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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 belowSelect...
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:Select...
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
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 moreSecondary 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 moreSide effects data
From 2023 Phase 1 trial • 62 Patients • NCT0259438486%
Nausea
71%
Constipation
57%
Vomiting
43%
Dizziness
43%
Pyrexia
29%
Dehydration
29%
Pneumonia
29%
Back pain
29%
Cough
29%
Abdominal distension
29%
Ascites
29%
Neutrophil count decreased
29%
Insomnia
29%
Anxiety
14%
Colitis
14%
Diffuse large B-cell lymphoma
14%
White blood cell count decreased
14%
Neoplasm malignant
14%
Lethargy
14%
Hypotension
14%
Pain in extremity
14%
Febrile neutropenia
14%
Muscular weakness
14%
Night sweats
14%
Disease progression
14%
Hemoglobin decreased
14%
Vitamin K deficiency
14%
Fall
14%
Fatigue
14%
Blood potassium decreased
14%
Alanine aminotransferase increased
14%
Nasal congestion
14%
Diplopia
14%
Confusional state
14%
Abdominal pain upper
14%
Spinal cord compression
14%
Femur fracture
14%
Blood creatinine increased
14%
Malnutrition
14%
Headache
14%
Asterixis
14%
Blood bilirubin increased
14%
Pulmonary oedema
14%
Atrial fibrillation
14%
Diarrhoea
14%
Dysphagia
14%
Dyspepsia
14%
Proctalgia
14%
Asthenia
14%
Oedema peripheral
14%
Pain
14%
Non-cardiac chest pain
14%
Platelet count decreased
14%
Aspartate aminotransferase increased
14%
Blood magnesium decreased
14%
Blood potassium increased
14%
Dysgeusia
14%
Hepatic encephalopathy
14%
Bronchitis
14%
Sinusitis
14%
Upper respiratory tract infection
14%
Eye infection
14%
Vulvovaginitis
14%
Arthralgia
14%
Decreased appetite
14%
Metabolic acidosis
14%
Pleural effusion
14%
Dysphonia
14%
Acute kidney injury
14%
Urinary incontinence
14%
Hyperhidrosis
14%
Pruritus
14%
Tachycardia
14%
Eyelid ptosis
100%
80%
60%
40%
20%
0%
Study treatment Arm
LAM-002A + Atezolizumab
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
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
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.
<i>Ganoderma lucidum</i> 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.
<i>Ganoderma lucidum</i> 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.
Find a Location
Who is running the clinical trial?
AI Therapeutics, Inc.Lead Sponsor
6 Previous Clinical Trials
266 Total Patients Enrolled
OrphAI TherapeuticsLead Sponsor
6 Previous Clinical Trials
266 Total Patients Enrolled
Suma Babu, M.D.Principal InvestigatorMassachusetts General Hospital
Katherine Nicholson, M.D.Principal InvestigatorMassachusetts General Hospital
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a brain disorder not caused by C9ORF72 gene changes.I do not have any unstable health conditions that could put me at risk.Your liver tests show that your liver may not be working properly.My kidneys are not working as well as they should.My previous health issue hasn't improved or enough time hasn't passed for a retest.I have ALS linked to a C9ORF72 gene mutation.I am not on edaravone or have finished at least one cycle and plan to continue.I am currently on medication to suppress my immune system.I am a male and unwilling to use contraception.You must have been diagnosed with ALS using specific guidelines called the revised El Escorial criteria.You have a mental health condition that is not well controlled or you have problems with substance abuse.My lung function is at least half of what is expected for someone my age and size.I am not taking medication that strongly affects liver enzymes.I do not have stomach or intestine problems that could affect how a drug works or is studied.You have used experimental treatments for ALS within a certain period of time.I have cancer or a history of it, with some exceptions.I have been on a stable dose of riluzole for over 30 days, or I am not taking it.I am 18 years old or older.I can safely swallow capsules.You have received experimental gene therapies for ALS.I have had a solid organ transplant.I am taking medication that is a strong influencer on the CYP2C9 enzyme.I cannot have a lumbar puncture due to health reasons.
Research Study Groups:
This trial has the following groups:- Group 1: Placebo
- Group 2: LAM-002A
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.