Autism Spectrum DisorderPhase 2NCT05081245
ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)
A Phase 2 study testing ML-004 (IR)/(ER) tablet in people with Autism Spectrum Disorder, run by MapLight Therapeutics at 29 sites in 3 countries.
Trial summary
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects… Read more about this study
- Who is it for?
- Ages 12 to 45 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- ML-004 (IR)/(ER) tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 168 participants
- Where
- 29 sitesin 3 countries
- Study dates
- 13 Sep 2022to 17 Apr 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age 12 to 45 at screening
- Has a designated care/study partner who can reliably report on symptoms
- Has a diagnosis of Autism Spectrum Disorder (ASD)
- Has a body mass index (BMI) 18 through 34 kg/m², inclusive
- Full scale IQ (or equivalent) ≥55 score, with adequate verbal fluency, as determined by the Principal Investigator.
- Psychoactive medications and adjunctive therapies are stable for 4 weeks prior to screening
- Must be able to swallow study medication
Who cannot join
- Has Rett syndrome or Child Disintegrative Disorder
- Has participated in any other study and received any other investigational medication (other than COVID-19 vaccination) or device within 60 days prior to screening
- History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- History of suicidal ideation or behavior in the past 12 months, or a positive response to C-SSRS questions 4 and/or 5
- Systolic blood pressure ≥140 mmHg (if adult) or >135 mmHg (if adolescent), or diastolic blood pressure ≥90 (if adult) or >85 mmHg (if adolescent), or a clinical history of uncontrolled or severe hypertension
- If female, is pregnant or lactating
Trial locations and contacts
29 sites in 3 countries.
29 sites in 3 countries
United States24 sites
BioBehavioral Research of Austin
Austin, Texas, 78759
Neurobehavioral Medicine Group
Bloomfield Hills, Michigan, 48302
University of Missouri, Thompson Center for Autism & Neurodevelopmental Disorders
Columbia, Missouri, 65211
Ohio State University Nisonger Center
Columbus, Ohio, 43210
Harmonex Neuroscience Research
Dothan, Alabama, 36303
Cedar Clinical Research
Draper, Utah, 84020
Cortica Healthcare
Glendale, California, 91203
Red Oak Psychiatry Associates, PA
Houston, Texas, 77090
Suburban Research Associates
Media, Pennsylvania, 19063
Abba Medical Group
Miami, Florida, 33176
Jersey Shore University Medical Center
Neptune City, New Jersey, 07753
Yale Child Study Center
New Haven, Connecticut, 06519
NRC Research Institute
Orange, California, 92868
Nathan Kline Institute for Psychiatric Research
Orangeburg, New York, 10962
APG Research, LLC
Orlando, Florida, 32803
Southwest Autism Research & Resource Center, Clinical Research
Phoenix, Arizona, 85006
Virginia Commonwealth University
Richmond, Virginia, 23220
Road Runner Research, Ltd.
San Antonio, Texas, 78249
Cortica
San Rafael, California, 94903
Richmond Behavioral Associates
Staten Island, New York, 10314
University of South Florida Psychiatry and Behavioral Neurosciences
Tampa, Florida, 33613
Family Psychiatry of The Woodlands
The Woodlands, Texas, 77381
Children's National Health System - The Children's Research Institute (CRI)
Washington D.C., District of Columbia, 20010
Center for Autism and the Developing Brain, New York State Psychiatric Institute
White Plains, New York, 10605
Canada3 sites
OCT Research ULC (dba Okanagan Clinical Trials)
Kelowna, British Columbia, V1Y 1Z9
Lawson Health Research Institute/ London Health Sciences Centre
London, Ontario, N6A 5W9
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, M4G 1R8
Australia2 sites
Brain and Mind Centre
Camperdown, New South Wales, 2050
The Royal Children's Hospital, Murdoch Children's Research Institute
Parkville, Victoria, 3052
About this study
Official title: A Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Investigate the Efficacy, Safety, and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects with ASD.
- Conditions
- Autism Spectrum Disorder
- Being tested
- ML-004 (IR)/(ER) tablet, ML-004 Placebo
- Keywords
- ASD, Autism, Autism Spectrum Disorder, Social Communication
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder, Communication
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ML-004 (IR)/(ER) tabletDrug
Participants will receive ML-004 once daily.
ML-004 PlaceboDrug
Participants will receive matching placebo once daily.
| Study group | Role | What they receive |
|---|---|---|
| ML-004 (IR)/(ER) tablet | Experimental | ML-004 is a bilayer immediate-release (IR)/extended-release (ER, gastroretentive) oral tablet formulation. ML-004 is taken orally once daily and provided as a tablet in two dose strengths of 12 mg (3 mg IR/9 mg ER) and 24 mg tablet (6 mg IR/18 mg ER). Dose levels for this study are 12 mg (provided as one 12 mg tablet), 24 mg (one 24 mg tablet), 48 mg (two 24 mg tablets), and 72 mg (three 24 mg tablets). Receives: Drug: ML-004 (IR)/(ER) tablet |
| ML-004 Placebo | Placebo comparator | Matched placebo oral tablets will consist of same excipients as the investigational drug but without any ML-004. Placebo taken orally once daily to match tablet number of ML-004 dose levels for 12 mg (1 tablet), 24 mg (1 tablet), 48 mg (2 tablets), and 72 mg (3 tablets). Receives: Drug: ML-004 Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
Change from baseline in the ABI-Social Communication Domain Score will be reported. The ABI is a 62-item questionnaire for reporting the behaviors of subjects (ages: 3 years-adulthood) diagnosed with ASD. The tool is suitable for completion by parents or care/study partners of people with ASD. Each item assesses either quality (from not at all to without help) or frequency (never to very often) of a particular behavior. The Social Communication domain score is the sum of the scores in the social communication domain divided by the number of items in the domain.
Measured: Baseline up to Day 110
Secondary outcomes (12)
Change from Baseline in Clinician Global Impression of Improvement (CGI-I)
The CGI-I score is a single-item instrument based on a 7-point scale routinely used in clinical trials to capture the Investigator's global impression of response. The Investigator or designee rates the improvement observed from 1 (very much improved) to 7 (very much worse).
Measured: Day 110
Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score
The ABI-Clinician (ABI-C) captures the clinician rating of behaviors of a person with ASD that occurred over the week prior to assessment. It contains 14 items reflecting the core and associated autism behavior domains: Social Communication, Restrictive Behaviors, Mood and Anxiety, Self Regulation, and Challenging Behavior. Each item is rated on a 7-point scale from 1 (none; no symptoms present) to 7 (very severe; persistent interference with function or adaptation).
Measured: Baseline up to Day 110
Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score
The ABC is a parent- or care/study partner-reported behavior rating assessment with five domains and 58 items, each rated on a 0 (not at all a problem) to 3 (the problem is severe in degree) scale. The irritability domain consists of 15 items.
Measured: Baseline up to Day 110
Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score
The CGI-S is a global assessment of the clinician-rater's impression of the severity of the participant's illness. It is rated on a scale of 1 (normal, not at all ill) to 7 (among the most extremely ill).
Measured: Baseline up to Day 110
Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score
Each item on the ABI-Repetitive/Restrictive Behavior Domain is rated by frequency (never to very often). The domain score is the sum of all domain items scores divided by the number of items in the domain.
Measured: Baseline up to Day 110
Change from baseline in the ABI Mood and Anxiety Domain Score
Each item on the ABI- Mood and Anxiety Domain is rated by frequency (never to very often). The domain score is the sum of all domain items scores divided by the number of items in the domain.
Measured: Baseline up to Day 110
Change from baseline in the ABI Challenging Behavior Domain Score
Each item on the ABI- Challenging Behavior Domain is rated by frequency (never to very often). The domain score is the sum of all domain items scores divided by the number of items in the domain.
Measured: Baseline up to Day 110
Change from baseline in the ABI Self-regulation Domain Score
Each item on the ABI- Self-regulation Domain is rated by frequency (never to very often). The domain score is the sum of all domain items scores divided by the number of items in the domain.
Measured: Baseline up to Day 110
Change from baseline in the ABI-Short Form (ABI-S) Score
The ABI-S is a 24-item short version of the ABI, containing items from each of the five domains. The domain score for each domain is calculated as the sum of scores all domain items divided by the number of items in the domain.
Measured: Baseline up to Day 110
Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score
The ABC-SW subscale consists of 16 items of the ABC-2 rated from 0 (not at all a problem) to 3 (the problem is severe in degree).
Measured: Baseline up to Day 110
Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score
The SRS-2 consists of 65 items across 5 subscales. Responses range from 1 (not true) to 4 (almost always true).
Measured: Baseline up to Day 110
Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains
The Vineland-3 Domain Level Version contains 5 domains. Responses on each item are rated from 0 (never) to 2 (usually).
Measured: Baseline up to Day 110
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2021
- Study start13 Sep 2022
- Primary completion10 Mar 2026
- Study completion17 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Multi-center, randomized, double-blind, placebo-controlled study
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 168 participants (actual)
Sponsor and investigators
- Lead sponsor
- MapLight Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05081245
- Sponsor's protocol ID
- ML-004-002
- Status last verified
- Jun 2025
- First submitted
- 5 Oct 2021
- Last updated
- 19 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Investigate the Efficacy, Safety, and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
- ClinicalTrials.gov ID
- NCT05081245
- Acronym / sponsor ID
- ML-004-002
- Conditions
- Autism Spectrum Disorder
- Keywords
- ASD, Autism, Autism Spectrum Disorder, Social Communication
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 168 participants (actual)
- Lead sponsor
- MapLight TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 13 Sep 2022 (actual)
- Primary completion
- 10 Mar 2026 (actual)
- Study completion
- 17 Apr 2026 (actual)
- First posted
- 18 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects with ASD.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Multi-center, randomized, double-blind, placebo-controlled study
| Arm | Type | Receives |
|---|---|---|
| ML-004 (IR)/(ER) tablet | Experimental | Drug: ML-004 (IR)/(ER) tabletML-004 is a bilayer immediate-release (IR)/extended-release (ER, gastroretentive) oral tablet formulation. ML-004 is taken orally once daily and provided as a tablet in two dose strengths of 12 mg (3 mg IR/9 mg ER) and 24 mg tablet (6 mg IR/18 mg ER). Dose levels for this study are 12 mg (provided as one 12 mg tablet), 24 mg (one 24 mg tablet), 48 mg (two 24 mg tablets), and 72 mg (three 24 mg tablets). |
| ML-004 Placebo | Placebo comparator | Drug: ML-004 PlaceboMatched placebo oral tablets will consist of same excipients as the investigational drug but without any ML-004. Placebo taken orally once daily to match tablet number of ML-004 dose levels for 12 mg (1 tablet), 24 mg (1 tablet), 48 mg (2 tablets), and 72 mg (3 tablets). |
Who may qualify
- Ages
- 12 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Age 12 to 45 at screening
- Has a designated care/study partner who can reliably report on symptoms
- Has a diagnosis of Autism Spectrum Disorder (ASD)
- Has a body mass index (BMI) 18 through 34 kg/m², inclusive
- Full scale IQ (or equivalent) ≥55 score, with adequate verbal fluency, as determined by the Principal Investigator.
Key exclusion criteria (6 listed)
- Has Rett syndrome or Child Disintegrative Disorder
- Has participated in any other study and received any other investigational medication (other than COVID-19 vaccination) or device within 60 days prior to screening
- History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- History of suicidal ideation or behavior in the past 12 months, or a positive response to C-SSRS questions 4 and/or 5
- Systolic blood pressure ≥140 mmHg (if adult) or >135 mmHg (if adolescent), or diastolic blood pressure ≥90 (if adult) or >85 mmHg (if adolescent), or a clinical history of uncontrolled or severe hypertension
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ML-004 (IR)/(ER) tabletParticipants will receive ML-004 once daily. | Drug | ML-004 (IR)/(ER) tablet |
| ML-004 PlaceboParticipants will receive matching placebo once daily. | Drug | ML-004 Placebo |
Primary outcome
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI-Social Communication Domain Score will be reported. The ABI is a 62-item questionnaire for reporting the behaviors of subjects (ages: 3 years-adulthood) diagnosed with ASD. The tool is suitable for completion by parents or care/study partners of people with ASD. Each item assesses either quality (from not at all to without help) or frequency (never to very often) of a particular behavior. The Social Communication domain score is the sum of the scores in the social communication domain divided by the number of items in the domain.
12 secondary outcomes
Change from Baseline in Clinician Global Impression of Improvement (CGI-I)
Time frame: Day 110
Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score
Time frame: Baseline up to Day 110
Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score
Time frame: Baseline up to Day 110
Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI Mood and Anxiety Domain Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI Challenging Behavior Domain Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI Self-regulation Domain Score
Time frame: Baseline up to Day 110
Change from baseline in the ABI-Short Form (ABI-S) Score
Time frame: Baseline up to Day 110
Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score
Time frame: Baseline up to Day 110
Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score
Time frame: Baseline up to Day 110
Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains
Time frame: Baseline up to Day 110
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
29 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ML-004 (IR)/(ER) tablet in people with Autism Spectrum Disorder, run by MapLight Therapeutics at 29 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in United States, Canada, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score (time frame: Baseline up to Day 110). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 May 2026.