What side effects are associated with this type of intervention?

Common treatments for Lou Gehrig's Disease (ALS) include riluzole, edaravone, sodium phenylbutyrate, and taurursodiol. Riluzole can cause side effects such as nausea, weakness, decreased lung function, and liver enzyme elevation. Edaravone may lead to bruising, gait disturbances, headaches, and allergic reactions. Sodium phenylbutyrate is associated with gastrointestinal issues like nausea and diarrhea, as well as fatigue. Taurursodiol can cause diarrhea, abdominal pain, and nausea. These side effects are generally manageable but should be monitored by healthcare providers.

What prior FDA approvals exist?

The FDA has approved several treatments for Lou Gehrig's Disease (ALS). Riluzole, the first drug approved, is believed to reduce damage to motor neurons by decreasing the release of glutamate. Edaravone, another approved treatment, acts as a free radical scavenger to reduce oxidative stress. More recently, the combination of sodium phenylbutyrate and taurursodiol has been approved, which aims to protect nerve cells from damage by targeting cellular stress pathways. These treatments are designed to slow disease progression and improve quality of life for ALS patients.

What other types of research exist?

Promising novel alternatives for ALS treatment in 2024 include: 1) Riluzole, which extends survival by reducing glutamate toxicity. 2) Edaravone, which slows functional decline by reducing oxidative stress. 3) Sodium phenylbutyrate and taurursodiol, which have shown efficacy in combination to slow disease progression. These treatments are either approved or widely accepted for ALS management and can be considered in consultation with a healthcare provider.

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Other

PrimeC for ALS (PARADIGM Trial)

Phase 2

Waitlist Available

Research Sponsored by NeuroSense Therapeutics Ltd.

Eligibility Criteria Checklist

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

Must have

Item 3 (swallowing) in ALSFRS-R ≥ 3

Upright slow vital capacity (SVC) ≥ 60% of predicted for age, height, weight and sex at screening according to the GLI-2012

Must not have

Impairment of renal function (creatinine ≥ 1.5)

Female who is pregnant or breastfeeding or with intention of becoming pregnant

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Summary

This trial involves ALS patients who will receive a new drug. The goal is to see if the new drug is safe, well-tolerated, and effective in improving their condition. Participants' health will be monitored regularly.

Who is the study for?

This trial is for adults aged 18-75 with ALS, who can understand and agree to the study terms. They must have an ALSFRS-R score ≥25, stable swallowing ability, a BMI between 18-30, disease duration less than 30 months from first symptom, and meet specific breathing capacity criteria. Women must meet reproductive standards. Exclusions include severe diabetes, heart issues, renal impairment, cognitive problems or other significant illnesses.

What is being tested?

The study tests PrimeC against a placebo in people with ALS. Participants are randomly assigned in a 2:1 ratio to either receive PrimeC or placebo twice daily for six months while continuing standard treatments like riluzole and edaravone. After six months of treatment, all participants will receive PrimeC during a year-long open label extension phase.

What are the potential side effects?

While the side effects of PrimeC are not detailed here specifically since it's under investigation; generally such trials monitor for adverse reactions including digestive issues, allergic responses to medication components if known allergies exist and potential interactions with existing medications.

Eligibility Criteria

Inclusion Criteria

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

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I can swallow without significant difficulty.

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My lung function test shows I'm at least 60% of the expected value for my age, height, weight, and sex.

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I have been diagnosed with ALS.

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I meet the specific reproductive health requirements.

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I am between 18 and 75 years old.

Exclusion Criteria

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

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My kidney function is impaired with high creatinine levels.

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I am currently pregnant, breastfeeding, or planning to become pregnant.

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I use a tracheostomy or feeding tube.

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I am a poor metabolizer for certain medications due to my CYP2C9 status.

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I have difficulty with memory or thinking clearly.

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I have a serious stomach issue, like an ulcer, tumor, or I've had weight loss surgery.

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I have a serious heart, lung, muscle, or mental health condition.

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I have heart problems like heart failure, heart attack, high blood pressure, or irregular heartbeat.

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My blood pressure and heart function meet the study's requirements.

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My diabetes is hard to control or fluctuates a lot.

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I have a history of heart rhythm issues or epilepsy.

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I cannot take certain medications due to adverse reactions.

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I understand what participating in this study involves.

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I regularly use aspirin or NSAIDs.

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I use daily medication for my COPD or asthma.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Incidence and severity of treatment-emergent adverse events (TEAEs)

Number of patients who discontinued treatment prematurely due to adverse events

Number of subjects who discontinued treatment prematurely

Secondary study objectives

Composite survival at 6 months of treatment

Survival at 6 months of treatment

Trial Design

2Treatment groups

Active Control

Placebo Group

Group I: PrimeCActive Control1 Intervention

2 tablets of PrimeC administered twice daily (4 tablets a day), at a daily dose of 1496 mg

Group II: PlaceboPlacebo Group1 Intervention

2 tablets of Placebo administered twice daily (4 tablets a day). Placebo tablets are matched in size, color and taste.

0 / 20Eligibility 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

Riluzole works by inhibiting glutamate release, which helps to reduce excitotoxicity, a process that damages nerve cells in ALS. Edaravone is an antioxidant that helps to reduce oxidative stress, another factor contributing to nerve cell damage. Sodium phenylbutyrate and taurursodiol are believed to work by reducing endoplasmic reticulum stress and mitochondrial dysfunction, respectively, both of which are implicated in ALS pathology. These mechanisms are crucial for ALS patients as they target different pathways involved in nerve cell degeneration, potentially slowing disease progression and improving quality of life.

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