Clinical trial NCT05081245: ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)
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).
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: ASD
Autism
Autism Spectrum Disorder
Social Communication
Lead sponsor: MapLight Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-10-18
Enrollment: 150
Sex: ALL
Minimum age (years): 12
Maximum age (years): 45
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Multi-center, randomized, double-blind, placebo-controlled study
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
timeFrame: Baseline up to Day 110
Secondary outcomes:
measure: Change from Baseline in Clinician Global Impression of Improvement (CGI-I)
timeFrame: Day 110
measure: Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score
timeFrame: Baseline up to Day 110
measure: Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABI Mood and Anxiety Domain Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABI Challenging Behavior Domain Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABI Self-regulation Domain Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABI-Short Form (ABI-S) Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score
timeFrame: Baseline up to Day 110
measure: Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains
timeFrame: Baseline up to Day 110
Other outcomes:
Locations:
city: Dothan
country: United States
facility: Harmonex Neuroscience Research
state: Alabama
city: Phoenix
country: United States
facility: Southwest Autism Research & Resource Center, Clinical Research
state: Arizona
city: Glendale
country: United States
facility: Cortica Healthcare
state: California
city: Orange
country: United States
facility: NRC Research Institute
state: California
city: Orange
country: United States
facility: Thompson Autism and Neurodevelopmental Center
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: San Rafael
country: United States
facility: Cortica
state: California
city: Upland
country: United States
facility: Pacific Clinical Research Management Group
state: California
city: New Haven
country: United States
facility: Yale Child Study Center
state: Connecticut
city: Washington
country: United States
facility: Children's National Health System - The Children's Research Institute (CRI)
state: District of Columbia
city: Miami
country: United States
facility: Abba Medical Group
state: Florida
city: Orlando
country: United States
facility: APG Research, LLC
state: Florida
city: Tampa
country: United States
facility: University of South Florida Psychiatry and Behavioral Neurosciences
state: Florida
city: Naperville
country: United States
facility: AMR-Baber Research Inc
state: Illinois
city: Lexington
country: United States
facility: Lurie Center for Autism Massachusetts General Hospital
state: Massachusetts
city: Bloomfield Hills
country: United States
facility: Neurobehavioral Medicine Group
state: Michigan
city: Columbia
country: United States
facility: University of Missouri, Thompson Center for Autism & Neurodevelopmental Disorders
state: Missouri
city: Berlin
country: United States
facility: Hassman Research Institute
state: New Jersey
city: Neptune
country: United States
facility: Jersey Shore University Medical Center
state: New Jersey
city: Orangeburg
country: United States
facility: Nathan Kline Institute for Psychiatric Research
state: New York
city: Staten Island
country: United States
facility: Richmond Behavioral Associates
state: New York
city: White Plains
country: United States
facility: Center for Autism and the Developing Brain, New York State Psychiatric Institute
state: New York
city: Columbus
country: United States
facility: Ohio State University Nisonger Center
state: Ohio
city: Media
country: United States
facility: Suburban Research Associates
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Austin
country: United States
facility: BioBehavioral Research of Austin
state: Texas
city: Houston
country: United States
facility: Red Oak Psychiatry Associates, PA
state: Texas
city: San Antonio
country: United States
facility: Road Runner Research, Ltd.
state: Texas
city: The Woodlands
country: United States
facility: Family Psychiatry of The Woodlands
state: Texas
city: Draper
country: United States
facility: Cedar Clinical Research
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Autism Center
state: Washington
city: Camperdown
country: Australia
facility: Brain and Mind Centre
state: New South Wales
city: South Brisbane
country: Australia
facility: Children's Health Queensland Hospital and Health Service
state: Queensland
city: South Brisbane
country: Australia
facility: Mater Research
state: Queensland
city: Adelaide
country: Australia
facility: Flinders Medical Centre
state: South Australia
city: Parkville
country: Australia
facility: The Royal Children's Hospital, Murdoch Children's Research Institute
state: Victoria
city: Kelowna
country: Canada
facility: OCT Research ULC (dba Okanagan Clinical Trials)
state: British Columbia
city: London
country: Canada
facility: Lawson Health Research Institute/ London Health Sciences Centre
state: Ontario
city: Toronto
country: Canada
facility: Holland Bloorview Kids Rehabilitation Hospital
state: Ontario
Study officials:
References: