Autism Spectrum DisorderPhase 2NCT05889273
ML-004 Open-Label Extension Study in Adults and Adolescents 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 21 sites in 3 countries.
Trial summary
ML-004-003 is a multi-center, open-label extension study that will enroll approximately 120 adolescent and adult subjects with ASD that have completed study ML-004-002. The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD. Read more about this study
- Who is it for?
- Ages 12 to 46 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?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 21 sitesin 3 countries
- Study dates
- 1 Jun 2023to Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 46 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Has completed Study ML-004-002 within the past 90 days
- Age 12 years to 46 years 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 kg/m²
- Psychoactive medications and adjunctive therapies stable for 4 weeks prior to screening
- Must be able to swallow study medication
Who cannot join
- Has Rett syndrome or Child Disintegrative Disorder
- History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- Has a positive response to C-SSRS questions 4 and/or 5, or is a significant risk for suicidal behavior
- Has a clinical history of uncontrolled or severe hypertension
- If female, is pregnant or lactating
Trial locations and contacts
21 sites in 3 countries.
21 sites in 3 countries
United States18 sites
BioBehavioral Research of Austin
Austin, Texas, 78759
Neurobehavioral Medicine Group
Bloomfield Hills, Michigan, 48302
University of Missouri, Thompson Center for Autism & Neurodevelopment
Columbia, Missouri, 65211
Harmonex Neuroscience Research
Dothan, Alabama, 36303
Cortica Healthcare
Glendale, California, 91203
Suburban Research Associates
Media, Pennsylvania, 19063
Abba Medical Group
Miami, Florida, 33176
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 and Resource Center
Phoenix, Arizona, 85006
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
The Children's Research Institute
Washington D.C., District of Columbia, 20010
Australia2 sites
Brain and Mind Centre
Camperdown, New South Wales, 2050
The Royal Children's Hospital, Murdoch Children's Research Institute
Parkville, Victoria, 3052
Canada1 site
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, M4G 1R8
About this study
Official title: An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
ML-004-003 is a multi-center, open-label extension study that will enroll approximately 120 adolescent and adult subjects with ASD that have completed study ML-004-002. The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD.
- Conditions
- Autism Spectrum Disorder
- Being tested
- ML-004 (IR)/(ER) tablet
- 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.
| 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Measured: Baseline up to Day 362
Secondary outcomes (4)
Frequency of occurrence of Serious Adverse Events (SAEs)
Measured: Baseline up to Day 362
Frequency of occurrence of TEAEs leading to discontinuation.
Measured: Baseline up to Day 362
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Measured: Baseline up to Day 362
Frequency of occurrence of suicidal ideation and behavior as assessed by the Columbia-Suicide Severity Rating Scale.
Measured: Baseline up to Day 362
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2023
- Study start1 Jun 2023
- Primary completionNov 2027 (estimated)
- Study completionDec 2027 (estimated)
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
- Not applicable
- Intervention model
- Single group
- Model description
- Multi-center, open-label study
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- MapLight Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05889273
- Sponsor's protocol ID
- ML-004-003
- Status last verified
- Mar 2026
- First submitted
- 22 May 2023
- Last updated
- 20 Jul 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 20 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
- ClinicalTrials.gov ID
- NCT05889273
- Acronym / sponsor ID
- ML-004-003
- Conditions
- Autism Spectrum Disorder
- Keywords
- ASD, Autism, Autism Spectrum Disorder, Social Communication
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- MapLight TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2023 (actual)
- Primary completion
- Nov 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 5 Jun 2023 (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-004-003 is a multi-center, open-label extension study that will enroll approximately 120 adolescent and adult subjects with ASD that have completed study ML-004-002. The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multi-center, open-label 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). |
Who may qualify
- Ages
- 12 to 46 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- Has completed Study ML-004-002 within the past 90 days
- Age 12 years to 46 years 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 kg/m²
Key exclusion criteria (5 listed)
- Has Rett syndrome or Child Disintegrative Disorder
- History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- Has a positive response to C-SSRS questions 4 and/or 5, or is a significant risk for suicidal behavior
- Has a clinical history of uncontrolled or severe hypertension
- If female, is pregnant or lactating
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 |
Primary outcome
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Time frame: Baseline up to Day 362
4 secondary outcomes
Frequency of occurrence of Serious Adverse Events (SAEs)
Time frame: Baseline up to Day 362
Frequency of occurrence of TEAEs leading to discontinuation.
Time frame: Baseline up to Day 362
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Time frame: Baseline up to Day 362
Frequency of occurrence of suicidal ideation and behavior as assessed by the Columbia-Suicide Severity Rating Scale.
Time frame: Baseline up to Day 362
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2026.
- ML-004-002Link provided by the sponsor.
- 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.
21 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 21 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 5 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: under way, not looking for participants, as of the sponsor's last update on 20 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Australia, Canada. 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Frequency of occurrence of treatment-emergent adverse events (TEAEs). (time frame: Baseline up to Day 362). 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 20 Jul 2026.