ML-004 in Adolescents and Adults With Autism Spectrum Disorders (ASD)

MapLight Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: Autism Spectrum Disorder

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT05081245

ML-004-002

Details and patient eligibility

About

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.

Enrollment: 150 estimated patients

Sex: All

Ages: 12 to 45 years old

Volunteers: No Healthy Volunteers

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

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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150 participants in 2 patient groups, including a placebo 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

ML-004 Placebo

Placebo Comparator group

Description:

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:

Drug: ML-004 Placebo