A 12-Week Crossover Study to Assess the Efficacy, Safety and Tolerability of L1-79 in Subjects Aged 12-21 Years With Autism Spectrum Disorder

NCT05067582 | Completed Phase 2 FDA Drug

• 1 other identifier: Y202
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 8

Brief Summary

This study will investigate the efficacy, safety and tolerability of L1-79 in participants aged 12-21 years who have been diagnosed with ASD with a score of \>/= 70 on the Wechsler Abbreviated Scale of Intelligence (WASI-II), and a score of \>/= 4 on the Clinical Global Impression of Severity of Illness (CGI-S) weighted for socialization.

Conditions

Autism Spectrum Disorder; Autism

Outcome Measures

Primary Outcomes (1)

• Vineland Adaptive Behavior Scale, Third Edition (Vineland-3), Responder Analysis

  at least 1 point improvement in 2/3 socialization subdomains or at least 3 point improvement in socialization domain

  Week 12

Secondary Outcomes (16)

• Vineland-3 Socialization Domain, Standard Score

  Week 12

• V-scale Score for Each of the 3 Socialization Subdomains of the Vineland-3

  Week 12

• Clinical Global Impression of Severity of Illness (CGI-S) Weighted for Socialization

  Week 12

• Clinical Global Impression of Change (CGI-C) Weighted for Socialization

  Week 12

• Growth Scale Value (GSV) of Each of the 3 Socialization Subdomains of the Vineland-3

  Week 12

Study Arms (2)

Placebo Capsules

PLACEBO COMPARATOR

1 capsule twice daily

Drug: L1-79

L1-79 200 mg or 300 mg Capsules

EXPERIMENTAL

1 capsule twice daily

Drug: L1-79

Interventions

L1-79 DRUG

tyrosine hydroxylase inhibitor

Also known as: DL-alpha-Methyltyrosine

L1-79 200 mg or 300 mg Capsules; Placebo Capsules

Eligibility Criteria

• Age: 12 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female adolescents or young adults between 12 and 21 years of age.
• WASI-II standard score with 95% CI containing 70 or greater at screening or within the last 12 months prior to screening.
• Fulfill language criteria required to complete ADOS-2 Modules 2, 3 or 4.
• Diagnosis of ASD based on tool that utilizes the DSM-5 criteria, confirmed with ADOS-2.
• CGI-S (weighted for socialization) of 4 or greater.
• A female is eligible to enter and participate in the study if she is of non-childbearing potential or childbearing potential, has negative pregnancy test at screening and, if sexually active, agrees to use acceptable contraception methods (defined in protocol), for the duration of the study and for at least 30 days after the last dose of study drug.
• Male subjects if sexually active and female partners of childbearing potential must agree to use acceptable contraceptive methods (defined in protocol), for the duration of the study and for at least 30 days after the last dose of study drug.
• Subjects and caregiver must be willing and able to participate in the testing procedures sufficient to obtain valid scores on the tests used herein.
• Must live with a parent/primary caregiver, or if not, during each week he/she must either spend at least 3 hours a day for at least 4 days or, spend the weekend with a parent/primary caregiver.
• In the opinion of the Investigator, be sufficiently tolerant and capable of complying with the requirements of this trial.
• Able to swallow study medication whole and self-administer medication if living independently or have a parent/caregiver be able to administer medication.
• Subjects or their legal guardians must be willing to sign informed consent and/or assent and caregivers participating in the study must be willing to sign informed consent.

You may not qualify if:

• Pregnancy or breastfeeding, or intention to become pregnant during the study.
• Uncontrolled inter-current illness including, but not limited to, ongoing or active infection, symptomatic cardio-vascular disease, hepatic disease, renal disease, musculo skeletal or rheumatologic disease, human immunodeficiency virus (HIV), hemorrhagic cerebrovascular accident (HCVA), hepatitis B virus (HBV), or psychiatric illness/social situations that would limit compliance with study requirements.
• Any disease that requires treatment with immunosuppressive drugs.
• A diagnosis of Fragile-X syndrome or Rett syndrome.
• A DSM-5 diagnosis of schizophrenia, schizoaffective disorder, alcohol use disorder, current or lifetime diagnosis of severe psychiatric disorder (e.g., bipolar disorder, etc.).
• Subjects at risk of suicidal behavior or with a history alcohol or substance abuse/dependence.
• Presence of any active chronic medical problem including, but not limited to uncontrolled: seizure disorder, heart disease, cancer, asthma, genetic disease.
• Requiring more than 3 medications for the treatment of autism, ADHD, seizures, depression, anxiety, aggression, agitation, obsessive compulsive disorder, tic disorder, or other disorder commonly co-occurring with ASD.
• Initiation of new or major change in psychosocial intervention within 12 weeks prior to screening and throughout the duration of the study.
• School or academic setting are expected to change during the course the study.
• Clinically significant ECG abnormalities including subjects with baseline QTc prolongation (QTcF \>450 msec for males and \>470 msec in females).
• On concomitant medications known to prolong the QTc interval.
• Presence of out of range hepatic or renal function tests or other unexplained abnormal laboratory value that is deemed clinically significant by the Investigator.
• On any of the following medications: alpha-2 agonists (including, but not limited to clonidine and guanfacine), beta-blockers, anti-hypertensives, and antipsychotics not approved for use in ASD.
• Taking disallowed concomitant medications within 2 months (antipsychotics) and 1 month (all other medications) prior to Baseline.

Sponsors & Collaborators

• Yamo Pharmaceuticals LLC: lead

Study Sites (8)

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

Thompson Autism Center CHOC

Orange, California, 92868, United States

Location

Cortica

San Rafael, California, 94093, United States

Location

Rush University

Chicago, Illinois, 60612, United States

Location

Thompson Center for Autism and Neurodevelopmental Disorders

Columbia, Missouri, 65211, United States

Location

Center for Autism and The Developing Brain

White Plains, New York, 10032, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Red Oak Psychiatry Associates

Houston, Texas, 77090, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Tom Megerian, MD, PhD

  CMO and Senior VP of Clinical Development

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Two Period Crossover

Study Record Dates

• First Submitted: September 22, 2021
• First Posted: October 5, 2021
• Study Start: January 25, 2022
• Primary Completion: June 18, 2024
• Study Completion: June 21, 2024
• Last Updated: July 31, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (8)