Safety of L1-79 in Adolescent and Adult Males With Autism

NCT02947048 | Completed Phase 2 Results FDA Drug

• 1 other identifier: HT 02-121
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 2

Brief Summary

This is a five-arm designed to assess the safety of L1-79 that incorporates 15 prospectively randomized, placebo controlled patients and 5 open label patients at either 100 tid (three times daily) or 200 tid dosing for 28 days. The open label patients will be assessed for the purpose of understanding PK/PD and to determine if there are any EKG changes associated with the administration of L1-79. Additional safety information will be provided by the 30 patients randomized 2:1 active:placebo.

Conditions

Autism

Outcome Measures

Primary Outcomes (1)

• Adverse Events

  Number of Participants with On-treatment Adverse Events. To be conservative, it was pre-specified that all placebo data be combined (open-label, first cohort, and second cohort) for the safety population. For the efficacy population open-label data was excluded due to potential bias and placebo arms from the first and second cohort were presented separately due to differences in efficacy questionnaires completion in the first and second cohorts. Theses differences were not relevant to the adverse event data.

  4 weeks

Secondary Outcomes (7)

• Change From Baseline in Clinical Global Impression Scale (CGI)

  Week 0 and Week 4

• Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Socialization Standard Score

  Day 0 and Week 4

• Change From Baseline in Vineland Adaptive Behavior Scale - 2nd Edition Communication Standard Score

  Week 0 and Week 4

• Change From Baseline in the Autism Diagnostic Observation Schedule 2nd Edition (ADOS-2) Overall Total Score

  Day 0 and Week 4

• Change From Baseline in the Social Responsiveness Scale - 2nd Edition (SRS-2) Total T-score

  Week 0 and Week 4

Study Arms (5)

100 mg open

EXPERIMENTAL

open-label lead-in 100 mg L1-79 t.i.d.

Drug: L1-79

100 mg blinded

EXPERIMENTAL

blinded and randomized 100 mg L1-79 t.i.d.

Drug: L1-79

200 mg open

EXPERIMENTAL

open-label lead-in 200 mg L1-79 t.i.d.

Drug: L1-79

200 mg blinded

EXPERIMENTAL

blinded and randomized 200 mg L1-79 t.i.d.

Drug: L1-79

Placebo

PLACEBO COMPARATOR

placebo t.i.d.

Drug: Placebo

Interventions

L1-79 DRUG

Also known as: D,L-α-Methyltyrosine

100 mg blinded; 100 mg open; 200 mg blinded; 200 mg open

Placebo DRUG

Placebo

Eligibility Criteria

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

You may qualify if:

• Males who are not sexually active
• and 21 years of age
• Signed informed consent
• Normal clinical laboratory values
• DSM-5 compliant diagnosis of autism spectrum disorder, confirmed by the Autistic Diagnosis Interview Review (ADIR), and by the Autism Diagnosis Observation Schedule (ADOS) score consistent with a diagnosis of autism
• No more than one concomitant medication for the treatment of autism, on a stable for at least 2 weeks prior to enrollment and no planned changes in psychosocial interventions during the trial
• No medications for any other pathology

You may not qualify if:

• Any co-morbidities, including Fragile-X syndrome, epilepsy, Retts syndrome, ADHD, or other disease or syndrome aside from autism that requires treatment
• Any other psychiatric disorder, or out of range lab values
• DSM-5 diagnosis of schizophrenia, schizoaffective disorder, alcohol use disorder
• Active medical problems: unstable seizures (\>2 in past month)
• Concomitant physical illness

Sponsors & Collaborators

• Yamo Pharmaceuticals LLC: lead

Study Sites (2)

Eric Bartky MD, Bartky Health Care Center

Livingston, New Jersey, 07039, United States

Location

F. Peter Halas MD, Sea Girt Pediatrics

Sea Girt, New Jersey, 07850, United States

Location

MeSH Terms

Conditions

Autistic Disorder

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Limitations and Caveats

The study enrolled a small number of patients and was not powered to detect statistically significant changes in efficacy parameters. Also note that assessments (except CGI and ADOS-2) for 100 mg cohort were completed by caregivers at home. Due to missing/incomplete data for endpoints completed at home, study procedures changed for 200 mg cohort and assessments were completed in clinic.

Results Point of Contact

• Title: Tracy Fischer
• Organization: Yamo Pharmaceuticals

tfischer@yamopharma.com

8596855862

Study Officials

• John Rothman, PhD

  Yamo Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

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

Study Record Dates

• First Submitted: May 25, 2016
• First Posted: October 27, 2016
• Study Start: October 1, 2016
• Primary Completion: December 1, 2017
• Study Completion: February 1, 2018
• Last Updated: August 21, 2024
• Results First Posted: August 21, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)