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

NCT05081245 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ML-004-002
• Study Type: interventional
• Target: 150
• Locations: 3 countries
• Sites: 29

Brief 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 with ASD.

Conditions

Autism Spectrum Disorder

Keywords

ASD; Autism; Autism Spectrum Disorder; Social Communication

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline up to Day 110

Secondary Outcomes (12)

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

  Day 110

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

  Baseline up to Day 110

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

  Baseline up to Day 110

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

  Baseline up to Day 110

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

  Baseline up to Day 110

Study Arms (2)

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).

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).

Drug: ML-004 Placebo

Interventions

ML-004 (IR)/(ER) tablet DRUG

Participants will receive ML-004 once daily.

ML-004 (IR)/(ER) tablet

ML-004 Placebo DRUG

Participants will receive matching placebo once daily.

ML-004 Placebo

Eligibility Criteria

• Age: 12 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• MapLight Therapeutics: lead

Study Sites (29)

Harmonex Neuroscience Research

Dothan, Alabama, 36303, United States

Location

Southwest Autism Research & Resource Center, Clinical Research

Phoenix, Arizona, 85006, United States

Location

Cortica Healthcare

Glendale, California, 91203, United States

Location

NRC Research Institute

Orange, California, 92868, United States

Location

Cortica

San Rafael, California, 94903, United States

Location

Yale Child Study Center

New Haven, Connecticut, 06519, United States

Location

Children's National Health System - The Children's Research Institute (CRI)

Washington D.C., District of Columbia, 20010, United States

Location

Abba Medical Group

Miami, Florida, 33176, United States

Location

APG Research, LLC

Orlando, Florida, 32803, United States

Location

University of South Florida Psychiatry and Behavioral Neurosciences

Tampa, Florida, 33613, United States

Location

Neurobehavioral Medicine Group

Bloomfield Hills, Michigan, 48302, United States

Location

University of Missouri, Thompson Center for Autism & Neurodevelopmental Disorders

Columbia, Missouri, 65211, United States

Location

Jersey Shore University Medical Center

Neptune City, New Jersey, 07753, United States

Location

Nathan Kline Institute for Psychiatric Research

Orangeburg, New York, 10962, United States

Location

Richmond Behavioral Associates

Staten Island, New York, 10314, United States

Location

Center for Autism and the Developing Brain, New York State Psychiatric Institute

White Plains, New York, 10605, United States

Location

Ohio State University Nisonger Center

Columbus, Ohio, 43210, United States

Location

Suburban Research Associates

Media, Pennsylvania, 19063, United States

Location

BioBehavioral Research of Austin

Austin, Texas, 78759, United States

Location

Red Oak Psychiatry Associates, PA

Houston, Texas, 77090, United States

Location

Road Runner Research, Ltd.

San Antonio, Texas, 78249, United States

Location

Family Psychiatry of The Woodlands

The Woodlands, Texas, 77381, United States

Location

Cedar Clinical Research

Draper, Utah, 84020, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23220, United States

Location

Brain and Mind Centre

Camperdown, New South Wales, 2050, Australia

Location

The Royal Children's Hospital, Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

OCT Research ULC (dba Okanagan Clinical Trials)

Kelowna, British Columbia, V1Y 1Z9, Canada

Location

Lawson Health Research Institute/ London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Holland Bloorview Kids Rehabilitation Hospital

Toronto, Ontario, M4G 1R8, Canada

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder; Communication

Interventions

Tablets

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Behavior

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Multi-center, randomized, double-blind, placebo-controlled study

Study Record Dates

• First Submitted: October 5, 2021
• First Posted: October 18, 2021
• Study Start: September 13, 2022
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: December 22, 2025

Record last verified: 2025-06

Locations

US (24); CA (3); AU (2)