L1-79 for Autism

No longer recruiting at 7 trial locations
UN
TF
Overseen ByTracy Fischer, PharmD
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called L1-79 to determine if it improves autism symptoms in teens and young adults. Participants will receive either L1-79 or a placebo (a pill that resembles the treatment but contains no active ingredients) to compare effects. The trial focuses on the treatment's effectiveness, safety, and potential side effects. Teens and young adults with autism who face noticeable social challenges and can manage daily activities with some assistance might be suitable candidates. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that L1-79 is likely to be safe for humans?

Research has shown that L1-79 has been tested for safety in people with autism spectrum disorder (ASD). In one study, 58 participants aged 12-21 took part in a 12-week trial. Neither the participants nor the researchers knew who received the actual treatment, while some participants received a placebo.

Initial results suggest that L1-79 is generally well-tolerated. Another study, which also included a placebo group, examined safety and found no severe side effects, although specific details on minor side effects were not provided.

L1-79 is still undergoing clinical trials. Researchers have gathered some safety information, but they continue to study it to ensure its safety for everyone.12345

Why do researchers think this study treatment might be promising for autism?

Researchers are excited about L1-79 for autism because it offers a new approach by targeting a different biological pathway than most current treatments. While traditional options like behavioral therapy and medications such as antipsychotics primarily manage symptoms, L1-79 aims to address underlying biochemical processes. This novel mechanism could potentially improve core symptoms of autism, providing a more comprehensive treatment option.

What evidence suggests that L1-79 might be an effective treatment for autism?

Research has shown that L1-79, which participants in this trial may receive, might help treat autism spectrum disorder (ASD), particularly with core symptoms like social interaction. Previous patients experienced improvements in these areas, almost doubling the smallest change considered important, suggesting it could lead to meaningful improvements. L1-79 affects a process in the brain related to behavior. Early studies indicate it is generally safe, with only mild side effects that resolved without stopping treatment. However, more detailed studies are needed to confirm these results.34678

Who Is on the Research Team?

TM

Tom Megerian, MD, PhD

Principal Investigator

CMO and Senior VP of Clinical Development

Are You a Good Fit for This Trial?

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
See 9 more

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.
See 14 more

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: L1-79 200 mg or 300 mg CapsulesExperimental Treatment1 Intervention
Group II: Placebo CapsulesPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Yamo Pharmaceuticals LLC

Lead Sponsor

Trials
2
Recruited
100+

Published Research Related to This Trial

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

Citations

A 12-Week Crossover Study to Assess the Efficacy, Safety ...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 ...
2.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/31492568/
Effect of L1-79 on Core Symptoms of Autism Spectrum ...These results suggest L1-79 may be a tolerable and effective treatment for the core symptoms of ASD, which must be confirmed with double-blind studies.
Yamo Pharma L1-79 Shows Strong Phase 2 Autism ResultsThe observed improvements in socialization, nearly double the minimal clinically important difference, suggest the potential for meaningful ...
Effect-of-L1-79-on-Core-Symptoms-of-Autism-Spectrum- ...Three adverse events were reported; symptoms were mild and resolved without change in therapy. Implications: These results suggest L1-79 may be a tolerable and ...
Yamo Pharma Presents Statistically Significant Phase 2 ...L1-79 is the first autism therapy to directly target catecholamine synthesis—an approach now backed by emerging research. “With these compelling ...
NCT05067582 | A 12-Week Crossover Study to Assess the ...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 ...
Safety of L1-79 in Adolescent and Adult Males With AutismThis is a five-arm designed to assess the safety of L1-79 that incorporates 15 prospectively randomized, placebo controlled patients and 5 ...
Effect of L1-79 on Core Symptoms of Autism Spectrum ...These results suggest L1-79 may be a tolerable and effective treatment for the core symptoms of ASD, which must be confirmed with double-blind studies.
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