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ML-004 for Autism

Phase 2
Waitlist Available
Research Sponsored by MapLight Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Screening 3 weeks
Treatment Varies
Follow Up baseline up to day 362
Awards & highlights

Study Summary

This trial will study the effects of a drug on people with Autism Spectrum Disorder (ASD). Approximately 120 people will take part.

Who is the study for?
This trial is for adolescents and adults aged 12 to 45 with Autism Spectrum Disorder who finished study ML-004-002 within the last 90 days. Participants must have a stable body weight, be on consistent psychoactive medications or therapies for at least four weeks, and be able to swallow pills. They also need a reliable care partner to report on their symptoms.Check my eligibility
What is being tested?
The study tests an oral medication called ML-004 in tablet form, both immediate-release (IR) and extended-release (ER), for individuals with Autism Spectrum Disorder. This extension of an earlier trial aims to gather more data about the drug's long-term effects.See study design
What are the potential side effects?
While specific side effects are not listed here, common ones associated with ASD medications may include sleepiness, stomach upset, changes in appetite or mood swings. The exact side effects will depend on individual reactions to ML-004.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to day 362
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to day 362 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Secondary outcome measures
Frequency of occurrence of Serious Adverse Events (SAEs)
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Frequency of occurrence of TEAEs leading to discontinuation.
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: ML-004 (IR)/(ER) tabletExperimental Treatment1 Intervention
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).

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Autism Spectrum Disorder (ASD) often target the core symptoms such as social-communication difficulties and repetitive behaviors. Pharmacological treatments, including antipsychotics and selective serotonin reuptake inhibitors (SSRIs), work by modulating neurotransmitter systems like dopamine and serotonin, which are believed to be dysregulated in ASD. Behavioral interventions, such as Applied Behavior Analysis (ABA), focus on reinforcing desired behaviors and reducing harmful ones through structured activities. These mechanisms are crucial as they aim to alleviate the core symptoms of ASD, thereby improving the quality of life and functional abilities of patients.
Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis.Neural Mechanisms of Qigong Sensory Training Massage for Children With Autism Spectrum Disorder: A Feasibility Study.Neurologic treatment strategies in autism: an overview of medical intervention strategies.

Find a Location

Who is running the clinical trial?

MapLight TherapeuticsLead Sponsor
1 Previous Clinical Trials
150 Total Patients Enrolled

Media Library

Autism Research Study Groups: ML-004 (IR)/(ER) tablet
Autism Clinical Trial 2023: ML-004 Highlights & Side Effects. Trial Name: NCT05889273 — Phase 2
~75 spots leftby May 2026