What is the mechanism of action of this treatment?

Common treatments for Autism Spectrum Disorder (ASD) often involve neurochemical modulation and behavioral interventions. Neurochemical modulation aims to balance neurotransmitter levels in the brain, which can help reduce symptoms like social withdrawal and repetitive behaviors. Behavioral interventions, on the other hand, focus on enhancing social skills, communication, and adaptive behaviors through structured therapy. These treatments are crucial as they can significantly improve the quality of life for individuals with ASD by addressing core symptoms and aiding in better social integration.

What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) that focus on neurochemical modulation or behavioral impact include antipsychotics like risperidone and aripiprazole, which can cause side effects such as weight gain, drowsiness, increased appetite, fatigue, and hormonal changes. Selective serotonin reuptake inhibitors (SSRIs) and stimulants, used off-label, may lead to gastrointestinal issues, sleep disturbances, and increased anxiety. Behavioral therapies are generally safe but can sometimes result in emotional distress or frustration.

What prior FDA approvals exist?

The FDA has approved a few medications for the treatment of Autism Spectrum Disorder (ASD), primarily focusing on managing symptoms rather than the core features of autism. Risperidone and aripiprazole are two antipsychotic medications approved for treating irritability associated with ASD in children and adolescents. These drugs work by modulating neurotransmitter activity in the brain, particularly targeting dopamine and serotonin receptors. While the specific mechanism of action for L1-79 is not detailed, it is likely aimed at similar neurochemical pathways to improve behavioral outcomes in individuals with ASD.

What other types of research exist?

Promising novel alternatives to L1-79 for ASD treatment include: 1) Arbaclofen, a GABA-B receptor agonist that has shown potential in improving social function and reducing anxiety in ASD patients. 2) Balovaptan, a vasopressin V1a receptor antagonist, which has demonstrated improvements in socialization and communication. 3) Bumetanide, a diuretic that reduces intracellular chloride levels, potentially improving core symptoms of ASD by modulating GABAergic signaling. 4) Oxytocin, a hormone that has been studied for its role in enhancing social bonding and reducing repetitive behaviors in ASD. These alternatives are in various stages of clinical trials and have shown promise in addressing core symptoms of ASD.

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L1-79 for Autism

Phase 2

Waitlist Available

Research Sponsored by Yamo Pharmaceuticals LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Able to swallow study medication whole and self-administer medication or have a caregiver administer medication

Must live with a parent/primary caregiver or spend specified time with them

Must not have

Any disease that requires treatment with immunosuppressive drugs

A diagnosis of Fragile-X syndrome or Rett syndrome

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 12

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects are not listed here, generally such trials monitor for any negative reactions ranging from mild discomforts like headaches or nausea to more serious issues that could affect mood, behavior, or physical health.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take pills by myself or have someone who can help me.

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I live with my parent or primary caregiver.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am on medication that weakens my immune system.

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

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I do not have any major health issues that would interfere with the study.

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I need more than 3 medications for conditions related to my autism.

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I do not have any uncontrolled chronic conditions like seizures, heart disease, or asthma.

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I am taking medication that affects my heart's electrical cycle.

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I am taking medications not approved for autism spectrum disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Side effects data

From 2018 Phase 2 trial • 42 Patients • NCT02947048

27%

diarrhoea

18%

crystal urine present

ear infections

blood TSH increased

generalized tonic-clonic seizure

otitis externa

100%

80%

60%

40%

20%

Study treatment Arm

Placebo Blinded (100 mg Cohort)

200 mg Blinded

100 mg Blinded

Placebo Blinded (200 mg Cohort)

100 mg Open

200 mg Open

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: L1-79 200 mg or 300 mg CapsulesExperimental Treatment1 Intervention

1 capsule twice daily

Group II: Placebo CapsulesPlacebo Group1 Intervention

1 capsule twice daily

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

L1-79

2016

Completed Phase 2

~100

0 / 9Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism Spectrum Disorder (ASD) often involve neurochemical modulation and behavioral interventions. Neurochemical modulation aims to balance neurotransmitter levels in the brain, which can help reduce symptoms like social withdrawal and repetitive behaviors. Behavioral interventions, on the other hand, focus on enhancing social skills, communication, and adaptive behaviors through structured therapy. These treatments are crucial as they can significantly improve the quality of life for individuals with ASD by addressing core symptoms and aiding in better social integration.

Measuring reinforcement learning and motivation constructs in experimental animals: relevance to the negative symptoms of schizophrenia.At the crossroads: the intersection of substance use disorders, anxiety disorders, and posttraumatic stress disorder.Dopaminergic modulation of semantic priming in healthy volunteers.