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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/NCT05889273




Registration number
NCT05889273
Ethics application status
Date submitted
22/05/2023
Date registered
5/06/2023
Date last updated
29/05/2024

Titles & IDs
Public title
ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)
Scientific title
An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
Secondary ID [1] 0 0
ML-004-003
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

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).


Treatment: Drugs: ML-004 (IR)/(ER) tablet
Participants will receive ML-004 once daily.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Timepoint [1] 0 0
Baseline up to Day 362
Secondary outcome [1] 0 0
Frequency of occurrence of Serious Adverse Events (SAEs)
Timepoint [1] 0 0
Baseline up to Day 362
Secondary outcome [2] 0 0
Frequency of occurrence of TEAEs leading to discontinuation.
Timepoint [2] 0 0
Baseline up to Day 362
Secondary outcome [3] 0 0
Frequency of occurrence of TEAEs arising from clinically important changes in other safety assessments.
Timepoint [3] 0 0
Baseline up to Day 362
Secondary outcome [4] 0 0
Frequency of occurrence of suicidal ideation and behavior as assessed by the Columbia-Suicide Severity Rating Scale.
Timepoint [4] 0 0
Baseline up to Day 362

Eligibility
Key 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
Minimum age
12 Years
Maximum age
46 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
1/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Brain and Mind Centre - Camperdown
Recruitment hospital [2] 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
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
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Utah
Country [9] 0 0
Canada
State/province [9] 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-004-003 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05889273
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05889273