ML-004 Open-Label Extension Study in Adults and Adolescents with Autism Spectrum Disorders (ASD)

MapLight Therapeutics

Status and phase: Invitation-only; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Drug: ML-004 (IR)/(ER) tablet

Study type: Interventional

Funder types: Industry

Identifiers

NCT05889273

ML-004-003

Details and patient eligibility

About

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.

Enrollment: 120 estimated patients

Sex: All

Ages: 12 to 46 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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120 participants in 1 patient group

ML-004 (IR)/(ER) tablet

Experimental group

Description:

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:

Drug: ML-004 (IR)/(ER) tablet