ML-004 for Autism

Recruiting at 17 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: MapLight Therapeutics

Disqualifiers: Rett syndrome, Epilepsy, Hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

120 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing the safety of a treatment called ML-004 in adolescents and adults with Autism Spectrum Disorder (ASD). The main goal is to ensure that the treatment does not cause any harmful side effects.

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.

What data supports the effectiveness of the drug ML-004 for treating autism?

Research on clomipramine, a drug used in adults with pervasive developmental disorders (which include autism), showed that 55% of patients improved significantly during a 12-week trial. This suggests that similar drugs might be effective for autism. ^{1 2 3 4 5}

Eligibility Criteria

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

Has completed Study ML-004-002 within the past 90 days

Has a designated care/study partner who can reliably report on symptoms

Has a diagnosis of Autism Spectrum Disorder (ASD)

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

Timeline

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

Treatment Details

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.

Participant Groups

Group I: ML-004 (IR)/(ER) tablet Experimental Treatment 1 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).

Who Is Running the Clinical Trial?

MapLight Therapeutics

Lead Sponsor

Trials

3

Recruited

570+

Findings from Research

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Clomipramine in adults with pervasive developmental disorders: a prospective open-label investigation. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Does an Autism Spectrum Disorder Care Pathway Improve Care for Children and Adolescents with ASD in Inpatient Psychiatric Units? [2018]

3. United States pubmed.ncbi.nlm.nih.gov

Investigating a Multimodal Intervention for Children With Limited Expressive Vocabularies Associated With Autism. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Integrating Measurement-Based Care into Treatment for Autism Spectrum Disorder: Insights from a Community Clinic. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Treatment outcome and outcome associations in children with pervasive developmental disorders treated with selective serotonin reuptake inhibitors: a chart review. [2022]