L1-79 for Autism

Recruiting in Columbus (<10 mi)

+7 other locations

Overseen By Tracy Fischer, PharmD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Yamo Pharmaceuticals LLC

Must not be taking: Alpha-2 agonists, Beta-blockers

Disqualifiers: Pregnancy, Schizophrenia, Bipolar, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

58 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests L1-79 to see if it helps young people with Autism Spectrum Disorder (ASD) who have certain levels of intelligence and socialization issues. The goal is to find out if L1-79 can improve their symptoms.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, such as antipsychotics, alpha-2 agonists, beta-blockers, and anti-hypertensives, at least 1 to 2 months before starting the study. If you're on more than 3 medications for conditions like autism, ADHD, or anxiety, you may not be eligible to participate.

How does the drug L1-79 for autism differ from other treatments?

L1-79, also known as LY293558, is unique because it acts as an AMPA receptor antagonist, which means it blocks certain receptors in the brain that are involved in excitatory neurotransmission. This mechanism is different from many existing autism treatments, which often focus on behavioral therapy or medications targeting other neurotransmitter systems. ^{1 2 3 4 5}

Research Team

Tom Megerian, MD, PhD

Principal Investigator

CMO and Senior VP of Clinical Development

Eligibility Criteria

This trial is for young people aged 12-21 with Autism Spectrum Disorder (ASD). They must have certain intelligence and severity scores, be able to swallow medication, and either live with a caregiver or spend significant time with one. Participants need to agree to use contraception if applicable.

Inclusion Criteria

Diagnosis of ASD based on tool that utilizes the DSM-5 criteria, confirmed with ADOS-2

I can follow the trial's requirements as per my doctor's opinion.

WASI-II standard score ≥70 at screening or within the last 12 months prior to screening

Exclusion Criteria

I am on medication that weakens my immune system.

Presence of out-of-range hepatic or renal function tests or other unexplained abnormal laboratory value deemed clinically significant

I have been diagnosed with Fragile-X syndrome or Rett syndrome.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive L1-79 or placebo capsules twice daily in a 12-week crossover design

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• L1-79

Trial Overview The study tests the effectiveness of L1-79 on ASD in adolescents. It's a 12-week crossover study, meaning participants will receive both the treatment and placebo at different times during the trial to compare effects.

Participant Groups

Group I: L1-79 200 mg or 300 mg Capsules Experimental Treatment 1 Intervention

1 capsule twice daily

Group II: Placebo Capsules Placebo Group 1 Intervention

1 capsule twice daily

Who Is Running the Clinical Trial?

Yamo Pharmaceuticals LLC

Lead Sponsor

Trials

2

Recruited

100+

Findings from Research

In a study involving 76 newborn piglets, the AMPA antagonist LY293558 did not show any significant effect on reducing the severity of hypoxic-ischemic brain injury when administered at two different doses (5 mg/kg and 15 mg/kg).

The results suggest that neither AMPA receptor activity nor NMDA receptor activity plays a crucial role in brain injury in this newborn model, indicating that alternative therapeutic strategies may be needed.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

AMPA antagonist LY293558 does not affect the severity of hypoxic-ischemic injury in newborn pigs. LeBlanc, MH., Li, XQ., Huang, M., et al.[2019]

References

1. England pubmed.ncbi.nlm.nih.gov

LY293558 prevents soman-induced pathophysiological alterations in the basolateral amygdala and the development of anxiety. [2021]

2. United States pubmed.ncbi.nlm.nih.gov

AMPA antagonist LY293558 does not affect the severity of hypoxic-ischemic injury in newborn pigs. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

The neuroprotective effect of the glycine site antagonist 3R-(+)-cis-4-methyl-HA966 (L-687,414) in a rat model of focal ischaemia. [2003]

4. England pubmed.ncbi.nlm.nih.gov

NMDA receptor channels: labeling of MK-801 with iodine-125 and fluorine-18. [2019]

5. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Targeted Blockade of TARP-γ8-Associated AMPA Receptors: Anticonvulsant Activity with the Selective Antagonist LY3130481 (CERC-611). [2019]