Start: Dec 2026Est. completion: Dec 202870 patients

N/ANot Yet Recruiting

NCT07476937 Human BioSciencesPower Down bedtime manipulation

Differences in Rest, Emotion, and Arousal Modulation in Youth

Start: Apr 2026Est. completion: Aug 203060 patients

N/ANot Yet Recruiting

NCT07081724 Human BioSciencesOnline Parent Training in Early Behavioral Intervention

"Opt-In" Early Intervention Program for Children Waiting to Receive an Autism Diagnosis

Start: Aug 2025Est. completion: Dec 2029120 patients

N/ARecruiting

NCT07084324 Colorado TherapeuticsUnstuck and On Target

Spanish-language Executive Function Intervention for Children With Autism and/or ADHD

Start: Jul 2025Est. completion: Mar 203040 patients

N/ARecruiting

NCT06703125 Human BioSciencesEyelink Portable Duo

The Alama Project: Autism Outcomes and Neurobehavioral Markers in Young Children Born to Mothers With HIV in Kenya

Start: Feb 2025Est. completion: Feb 2029850 patients

N/ARecruiting

NCT06705907 Human BioSciencesAchieving Independence and Mastery in School

Improving Academic and Social Functioning in Middle-Schoolers With Autism

Start: Dec 2024Est. completion: Aug 2029224 patients

N/ARecruiting

NCT06685822 Purdue PharmaEyelink Portable Duo

Expanding Autism Diagnostic Biomarkers to Kenya

Start: Nov 2024Est. completion: Aug 202527 patients

N/ACompleted

NCT05874466 Human BioSciencesSenseToKnow Autism Screening Device Validation Study

SenseToKnow Autism Screening Device Validation Study

Start: Jul 2023Est. completion: Dec 2027350 patients

N/ARecruiting

NCT05702658 Temple TherapeuticsCAPS

The Effectiveness of an Autistic-delivered Peer-support Intervention for Autistic Adults: Community Autism Peer Specialist (CAPS) Program

Start: Feb 2023Est. completion: Aug 202540 patients

N/AActive Not Recruiting

NCT05031364 Angeles TherapeuticsBehavioral Interventions for Anxiety in Children with Autism

CBT for Youth With Autism and Emotional/Behavioral Needs in Community Care Settings

Start: Jul 2021Est. completion: Jun 2026100 patients

N/ARecruiting

NCT07025603 Jerusalem PharmaceuticalsThe social ABCs

Evaluation of the Social ABCs in Israel

Start: Jun 2021Est. completion: Dec 202320 patients

N/ACompleted

NCT04209452 Sunshine BiopharmaRehearsal strategy

Improving Working Memory of Children With Autism

Start: Jan 2020Est. completion: Nov 202015 patients

N/ATerminated

NCT06586788 Purdue PharmaEyelink Portable Duo

Validation of Indiana's Early Evaluation Hub System

Start: Jun 2019Est. completion: Sep 2022154 patients

N/ACompleted

NCT03179787 OtsukaAripiprazole oral product

Post-Marketing Surveillance Study of Aripiprazole in Patients With Autism

Start: Apr 2017Est. completion: Jul 2020528 patients

N/ACompleted

NCT02998684 Human BioSciencesActive Transcranial Direct Current Stimulation

Learning Enhancement Through Neurostimulation in Autism

Start: Jul 2016Est. completion: Jul 201725 patients

N/ACompleted

NCT02536365 Human BioSciencesSensory Integration Therapy

Sensory Integration Therapy in Autism: Mechanisms and Effectiveness

Start: Sep 2015Est. completion: Oct 2022135 patients

N/ACompleted

NCT02430051 Angeles TherapeuticsSensory Adapted Dental Environment

Sensory Adapted Dental Environments to Enhance Oral Care for Children

Start: May 2015Est. completion: Apr 2021220 patients

N/ACompleted

NCT02222662 Oregon TherapeuticsParent massage tx group

Parent-delivered Massage for Children With Autism - Ages 6 to 11

Start: Jun 2014Est. completion: Feb 201635 patients

N/ACompleted

NCT01751698 Angeles TherapeuticsJASP-EMT

Adaptive Interventions for Minimally Verbal Children With ASD in the Community

Start: Jan 2013Est. completion: Dec 2017192 patients

N/ACompleted

NCT02077985 Angeles TherapeuticsSensory Adapted Dental Environment

Sensory Adapted Dental Environment to Enhance Oral Care for Children With ASD

Start: Oct 2012Est. completion: Jun 201345 patients

N/ACompleted

NCT01092208 Human BioSciencesStudies of Autistic Patients: Gene Networks and Clinical Subtypes

Studies of Autistic Patients: Gene Networks and Clinical Subtypes

Start: Mar 2010Est. completion: Sep 201311 patients

N/ATerminated

NCT01018407 Angeles TherapeuticsDiscrete Trial Training

Interventions for Communication in Autism Network

Start: Dec 2009Est. completion: Jun 2015192 patients

N/ACompleted

NCT00698997 Angeles TherapeuticsEarly Start Denver Model

Intensive Intervention for Toddlers With Autism (EARLY STEPS)

Start: Apr 2008Est. completion: Dec 2013118 patients

N/ACompleted

NCT00496210 Human BioSciencesPredictors of Caregiver Adaptation to Pervasive Developmental Disorders

Predictors of Caregiver Adaptation to Pervasive Developmental Disorders

Start: Jun 2007Est. completion: Dec 2015324 patients

N/ACompleted

NCT01727466 Colorado TherapeuticsFacing Your Fears

Treatment for Children With Autism and Anxiety

Start: Aug 2006Est. completion: Dec 200950 patients

N/ACompleted

NCT00277407 Allergy TherapeuticsNAET with acupressure and/or chiropractic procedures

Treatment of Autistic Children Using NAET Procedures

Start: Jun 2006Est. completion: May 200748 patients

N/ACompleted

Phase Legend

Preclinical— Lab & animal studies

Phase 1— Safety & dosing

Phase 2— Efficacy testing

Phase 3— Large-scale trials

On Market— Approved & available

Key Insights

11 late-stage (Phase 3) programs, potential near-term approvals

7 actively recruiting trials targeting 5,848 patients

27 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.