An Open-label Extension Study to Investigate Safety and Tolerability of ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD).
Trial Snapshot
- Phase
- Phase 2
- Status
- Active, not recruiting
- Sponsor
- MapLight Therapeutics
- Enrollment
- 120
- Locations
- 37
- Primary Endpoint
- Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to 46 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
- •Must be 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
Arms & Interventions
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).
Intervention: ML-004 (IR)/(ER) tablet (Drug)
Outcomes
Primary Outcomes
Frequency of occurrence of treatment-emergent adverse events (TEAEs).
Time Frame: Baseline up to Day 362
Secondary Outcomes
- 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)
- 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)