Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05081245




Registration number
NCT05081245
Ethics application status
Date submitted
5/10/2021
Date registered
18/10/2021
Date last updated
29/05/2024

Titles & IDs
Public title
ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)
Scientific title
A Randomized, Double-blind, Parallel Group, Placebo-controlled Study to Investigate the Efficacy, Safety, and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
Secondary ID [1] 0 0
ML-004-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Autistic spectrum disorders
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ML-004 (IR)/(ER) tablet
Treatment: Drugs - ML-004 Placebo

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).

Placebo Comparator: ML-004 Placebo - 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).


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

Treatment: Drugs: ML-004 Placebo
Participants will receive matching placebo once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in Autism Behavior Inventory (ABI)-Social Communication Domain Score
Timepoint [1] 0 0
Baseline up to Day 110
Secondary outcome [1] 0 0
Change from Baseline in Clinician Global Impression of Improvement (CGI-I)
Timepoint [1] 0 0
Day 110
Secondary outcome [2] 0 0
Change from Baseline in Autism Behavior Inventory-Clinician (ABI-C) Score
Timepoint [2] 0 0
Baseline up to Day 110
Secondary outcome [3] 0 0
Change from Baseline in Aberrant Behavior Checklist 2-Irritability (ABC-I) Subscale Score
Timepoint [3] 0 0
Baseline up to Day 110
Secondary outcome [4] 0 0
Change from baseline in the Clinician Global Impression of Severity (CGI-S) Score
Timepoint [4] 0 0
Baseline up to Day 110
Secondary outcome [5] 0 0
Change from baseline in the ABI Repetitive/Restrictive Behavior Domain Score
Timepoint [5] 0 0
Baseline up to Day 110
Secondary outcome [6] 0 0
Change from baseline in the ABI Mood and Anxiety Domain Score
Timepoint [6] 0 0
Baseline up to Day 110
Secondary outcome [7] 0 0
Change from baseline in the ABI Challenging Behavior Domain Score
Timepoint [7] 0 0
Baseline up to Day 110
Secondary outcome [8] 0 0
Change from baseline in the ABI Self-regulation Domain Score
Timepoint [8] 0 0
Baseline up to Day 110
Secondary outcome [9] 0 0
Change from baseline in the ABI-Short Form (ABI-S) Score
Timepoint [9] 0 0
Baseline up to Day 110
Secondary outcome [10] 0 0
Change from baseline in the ABC-Social Withdrawal (ABC-SW) Subscale Score
Timepoint [10] 0 0
Baseline up to Day 110
Secondary outcome [11] 0 0
Change from baseline in the Social Responsiveness Scale 2 (SRS-2) Score
Timepoint [11] 0 0
Baseline up to Day 110
Secondary outcome [12] 0 0
Change from baseline in the Vineland-3 (Domain Level Version) Score: total of Communication, Socialization, and Maladaptive behavior domains
Timepoint [12] 0 0
Baseline up to Day 110

Eligibility
Key inclusion criteria
- Age 12 to 45 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 through 34 kg/m², inclusive

- Full scale IQ (or equivalent) =55 score, with adequate verbal fluency, as determined
by the Principal Investigator.

- Psychoactive medications and adjunctive therapies are stable for 4 weeks prior to
screening

- Must be able to swallow study medication
Minimum age
12 Years
Maximum age
45 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Has Rett syndrome or Child Disintegrative Disorder

- Has participated in any other study and received any other investigational medication
(other than COVID-19 vaccination) or device within 60 days prior to screening

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

- History of suicidal ideation or behavior in the past 12 months, or a positive response
to C-SSRS questions 4 and/or 5

- Systolic blood pressure =140 mmHg (if adult) or >135 mmHg (if adolescent), or
diastolic blood pressure =90 (if adult) or >85 mmHg (if adolescent), or a clinical
history of uncontrolled or severe hypertension

- If female, is pregnant or lactating

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/09/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/04/2025
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Brain and Mind Centre - Camperdown
Recruitment hospital [2] 0 0
Children's Health Queensland Hospital and Health Service - South Brisbane
Recruitment hospital [3] 0 0
Mater Research - South Brisbane
Recruitment hospital [4] 0 0
Flinders Medical Centre - Adelaide
Recruitment hospital [5] 0 0
The Royal Children's Hospital, Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
5042 - Adelaide
Recruitment postcode(s) [4] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
District of Columbia
Country [6] 0 0
United States of America
State/province [6] 0 0
Florida
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Utah
Country [17] 0 0
United States of America
State/province [17] 0 0
Washington
Country [18] 0 0
Canada
State/province [18] 0 0
British Columbia
Country [19] 0 0
Canada
State/province [19] 0 0
Ontario

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
MapLight Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled
study that will enroll approximately 150 adolescent and adult subjects with ASD. 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05081245
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexis Levine
Address 0 0
Country 0 0
Phone 0 0
+1 650-839-4388
Fax 0 0
Email 0 0
alevine@maplightrx.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05081245