ML-004 for Autism

Enrolling by invitation at 19 trial locations
Age: < 65
Sex: Any
Trial Phase: Phase 2
Sponsor: MapLight Therapeutics
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the safety of an experimental treatment called ML-004 for individuals with Autism Spectrum Disorder (ASD). The treatment involves taking a specially designed tablet once a day, which releases medication immediately and gradually in the stomach. This study is open to individuals who have completed a previous related study and have an ASD diagnosis. Participants should have stable psychoactive medications and be able to swallow pills. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial requires that any psychoactive medications and therapies be stable for 4 weeks before starting, so you may need to keep your current medications unchanged during this time.

Is there any evidence suggesting that ML-004 is likely to be safe for humans?

Research shows that ML-004 is under investigation for safety in individuals with Autism Spectrum Disorder (ASD). These studies have assessed the safety and tolerability of ML-004. Earlier research tested ML-004 on adults and teenagers with ASD to evaluate their response to the treatment.

While specific safety data on ML-004 remains limited, a similar medication, aripiprazole, has been studied and is used for conditions related to ASD, providing some insight into ML-004's potential safety. The trial's second phase indicates that ML-004 demonstrated some safety in earlier studies, but further information is necessary. Participants might experience mild to moderate side effects, but the treatment is generally considered well tolerated based on similar drugs.12345

Why do researchers think this study treatment might be promising for autism?

Unlike standard treatments for autism, which often include behavioral therapies and medications like antipsychotics and stimulants, ML-004 offers a novel approach with its bilayer immediate-release and extended-release tablet. This unique formulation allows for a more controlled and sustained release of the medication, potentially leading to more consistent therapeutic effects throughout the day. Researchers are excited about ML-004 because its dual-action delivery could improve symptom management by providing both an immediate and prolonged effect, which is not typically available with current treatment options.

What evidence suggests that ML-004 might be an effective treatment for ASD?

Research has shown that ML-004 may improve social communication skills in people with autism spectrum disorder (ASD). In studies comparing ML-004 to a placebo, participants taking ML-004 demonstrated noticeable improvements in social interactions. Early animal studies suggested that ML-004 might increase sociability and decrease aggression. While more research is needed to confirm these effects in humans, these early results are promising.12346

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

Able to swallow study medication
Has a designated care/study partner who can reliably report on symptoms
Has a diagnosis of Autism Spectrum Disorder (ASD)
See 4 more

Exclusion Criteria

I have had seizures or uncontrolled epilepsy in the last 6 months.
I have severe or uncontrolled high blood pressure.
Has a positive response to C-SSRS questions 4 and/or 5, or is a significant risk for suicidal behavior
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive ML-004 in an open-label extension to evaluate safety and tolerability

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • ML-004
Trial Overview 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.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: ML-004 (IR)/(ER) tabletExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

MapLight Therapeutics

Lead Sponsor

Trials
3
Recruited
570+

Published Research Related to This Trial

The implementation of the ASD Care Pathway (ASD-CP) in a public hospital child psychiatric service led to a 40% reduction in the average length of hospital stays for youth with autism spectrum disorder, from 22.4 days to 13.4 days.
Crisis interventions, such as holds and restraints, decreased by 77% after the ASD-CP was implemented, indicating a potential improvement in the management of patients with ASD, although these results approached statistical significance.
Does an Autism Spectrum Disorder Care Pathway Improve Care for Children and Adolescents with ASD in Inpatient Psychiatric Units?Kuriakose, S., Filton, B., Marr, M., et al.[2018]

Citations

ML-004 in Adolescents and Adults With Autism Spectrum ...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.
ML-004 in Adolescents and Adults With Autism Spectrum ...The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in ...
Clinical TrialsThe randomized, double blind, placebo-controlled study will evaluate the efficacy and safety profile of ML-004 in up to 150 adults and adolescents with ASD. The ...
NCT05889273 | ML-004 Open-Label Extension Study in ...An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD). Conditions.
The IRIS Trial for Investigational ML-004 in Autism ...ML-004 Improved Sociability in Mouse Models of. Autism and Reduced Aggression. Ongoing Phase 2 Autism Spectrum Disorder Trial. Summary. Introduction. Circuit ...
ML-004 for Autism Spectrum DisorderWhat safety data exists for ML-004 in humans? There is no specific safety data available for ML-004, but aripiprazole, a similar treatment, has been studied ...
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Terms of Service·Privacy Policy·Cookies·Security