ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)
An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
1 other identifier
interventional
120
3 countries
21
Brief Summary
ML-004-003 is a multi-center, open-label extension study that will enroll approximately 120 adolescent and adult subjects with ASD that have completed study ML-004-002. The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Jun 2023
Typical duration for phase_2
21 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 22, 2023
CompletedStudy Start
First participant enrolled
June 1, 2023
CompletedFirst Posted
Study publicly available on registry
June 5, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2027
March 16, 2026
March 1, 2026
4.4 years
May 22, 2023
March 13, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Baseline up to Day 362
Secondary Outcomes (4)
Frequency of occurrence of Serious Adverse Events (SAEs)
Baseline up to Day 362
Frequency of occurrence of TEAEs leading to discontinuation.
Baseline up to Day 362
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Baseline up to Day 362
Frequency of occurrence of suicidal ideation and behavior as assessed by the Columbia-Suicide Severity Rating Scale.
Baseline up to Day 362
Study Arms (1)
ML-004 (IR)/(ER) tablet
EXPERIMENTALML-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).
Interventions
Eligibility Criteria
You may qualify if:
- Has completed Study ML-004-002 within the past 90 days
- Age 12 years to 46 years 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 kg/m²
- Psychoactive medications and adjunctive therapies 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
- History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening
- Has a positive response to C-SSRS questions 4 and/or 5, or is a significant risk for suicidal behavior
- Has a clinical history of uncontrolled or severe hypertension
- If female, is pregnant or lactating
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (21)
Harmonex Neuroscience Research
Dothan, Alabama, 36303, United States
Southwest Autism Research and Resource Center
Phoenix, Arizona, 85006, United States
Cortica Healthcare
Glendale, California, 91203, United States
NRC Research Institute
Orange, California, 92868, United States
Cortica
San Rafael, California, 94903, United States
Yale Child Study Center
New Haven, Connecticut, 06519, United States
The Children's Research Institute
Washington D.C., District of Columbia, 20010, United States
Abba Medical Group
Miami, Florida, 33176, United States
APG Research, LLC
Orlando, Florida, 32803, United States
University of South Florida Psychiatry and Behavioral Neurosciences
Tampa, Florida, 33613, United States
Neurobehavioral Medicine Group
Bloomfield Hills, Michigan, 48302, United States
University of Missouri, Thompson Center for Autism & Neurodevelopment
Columbia, Missouri, 65211, United States
Nathan Kline Institute for Psychiatric Research
Orangeburg, New York, 10962, United States
Richmond Behavioral Associates
Staten Island, New York, 10314, United States
Suburban Research Associates
Media, Pennsylvania, 19063, United States
BioBehavioral Research of Austin
Austin, Texas, 78759, United States
Road Runner Research, Ltd.
San Antonio, Texas, 78249, United States
Family Psychiatry of The Woodlands
The Woodlands, Texas, 77381, United States
Brain and Mind Centre
Camperdown, New South Wales, 2050, Australia
The Royal Children's Hospital, Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
Holland Bloorview Kids Rehabilitation Hospital
Toronto, Ontario, M4G 1R8, Canada
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 22, 2023
First Posted
June 5, 2023
Study Start
June 1, 2023
Primary Completion (Estimated)
November 1, 2027
Study Completion (Estimated)
December 1, 2027
Last Updated
March 16, 2026
Record last verified: 2026-03