ML-004 in Adolescents and Adults with Autism Spectrum Disorders (ASD)
- Conditions
- Autism Spectrum Disorder
- Interventions
- Drug: ML-004 (IR)/(ER) tabletDrug: ML-004 Placebo
- Registration Number
- NCT05081245
- Lead Sponsor
- MapLight Therapeutics
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description ML-004 (IR)/(ER) tablet ML-004 (IR)/(ER) tablet 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). ML-004 Placebo ML-004 Placebo 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).
- Primary Outcome Measures
Name Time Method Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score 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 Outcome Measures
Name Time Method Change from Baseline in Clinician Global Impression of Improvement (CGI-I) 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).
Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score 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).
Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score 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.
Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score 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).
Change from baseline in the ABI Self-regulation Domain Score 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.
Change from baseline in the ABI-Short Form (ABI-S) Score 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.
Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score 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).
Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score 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).
Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains 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).
Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score 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.
Change from baseline in the ABI Mood and Anxiety Domain Score 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.
Change from baseline in the ABI Challenging Behavior Domain Score 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.
Trial Locations
- Locations (39)
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
Thompson Autism and Neurodevelopmental Center
🇺🇸Orange, California, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
Cortica
🇺🇸San Rafael, California, United States
Pacific Clinical Research Management Group
🇺🇸Upland, California, United States
Yale Child Study Center
🇺🇸New Haven, Connecticut, United States
Children's National Health System - The Children's Research Institute (CRI)
🇺🇸Washington, 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
AMR-Baber Research Inc
🇺🇸Naperville, Illinois, United States
Lurie Center for Autism Massachusetts General Hospital
🇺🇸Lexington, Massachusetts, United States
Neurobehavioral Medicine Group
🇺🇸Bloomfield Hills, Michigan, United States
University of Missouri, Thompson Center for Autism & Neurodevelopmental Disorders
🇺🇸Columbia, Missouri, United States
Hassman Research Institute
🇺🇸Berlin, New Jersey, United States
Jersey Shore University Medical Center
🇺🇸Neptune, New Jersey, United States
Nathan Kline Institute for Psychiatric Research
🇺🇸Orangeburg, New York, United States
Richmond Behavioral Associates
🇺🇸Staten Island, New York, United States
Center for Autism and the Developing Brain, New York State Psychiatric Institute
🇺🇸White Plains, New York, United States
Ohio State University Nisonger Center
🇺🇸Columbus, Ohio, United States
Suburban Research Associates
🇺🇸Media, Pennsylvania, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
BioBehavioral Research of Austin
🇺🇸Austin, Texas, United States
Red Oak Psychiatry Associates, PA
🇺🇸Houston, Texas, United States
Road Runner Research, Ltd.
🇺🇸San Antonio, Texas, United States
Family Psychiatry of The Woodlands
🇺🇸The Woodlands, Texas, United States
Cedar Clinical Research
🇺🇸Draper, Utah, United States
Seattle Children's Autism Center
🇺🇸Seattle, Washington, United States
Brain and Mind Centre
🇦🇺Camperdown, New South Wales, Australia
Children's Health Queensland Hospital and Health Service
🇦🇺South Brisbane, Queensland, Australia
Mater Research
🇦🇺South Brisbane, Queensland, Australia
Flinders Medical Centre
🇦🇺Adelaide, South Australia, Australia
The Royal Children's Hospital, Murdoch Children's Research Institute
🇦🇺Parkville, Victoria, Australia
OCT Research ULC (dba Okanagan Clinical Trials)
🇨🇦Kelowna, British Columbia, Canada
Lawson Health Research Institute/ London Health Sciences Centre
🇨🇦London, Ontario, Canada
Holland Bloorview Kids Rehabilitation Hospital
🇨🇦Toronto, Ontario, Canada