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).

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Autism Spectrum Disorder
• Intervention / Treatment: Drug: ML-004 (IR)/(ER) tablet

Detailed Description

ML-004-003 is a Phase 2, multi-center, open-label extension study to assess the safety and tolerability in approximately 120 adolescent and adult subjects (age 12 years to 45 years) with ASD who completed the antecedent Phase 2, double-blind, placebo-controlled, study ML-004-002.

The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD.

Subjects will enroll into the study within 3 months of completing antecedent study ML-004-002. Treatment will be once daily and begin with a 12 day dose-titration phase until the maximum allowable tolerated dose is reached. This dose will become the maintenance dose (MD) for that subject, and the subject will remain on this dose for 50 weeks, followed by a down titration.

• Study Type: Interventional
• Enrollment (Estimated): 120
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Camperdown, New South Wales, Australia, 2050
      • Brain and Mind Centre
  • Victoria
    • Parkville, Victoria, Australia, 3052
      • The Royal Children's Hospital, Murdoch Children's Research Institute
• United States
  • Alabama
    • Dothan, Alabama, United States, 36303
      • Harmonex Neuroscience Research
  • Arizona
    • Phoenix, Arizona, United States, 85006
      • Southwest Autism Research and Resource Center
  • Florida
    • Tampa, Florida, United States, 33613
      • University of South Florida Psychiatry and Behavioral Neurosciences
  • Missouri
    • Columbia, Missouri, United States, 65211
      • University of Missouri, Thompson Center for Autism & Neurodevelopment
  • New York
    • Staten Island, New York, United States, 10314
      • Richmond Behavioral Associates
  • Pennsylvania
    • Media, Pennsylvania, United States, 19063
      • Suburban Research Associates
  • Texas
    • Houston, Texas, United States, 77090
      • Red Oak Psychiatry Associates, PA
    • San Antonio, Texas, United States, 78249
      • Road Runner Research, Ltd.
  • Utah
    • Draper, Utah, United States, 84020
      • Cedar Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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
• Able to swallow study medication

Exclusion Criteria:

• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: ML-004 (IR)/(ER) tablet; 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).

Drug: ML-004 (IR)/(ER) tablet; Participants will receive ML-004 once daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Frequency of occurrence of treatment-emergent adverse events (TEAEs).	Baseline up to Day 362

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: MapLight Therapeutics

Publications and helpful links

Helpful Links

• ML-004-002

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2023
• Primary Completion (Estimated): May 1, 2026
• Study Completion (Estimated): June 1, 2026

Study Registration Dates

• First Submitted: May 22, 2023
• First Submitted That Met QC Criteria: June 1, 2023
• First Posted (Actual): June 5, 2023

Study Record Updates

• Last Update Posted (Actual): April 22, 2024
• Last Update Submitted That Met QC Criteria: April 19, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: ASD; Autism; Autism Spectrum Disorder; Social Communication
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
• Other Study ID Numbers: ML-004-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No