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

Autism Spectrum Disorder Clinical Trial

Official 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).

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05081245
• Other study ID #: ML-004-002
• Status: Recruiting
• Phase: Phase 2
• Start date: September 13, 2022
• Est. completion date: April 30, 2025

Study information

• Verified date: May 2024
• Source: MapLight Therapeutics
• Contact: Alexis Levine
• Phone: +1 650-839-4388
• Email: alevine[@]maplightrx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: April 30, 2025
• Est. primary completion date: March 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder
• Child Development Disorders, Pervasive

Intervention

Drug:
• ML-004 (IR)/(ER) tablet
Participants will receive ML-004 once daily.
• ML-004 Placebo
Participants will receive matching placebo once daily.

Locations

Country	Name	City	State
Australia	Flinders Medical Centre	Adelaide	South Australia
Australia	Brain and Mind Centre	Camperdown	New South Wales
Australia	The Royal Children's Hospital, Murdoch Children's Research Institute	Parkville	Victoria
Australia	Children's Health Queensland Hospital and Health Service	South Brisbane	Queensland
Australia	Mater Research	South Brisbane	Queensland
Canada	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
United States	Hassman Research Institute	Berlin	New Jersey
United States	Neurobehavioral Medicine Group	Bloomfield Hills	Michigan
United States	University of Missouri, Thompson Center for Autism & Neurodevelopmental Disorders	Columbia	Missouri
United States	Ohio State University Nisonger Center	Columbus	Ohio
United States	Harmonex Neuroscience Research	Dothan	Alabama
United States	Cedar Clinical Research	Draper	Utah
United States	Cortica Healthcare	Glendale	California
United States	Red Oak Psychiatry Associates, PA	Houston	Texas
United States	Massachusetts General Hospital's Lurie Center for Autism	Lexington	Massachusetts
United States	Suburban Research Associates	Media	Pennsylvania
United States	AMR-Baber Research Inc	Naperville	Illinois
United States	Jersey Shore University Medical Center	Neptune	New Jersey
United States	Yale Child Study Center	New Haven	Connecticut
United States	NRC Research Institute	Orange	California
United States	Nathan Kline Institute for Psychiatric Research	Orangeburg	New York
United States	APG Research, LLC	Orlando	Florida
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Southwest Autism Research & Resource Center, Clinical Research	Phoenix	Arizona
United States	Road Runner Research, Ltd.	San Antonio	Texas
United States	University of California, San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	Richmond Behavioral Associates	Staten Island	New York
United States	University of South Florida Psychiatry and Behavioral Neurosciences	Tampa	Florida
United States	Children's National Health System - The Children's Research Institute (CRI)	Washington	District of Columbia
United States	Center for Autism and the Developing Brain, New York State Psychiatric Institute	White Plains	New York

Sponsors (1)

Lead Sponsor	Collaborator
MapLight Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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).	Day 110
Secondary	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).	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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).	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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.	Baseline up to Day 110
Secondary	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).	Baseline up to Day 110
Secondary	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).	Baseline up to Day 110
Secondary	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).	Baseline up to Day 110