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Completed

NCT Number: NCT05081245

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.

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Key information

Conditions

Autism Spectrum Disorder Autistic Disorder Behavior Child Development Disorders, Pervasive Communication Mental Disorders Neurodevelopmental Disorders

Age range

12 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Brain and Mind Centre, Camperdown, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

ML-004 (IR)/(ER) tablet

Drug

Participants will receive ML-004 once daily.

ML-004 Placebo

Drug

Participants will receive matching placebo once daily.

Primary outcomes

  1. 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.

Secondary outcomes

  1. Change from Baseline in Clinician Global Impression of Improvement (CGI-I)

    Time frame: Day 110

    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).

  2. Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score

    Time frame: Baseline up to Day 110

    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).

  3. Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score

    Time frame: Baseline up to Day 110

    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.

  4. Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score

    Time frame: Baseline up to Day 110

    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).

  5. Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score

    Time frame: Baseline up to Day 110

    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.

  6. Change from baseline in the ABI Mood and Anxiety Domain Score

    Time frame: Baseline up to Day 110

    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.

  7. Change from baseline in the ABI Challenging Behavior Domain Score

    Time frame: Baseline up to Day 110

    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.

  8. Change from baseline in the ABI Self-regulation Domain Score

    Time frame: Baseline up to Day 110

    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.

  9. Change from baseline in the ABI-Short Form (ABI-S) Score

    Time frame: Baseline up to Day 110

    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.

  10. Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score

    Time frame: Baseline up to Day 110

    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).

  11. Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score

    Time frame: Baseline up to Day 110

    The SRS-2 consists of 65 items across 5 subscales. Responses range from 1 (not true) to 4 (almost always true).

  12. 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

    The Vineland-3 Domain Level Version contains 5 domains. Responses on each item are rated from 0 (never) to 2 (usually).

Sponsors and collaborators

Lead sponsor

MapLight Therapeutics

Industry

Registry information

Official study 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).

Important dates

Study start
2022
Primary completion
2026
Study completion
2026
First posted
Oct 18, 2021
Registry last updated
May 19, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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