What is the mechanism of action of this treatment?

The most common treatments for Lou Gehrig's Disease (ALS) include autophagy activators, such as LAM-002A, which aim to enhance the cellular process of autophagy. Autophagy helps in the degradation and recycling of damaged cellular components, potentially reducing the accumulation of toxic proteins that contribute to motor neuron degeneration in ALS. Other treatments, like antisense oligonucleotides (e.g., Tofersen), target specific genetic mutations to reduce the production of harmful proteins. These mechanisms are crucial for ALS patients as they address the underlying cellular dysfunctions, potentially slowing disease progression and improving quality of life.

What side effects are associated with this type of intervention?

Common treatments for Lou Gehrig's Disease (ALS) include riluzole and edaravone. Riluzole can cause side effects such as nausea, weakness, decreased lung function, and liver damage. Edaravone may lead to bruising, gait disturbances, and allergic reactions. Treatments focusing on autophagy modulation, like the investigational LAM-002A, may have side effects related to immune response and cellular metabolism, but specific data on LAM-002A's side effects are not yet available.

What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Lou Gehrig's Disease (ALS). Riluzole, approved in 1995, is believed to reduce damage to motor neurons by decreasing the release of glutamate. Edaravone, approved in 2017, is thought to act as an antioxidant, reducing oxidative stress in neurons. While these drugs do not specifically target autophagy, they aim to protect neurons from damage. Currently, there are no FDA-approved ALS treatments that function as autophagy activators like LAM-002A, which is being studied for its potential to enhance cellular cleanup processes and improve neuronal health.

What other types of research exist?

Promising novel alternatives to LAM-002A for ALS patients include sodium phenylbutyrate/taurursodiol, antisense oligonucleotide Tofersen for SOD1 ALS, and oral levosimendan. These treatments have demonstrated potential in clinical trials and may offer new therapeutic options for ALS management in 2024.

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Unknown

LAM-002A for ALS

Phase 2

Waitlist Available

Led By Suma Babu, M.D.

Research Sponsored by AI Therapeutics, Inc.

Eligibility Criteria Checklist

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

Must have

Diagnosis of C9ORF72-associated ALS with documentation of a clinical genetic test demonstrating the presence of a pathogenic repeat expansion in C9ORF72

Vital Capacity greater than and equal to 50% of predicted at the time of the Screening Visit

Must not have

Presence of a neurodegenerative cognitive or motor syndrome not related to the C9ORF72 repeat expansion

Renal profile showing reduced kidney function

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 28 weeks

Summary

This trial is testing a medication called LAM-002A in adults with a specific genetic form of ALS. The goal is to see if the medication is safe and if it helps with the disease. Participants will take the medication and be monitored for any effects.

Who is the study for?

Adults with C9ORF72-associated ALS who can consent, swallow capsules, and have a vital capacity ≥50% of predicted. They must not be on certain medications or treatments for ALS, have unstable medical conditions besides ALS, active cancer (with exceptions), or severe liver/kidney issues. Men and women must agree to use contraception.

What is being tested?

The trial is testing LAM-002A's safety and biological effect in C9ALS patients compared to a placebo. Participants will receive either the drug or placebo while being monitored for tolerance and any changes in their condition.

What are the potential side effects?

While specific side effects are not listed here, participants will be closely observed for any adverse reactions related to LAM-002A or the placebo during the trial period.

Eligibility Criteria

Inclusion Criteria

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

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I have ALS linked to a C9ORF72 gene mutation.

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My lung function is at least half of what is expected for someone my age and size.

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I am 18 years old or older.

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I can safely swallow capsules.

Exclusion Criteria

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

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I have a brain disorder not caused by C9ORF72 gene changes.

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My kidneys are not working as well as they should.

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I am currently on medication to suppress my immune system.

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I do not have any unstable health conditions that could put me at risk.

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I am a male and unwilling to use contraception.

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I am not taking medication that strongly affects liver enzymes.

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I have had a solid organ transplant.

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I am taking medication that is a strong influencer on the CYP2C9 enzyme.

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I cannot have a lumbar puncture due to health reasons.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 28 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~28 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

CSF Pharmacokinetics of LAM-002A

Plasma Pharmacokinetics of LAM-002A

Safety of LAM-002A: occurrence of TEAEs

+1 more

Secondary study objectives

Changes in biomarkers

Tolerability of LAM-002A: completion of open-label study treatment

Other study objectives

Changes in ALS-CBS

Changes in ALSFRS-R

Changes in Vital Capacity

+2 more

Side effects data

From 2023 Phase 1 trial • 62 Patients • NCT02594384

33%

Anal incontinence

33%

Rash maculo-papular

33%

Diarrhoea

33%

Oedema peripheral

33%

Adverse drug reaction

33%

Oedema

33%

Blood phosphorus increased

33%

Upper respiratory tract infection

100%

80%

60%

40%

20%

Study treatment Arm

LAM-002A Continuous Monotherapy - 50 mg BID

LAM-002A Continuous Monotherapy - 125 mg BID

LAM-002A Continuous Monotherapy - 100 mg BID

LAM-002A Continuous Monotherapy - 150 mg BID

LAM-002A Continuous Monotherapy - 75 mg TID

LAM-002A Intermittent Monotherapy - 150 mg BID

LAM-002A + Rituximab

LAM-002A + Atezolizumab

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: LAM-002AExperimental Treatment1 Intervention

LAM-002A will be administered orally in five 25 mg capsules twice a day (250 mg total daily dose).

Group II: PlaceboPlacebo Group1 Intervention

Placebo matching LAM-002A will be administered orally in 5 capsules twice a day.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

LAM-002A

2015

Completed Phase 1

~70

0 / 13Eligibility 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

The most common treatments for Lou Gehrig's Disease (ALS) include autophagy activators, such as LAM-002A, which aim to enhance the cellular process of autophagy. Autophagy helps in the degradation and recycling of damaged cellular components, potentially reducing the accumulation of toxic proteins that contribute to motor neuron degeneration in ALS. Other treatments, like antisense oligonucleotides (e.g., Tofersen), target specific genetic mutations to reduce the production of harmful proteins. These mechanisms are crucial for ALS patients as they address the underlying cellular dysfunctions, potentially slowing disease progression and improving quality of life.

Ganoderma lucidum Modulates Inflammatory Responses following 1-Methyl-4-Phenyl-1,2,3,6-Tetrahydropyridine (MPTP) Administration in Mice.Tizoxanide induces autophagy by inhibiting PI3K/Akt/mTOR pathway in RAW264.7 macrophage cells.Late autophagy inhibitor chloroquine improves efficacy of the histone deacetylase inhibitor SAHA and temozolomide in gliomas.