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 headaches. Riluzole is associated with nausea, liver function abnormalities, and dizziness. Sodium phenylbutyrate-taurursodiol may lead to gastrointestinal issues like diarrhea and abdominal pain. These treatments aim to slow disease progression but come with potential adverse effects that should be monitored by healthcare providers.

What other types of research exist?

Promising novel alternatives to SAR443820 for ALS patients in 2024 include: 1. Tofersen: An antisense oligonucleotide targeting SOD1 mutations, showing promise in clinical trials. 2. AMX0035 (Relyvrio/Albrioza): A combination of sodium phenylbutyrate and taurursodiol, approved for ALS treatment in the USA and Canada. 3. NurOwn: A stem cell therapy that has shown potential in improving ALS symptoms in clinical trials. 4. TUDCA (Tauroursodeoxycholic acid): An investigational drug with neuroprotective properties, currently in clinical trials for ALS.

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SAR443820 for ALS (HIMALAYA Trial)

Q: What prior FDA approvals exist?

The FDA has approved several drugs for the treatment of Amyotrophic Lateral Sclerosis (ALS). Riluzole, approved in 1995, is a glutamate pathway antagonist that has been shown to extend survival. Edaravone, approved in 2017, is an antioxidant that may slow the decline of physical function. More recently, the combination of sodium phenylbutyrate and taurursodiol (marketed as Relyvrio in the USA and Albrioza in Canada) has been approved for its potential neuroprotective effects. These treatments aim to slow disease progression and improve quality of life, similar to the investigational drug SAR443820, which is being studied for its efficacy and safety in ALS patients.

Phase 2

Waitlist Available

Research Sponsored by Sanofi

Eligibility Criteria Checklist

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

Must have

Either not currently receiving the combination of sodium phenylbutyrate and taurursodiol or on the approved standard schedule of the combination of sodium phenylbutyrate and taurursodiol treatment for at least 4 weeks before the screening visit. Participants receiving the combination of sodium phenylbutyrate and taurursodiol are expected to remain on the approved standard schedule throughout the duration of the study

Participants with a body weight no less than 45 kg and body mass index no less than 18 kg/m2 at the screening visit

Must not have

Participants who have received stem cell or gene therapy for ALS at any time in the past

History of recent serious infection (eg, pneumonia, septicemia) within 4 weeks of the screening visit; infection requiring hospitalization or treatment with IV antibiotics, antivirals, or antifungals within 4 weeks of screening; or chronic bacterial infection (such as tuberculosis) deemed unacceptable as per the Investigator's judgment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to week 24

Summary

This trial tests SAR443820, a pill taken regularly, in adults aged 18-80 with ALS. It aims to see if the new medication helps ALS patients and is safe over time.

Who is the study for?

Adults aged 18-80 with ALS, able to swallow tablets, and not severely ill from other conditions can join. They must have had symptoms for less than 2 years and a breathing capacity over 60% of expected. If taking riluzole, edaravone or sodium phenylbutyrate/taurursodiol (Relyvrio/Albrioza), doses must be stable for at least 4 weeks.

What is being tested?

The trial is testing SAR443820's effectiveness in slowing down ALS progression compared to a placebo. Participants are randomly assigned to either the drug or placebo group and will switch to open-label SAR443820 after the initial double-blind phase.

What are the potential side effects?

Specific side effects of SAR443820 aren't listed but may include typical drug reactions like nausea, headaches, allergic responses or potential interactions with existing medications.

Eligibility Criteria

Inclusion Criteria

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

Select...

I am not on sodium phenylbutyrate and taurursodiol, or have been on it for 4 weeks.

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I weigh at least 45 kg and my BMI is at least 18.

Select...

I have been diagnosed with ALS.

Select...

I can swallow pills.

Select...

My first ALS symptoms appeared less than 2 years ago.

Exclusion Criteria

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

Select...

I have had stem cell or gene therapy for ALS.

Select...

I haven't had a serious infection or been hospitalized for one in the last month.

Select...

I don't have severe health issues other than ALS that would make it unsafe for me to join this study.

Select...

I have not received a live vaccine in the last 14 days.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to week 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 24 for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Muscle Strength - Part A

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: SAR443820Experimental Treatment1 Intervention

twice daily (BID) oral SAR443820

Group II: PlaceboPlacebo Group1 Intervention

twice daily (BID) oral placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SAR443820

2022

Completed Phase 1

~120

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

The most common treatments for Amyotrophic Lateral Sclerosis (ALS) include riluzole, edaravone, and sodium phenylbutyrate-taurursodiol (PB-TURSO). Riluzole inhibits glutamate release, reducing excitotoxicity that damages neurons. Edaravone acts as a free radical scavenger, mitigating oxidative stress implicated in neuronal damage. PB-TURSO combines sodium phenylbutyrate, which reduces endoplasmic reticulum stress, and taurursodiol, which stabilizes mitochondrial function. These treatments are important for ALS patients as they target different pathways involved in neuronal degeneration, potentially slowing disease progression and improving quality of life.