Children's National

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

Purpose

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.

Condition

Autism Spectrum Disorder

Eligibility

Eligible Ages: Between 12 Years and 45 Years
Eligible Genders: All
Accepts Healthy Volunteers: No

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

Study Design

Phase: Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Multi-center, randomized, double-blind, placebo-controlled study
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-blind

Arm Groups

Arm	Description	Assigned Intervention
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).	Drug: ML-004 (IR)/(ER) tablet Participants will receive ML-004 once daily.
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).	Drug: ML-004 Placebo Participants will receive matching placebo once daily.

Recruiting Locations

Children's National and nearby locations

Children's National Health System - The Children's Research Institute (CRI)
Washington, District of Columbia 20010

Contact:
Adelaide Robb, MD
202-476-3042

More Details

NCT ID: NCT05081245
Status: Recruiting
Sponsor: MapLight Therapeutics

Study Contact

Alexis Levine
+1 650-839-4388
alevine@maplightrx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.