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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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05067582
Recruitment Status : Recruiting
First Posted : October 5, 2021
Last Update Posted : March 9, 2022
Sponsor:
Information provided by (Responsible Party):
Yamo Pharmaceuticals LLC

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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Autism Drug: L1-79 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description: Two Period Crossover
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Chronic Dosing (12-weeks), Two-Period, Placebo-Controlled, Crossover, Multi-Center Study to Assess the Efficacy, Safety, and Tolerability of Two Doses of L1-79 for the Treatment of the Core Deficits in Social-Communication Interaction in Adolescents and Young Adults With Autism Spectrum Disorder
Actual Study Start Date : January 25, 2022
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : March 2023


Arm Intervention/treatment
Placebo Comparator: Placebo Capsules
1 capsule twice daily
Drug: L1-79
tyrosine hydroxylase inhibitor
Other Name: DL-alpha-Methyltyrosine

Experimental: L1-79 200 mg or 300 mg Capsules
1 capsule twice daily
Drug: L1-79
tyrosine hydroxylase inhibitor
Other Name: DL-alpha-Methyltyrosine




Primary Outcome Measures :
  1. Vineland Adaptive Behavior Scale, Third Edition (Vineland-3), Average of the Growth Scale Value (GSV) score of the three Socialization Subdomains (combined) [ Time Frame: Week 12 ]

Secondary Outcome Measures :
  1. Brief Observation of Social Communication Change (BOSCC) [ Time Frame: Week 12 ]
  2. Clinical Global Impression of Severity of Illness (CGI-S) weighted for socialization [ Time Frame: Week 12 ]
  3. Clinical Global Impression of Change (CGI-C) weighted for socialization [ Time Frame: Week 12 ]
  4. Percent of subjects showing a statistically significant improvement in GSV on 2 of the Socialization Subdomains [ Time Frame: Week 12 ]
  5. Caregiver Global Impression of Change of 3 Most Bothersome Symptoms of ASD (CGI-3P) [ Time Frame: Week 12 ]
  6. Social Responsiveness Scale, Second Edition (SRS-2) Social-Communication and Interaction - DSM-5 Composite T-score [ Time Frame: Week 12 ]
  7. Social Responsiveness Scale, Second Edition (SRS-2) Total T-score [ Time Frame: Week 12 ]
  8. Social Responsiveness Scale, Second Edition (SRS-2) Social Motivation T-score [ Time Frame: Week 12 ]
  9. Vineland-3 Socialization Domain, Standard Score [ Time Frame: Week 12 ]
  10. Parent-rated Anxiety Scale for ASD (PRAS-ASD) [ Time Frame: Week 12 ]
  11. Child's Sleep Habits Questionnaire (CSHQ) [ Time Frame: Week 12 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female adolescents or young adults between 12 and 21 years of age.
  • WASI-II standard score ≥70 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.

Exclusion Criteria:

  • 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.
  • Any subject or caregiver who is unwilling or unable to give informed consent.
  • Participated in an investigational drug study within 90 days prior to Baseline.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05067582


Contacts
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Contact: Uyen Nguyen 949-769-0046 unguyen@yamopharma.com
Contact: Tracy Fischer, PharmD 859-685-5862 tfischer@yamopharma.com

Locations
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United States, Arizona
Southwest Autism Research and Resource Center Recruiting
Phoenix, Arizona, United States, 85006
Contact: Zakiyyah Merritt    480-701-3681    zmerritt@autismcenter.org   
Principal Investigator: Raun Melmed, MD         
United States, California
Thompson Autism Center CHOC Recruiting
Orange, California, United States, 92868
Contact: Julie Hernandez    714-288-7683    jhernandez11@choc.org   
Principal Investigator: Sailaja Golla, MD         
United States, Illinois
Rush University Recruiting
Chicago, Illinois, United States, 60612
Contact: Aneta Kwak       Aneta_Z_Kwak@rush.edu   
Principal Investigator: Cesar Ochoa-Lubinoff, MD         
United States, Missouri
Thompson Center for Autism and Neurodevelopmental Disorders Recruiting
Columbia, Missouri, United States, 65211
Contact: Kathy Hirst    573-882-5643    hirstka@missouri.edu   
Principal Investigator: David Beversdorf, MD         
United States, Texas
Red Oak Psychiatry Associates Recruiting
Houston, Texas, United States, 77090
Contact: Thomas Vu    281-893-4111 ext 249    ropa29@earthlink.net   
Principal Investigator: Lawrence Ginsberg, MD         
Sponsors and Collaborators
Yamo Pharmaceuticals LLC
Investigators
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Study Director: Tom Megerian, MD, PhD CMO and Senior VP of Clinical Development
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Responsible Party: Yamo Pharmaceuticals LLC
ClinicalTrials.gov Identifier: NCT05067582    
Other Study ID Numbers: Y202
First Posted: October 5, 2021    Key Record Dates
Last Update Posted: March 9, 2022
Last Verified: January 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
alpha-Methyltyrosine
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action