What side effects are associated with this type of intervention?

Common treatments for ALS, such as riluzole, edaravone, and PB-TURSO, have various side effects. Riluzole can cause nausea, weakness, and liver function abnormalities. Edaravone is associated with bruising, gait disturbances, and allergic reactions. PB-TURSO may lead to gastrointestinal issues and fatigue. Investigational treatments like masitinib can cause gastrointestinal symptoms and skin reactions, while reldesemtiv may lead to muscle cramps and fatigue. These side effects are generally mild to moderate but should be discussed with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved two main drugs for the treatment of Amyotrophic Lateral Sclerosis (ALS): riluzole and edaravone. 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 a free radical scavenger, reducing oxidative stress in neurons. These treatments aim to slow the progression of ALS and improve the quality of life for patients. While the mechanism of action for PTC857 is not specified, it is being studied for its potential efficacy and safety in ALS patients.

What other types of research exist?

Promising novel alternatives to PTC857 for ALS patients include the Sigma-1 receptor (S1R) agonist PRE-084, which has shown improvements in locomotor function and motor neuron survival in preclinical models. Additionally, antisense oligonucleotide therapies targeting huntingtin expression and RNA interference therapies are being explored for their potential in treating ALS.

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Placebo

PTC857 for ALS (CARDINALS Trial)

Phase 2

Waitlist Available

Research Sponsored by PTC Therapeutics

Eligibility Criteria Checklist

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

Must have

No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period

Standard-of-care therapy for the treatment of ALS (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol) should be stable and unchanged from 30 (-3) days prior to the start of the Screening Period and intend to remain stable and unchanged throughout the course of the study

Must not have

Participant has previously received PTC857

For female participants, any past medical history of breast cancer, regardless of remission status, or any first degree relative with history of breast cancer

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 24

Summary

This trial is testing a new drug called PTC857 to see if it can help people with ALS. Participants will either receive PTC857 or a non-active substance. If they complete the initial treatment, they can continue taking PTC857 for an extended period to further assess its effects.

Who is the study for?

This trial is for people with ALS who have had symptoms for less than 24 months and a score of at least 34 on the ALSFRS-R scale. They must not be pregnant, nursing, or planning pregnancy, have no significant respiratory issues (slow vital capacity ≥60%), and no recent use of certain ALS treatments together. Participants should not have breast cancer history or be in other studies.

What is being tested?

The study tests PTC857's effectiveness and safety against a placebo in treating ALS. Participants will randomly receive either PTC857 or an inactive substance to compare outcomes between the two groups.

What are the potential side effects?

While specific side effects are not listed here, participants may experience adverse reactions related to PTC857 treatment which will be monitored throughout the trial for safety assessment.

Eligibility Criteria

Inclusion Criteria

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

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My lung function is good enough to pass a basic breathing test.

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My ALS medication has been the same for the last month and won't change during the study.

Exclusion Criteria

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

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I have previously been treated with PTC857.

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I have had breast cancer or a close family member has.

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I have been treated with edaravone and sodium phenylbutyrate/taurursodiol within the last 30 days.

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I am not pregnant, nursing, or planning to become pregnant during the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 24

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

Treatment Details

Study Objectives

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

Primary study objectives

Participant Ranks Based on the Combined Assessment of ALS Functional Rating Scale-Revised (ALSFRS-R) and Survival After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population)

Secondary study objectives

Change from Baseline in ALSFRS-R Score at Week 24 (ITT1 Analysis Population)

Change from Baseline in ALSFRS-R Score at Week 24 (ITT2 Analysis Population)

Participant Ranks Based on the Combined Assessment of ALSFRS-R and Survival After 24 Weeks of Treatment (ITT 2 Analysis Population)

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: PTC857Experimental Treatment1 Intervention

Participants will receive PTC857 during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who enter the Long-term Extension (LTE) Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

Group II: PlaceboActive Control1 Intervention

Participants will receive matching placebo during the 24-Week Treatment Period. Following successful completion of the Treatment Period, participants who enter the LTE Period, will receive open-label PTC857 for 28 weeks. Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

0 / 6Eligibility 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 works by inhibiting glutamate release, which helps reduce excitotoxicity—a process that damages neurons due to excessive stimulation by neurotransmitters. Edaravone acts as an antioxidant, reducing oxidative stress and preventing cell damage. PB-TURSO combines two compounds that reduce neuronal cell death by targeting different cellular pathways involved in ALS progression. These treatments are crucial for ALS patients as they help slow the functional decline and potentially extend survival, offering a better quality of life despite the progressive nature of the disease.

Translating biological findings into new treatment strategies for amyotrophic lateral sclerosis (ALS).