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

Phase 2Active Not RecruitingNCT05081245

MapLight Therapeutics150 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

150

Conditions

Autism Spectrum Disorder

Interventions

ML-004 (IR)/(ER) tabletDRUG

Participants will receive ML-004 once daily.

ML-004 PlaceboDRUG

Participants will receive matching placebo once daily.

Eligibility

Sex: ALLMin age: 12 YearsMax age: 45 Years

Medical Language ↔ Plain English

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

Locations (29)

Outcomes

Primary Outcomes

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.

Time frame: Baseline up to Day 110

Secondary Outcomes

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).

Time frame: 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).

Time frame: 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.

Data from ClinicalTrials.gov

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Harmonex Neuroscience Research

Dothan, Alabama, United States

Southwest Autism Research & Resource Center, Clinical Research

Phoenix, Arizona, United States

Cortica Healthcare

Glendale, California, United States

NRC Research Institute

Orange, California, United States

Cortica

San Rafael, California, United States

Yale Child Study Center

New Haven, Connecticut, United States

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

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

Abba Medical Group

Miami, Florida, United States

APG Research, LLC

Orlando, Florida, United States

University of South Florida Psychiatry and Behavioral Neurosciences

Tampa, Florida, United States

...and 19 more locations

Time frame: 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).

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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).

Time frame: 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).

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

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

Time frame: Baseline up to Day 110