What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) targeting social communication deficits include omega-3 fatty acids, oxytocin, and laser acupuncture. Omega-3 fatty acids are generally well-tolerated but can cause gastrointestinal issues like nausea and diarrhea. Oxytocin treatment has not shown significant effects on social symptoms, and no specific side effects were reported. Laser acupuncture combined with sensory-occupational therapy was studied, but specific side effects were not detailed. Overall, these treatments appear to have minimal severe side effects, but gastrointestinal distress is a noted concern with omega-3 supplementation.

What prior FDA approvals exist?

The FDA has approved limited pharmacological treatments specifically for Autism Spectrum Disorder (ASD). Most approved medications, such as antipsychotics like risperidone and aripiprazole, primarily address irritability and behavioral symptoms rather than core social communication deficits. The trial ML-004 is investigating a treatment aimed at improving social communication deficits in ASD, an area where current FDA-approved options are lacking. Therefore, while there are treatments for associated symptoms, there are no FDA-approved drugs specifically targeting social communication deficits in ASD.

What other types of research exist?

Promising novel alternatives to ML-004 for treating social communication deficits in ASD include intranasal oxytocin, which has been studied in a randomized, placebo-controlled trial, and ORG 2766, an ACTH-(4-9) analog, which showed some individual improvements in hyperactivity and other behaviors in a controlled trial. Further research is needed to confirm their efficacy and safety.

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ML-004 for Autism Spectrum Disorder

Phase 2

Recruiting

Research Sponsored by MapLight Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 12 to 45 at screening

Be younger than 65 years old

Must not have

History of epilepsy without current adequate control, or any seizure in the 6 months preceding screening

If female, is pregnant or lactating

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 110

Awards & highlights

Summary

This trial is testing a treatment called ML-004 to see if it can help improve social communication skills in adolescents and adults with Autism Spectrum Disorder (ASD). The study will involve about 150 participants to determine its effectiveness.

Who is the study for?

This trial is for adolescents and adults aged 12 to 45 with Autism Spectrum Disorder (ASD) who can swallow pills. Participants need a care partner to report on symptoms, an IQ score of at least 70, and stable use of psychoactive meds for the past month. Excluded are those with certain other disorders, recent seizures or study participation, history of severe suicidal thoughts or actions, uncontrolled high blood pressure, or if pregnant/lactating.

What is being tested?

The study tests ML-004 in tablet form against a placebo to see its effects on ASD. It's randomized meaning participants are put into the ML-004 or placebo group by chance and double-blind so neither researchers nor participants know who gets what treatment until after the results are collected.

What are the potential side effects?

Potential side effects aren't specified here but generally could include reactions similar to other medications affecting mood or cognition such as headaches, nausea, sleep disturbances or behavioral changes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 12 and 45 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had seizures or uncontrolled epilepsy in the last 6 months.

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 110

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 110

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 110 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score

Secondary study objectives

Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score

Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score

Change from Baseline in Clinician Global Impression of Improvement (CGI-I)

+9 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

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).

Group II: ML-004 PlaceboPlacebo Group1 Intervention

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).

0 / 3Eligibility criteria met

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, including social communication deficits. Pharmacological treatments, such as those being studied in the ML-004 trial, aim to modulate neurotransmitter systems to improve social interactions and communication. For instance, medications may target the serotonin or glutamate systems to enhance social behavior and reduce repetitive behaviors. Behavioral interventions, such as Applied Behavior Analysis (ABA), focus on reinforcing positive social behaviors and communication skills through structured, repetitive tasks and positive reinforcement. These treatments are crucial for ASD patients as they address the fundamental challenges in social communication, thereby improving their ability to interact effectively and enhancing their overall quality of life.

Pharmacological and dietary-supplement treatments for autism spectrum disorder: a systematic review and network meta-analysis.What are we targeting when we treat autism spectrum disorder? A systematic review of 406 clinical trials.Neural Mechanisms of Qigong Sensory Training Massage for Children With Autism Spectrum Disorder: A Feasibility Study.