What side effects are associated with this type of intervention?

Common treatments for ALS, such as edaravone, riluzole, and sodium phenylbutyrate-taurursodiol, have various side effects. Edaravone can cause bruising, gait disturbances, and allergic reactions. Riluzole is associated with nausea, liver enzyme elevation, and fatigue. Sodium phenylbutyrate-taurursodiol may lead to gastrointestinal issues, such as diarrhea and abdominal pain. Patients should discuss these potential side effects with their healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Amyotrophic Lateral Sclerosis (ALS). Riluzole, the first drug approved, works by inhibiting glutamate release and has been shown to extend survival. Edaravone, another approved drug, acts as a free radical scavenger to reduce oxidative stress in neurons. Additionally, there are investigational treatments like masitinib, which is used as an add-on therapy to riluzole, and various stem cell therapies that are being explored for their potential neuroprotective effects. These treatments aim to slow disease progression and improve quality of life for ALS patients.

What other types of research exist?

Promising novel alternatives to AP-101 for ALS patients include: 1) Electroacupuncture, which has shown potential in reducing neuroinflammatory responses in ALS models. 2) Tofersen, an antisense oligonucleotide targeting SOD1 mutations, currently in clinical trials. 3) AMX0035, a combination of sodium phenylbutyrate and taurursodiol, which has shown efficacy in slowing disease progression. 4) Reldesemtiv, a fast skeletal muscle troponin activator, aimed at improving muscle function. These alternatives offer different mechanisms of action and are in various stages of research and clinical trials.

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AP-101 for ALS

Phase 2

Waitlist Available

Research Sponsored by AL-S Pharma

Eligibility Criteria Checklist

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

Must have

Have possible, clinically probable, clinically probable-laboratory supported or definite familial or sporadic ALS in accordance with the El-Escorial criteria or who have a diagnosis of ALS as defined by the Gold Coast Criteria; progressive motor impairment documented by history or repeated clinical examination, preceded by normal motor development, and presence of upper and lower motor neuron dysfunction in at least 1 body region or lower motor neuron dysfunction in at least 2 body regions and investigations excluding other conditions

In familial ALS participants, a confirmed pathogenic superoxide dismutase 1 (SOD1) mutation

Must not have

Have undergone stem cell therapy

Are on nasal intermittent positive pressure ventilation (NIPPV) >4 hours per day for the treatment of ALS related symptoms

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from start of the study up to week 51

Summary

This trial is testing a new treatment called AP-101 to see if it is safe and well-tolerated. It focuses on people with familial and sporadic ALS, a condition that affects nerve cells and muscle control. Researchers want to understand how AP-101 moves through the body and its effects on ALS symptoms.

Who is the study for?

This trial is for adults with familial or sporadic ALS who meet specific clinical criteria, including normal lab results and a slow vital capacity of at least 50% predicted values. Participants must not be pregnant, nursing, or have other neuromuscular conditions. They should not have had certain treatments like stem cell therapy recently and must agree to use contraception.

What is being tested?

The study tests the safety and effects of AP-101 on people with ALS compared to a placebo. It looks at how the body processes the drug (pharmacokinetics) and its impact on disease markers (pharmacodynamics). Some participants may continue in an open-label extension based on investigator judgment.

What are the potential side effects?

While specific side effects are not listed here, generally this type of trial will monitor for any adverse reactions ranging from mild symptoms like headaches or nausea to more serious issues affecting organ function which could arise from taking AP-101.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with ALS based on specific criteria and show progressive motor impairment.

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I have a confirmed SOD1 mutation linked to my familial ALS.

Exclusion Criteria

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

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I have undergone stem cell therapy.

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I use a breathing machine for my ALS symptoms more than 4 hours daily.

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I do not have severe heart, blood, kidney, brain diseases, lung problems, or mental health issues.

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I have had a tracheostomy for my ALS symptoms.

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I have conditions that cause muscle weakness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from start of the study up to week 51

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from start of the study up to week 51

This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of the study up to week 51 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: AP-101Experimental Treatment1 Intervention

AP-101 is administered by IV.

Group II: PlaceboPlacebo Group1 Intervention

Placebo is administered by IV.

0 / 7Eligibility 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 Amyotrophic Lateral Sclerosis (ALS) include riluzole, edaravone, and sodium phenylbutyrate-taurursodiol. Riluzole works by inhibiting glutamate release, which helps reduce excitotoxicity—a process that damages neurons in ALS. Edaravone acts as an antioxidant, reducing oxidative stress and protecting motor neurons from damage. Sodium phenylbutyrate-taurursodiol combines two compounds that reduce neuronal cell death by targeting mitochondrial and endoplasmic reticulum stress pathways. These mechanisms are crucial for ALS patients as they aim to slow disease progression, preserve motor function, and improve quality of life.

Inflammation, immunity, and amyotrophic lateral sclerosis: II. immune-modulating therapies.Translating biological findings into new treatment strategies for amyotrophic lateral sclerosis (ALS).