What side effects are associated with this type of intervention?

Common treatments for Multiple System Atrophy (MSA) include medications to manage symptoms such as parkinsonism, autonomic dysfunction, and other motor symptoms. Levodopa is often used to alleviate parkinsonian features, but it can cause side effects like nausea, dizziness, and dyskinesia. VMAT2 inhibitors like tetrabenazine, deutetrabenazine, and valbenazine, used for movement disorders, can cause sedation, akathisia, parkinsonism, and depressed mood. Additionally, neuroleptics may be used but can lead to side effects such as weight gain, metabolic syndrome, and tardive dyskinesia. Treatments targeting alpha-synuclein aggregation, like ATH434, are still under investigation, and their side effect profiles are not fully established.

What prior FDA approvals exist?

There are currently no FDA-approved treatments specifically targeting alpha-synuclein aggregation for Multiple System Atrophy (MSA). The management of MSA is primarily symptomatic, focusing on alleviating motor and non-motor symptoms. Levodopa is sometimes used, although its efficacy is often limited. Other treatments may include medications for managing orthostatic hypotension, dystonia, and depression. Research, such as the study on ATH434, is ongoing to find more targeted therapies for MSA.

What other types of research exist?

Promising novel alternatives to ATH434 for Multiple System Atrophy patients include compounds targeting alpha-synuclein aggregation, neuroinflammation, and neuroprotection. Examples include anle138b (an alpha-synuclein aggregation inhibitor), immunotherapies targeting alpha-synuclein, and neuroprotective agents like inosine. These alternatives are in various stages of clinical development and may offer new therapeutic options for MSA patients in 2024.

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Small Molecule

ATH434 for Multiple System Atrophy

Phase 2

Waitlist Available

Research Sponsored by Alterity Therapeutics

Eligibility Criteria Checklist

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

Must have

Participant has clinical features of parkinsonism.

Participant has evidence of orthostatic hypotension and/or bladder dysfunction.

Must not have

Participant has an unstable medical or psychiatric illness.

Participant has any significant neurological disorder other than MSA.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up change from baseline to week 52

Awards & highlights

No Placebo-Only Group

Summary

This trial will test a new drug called ATH434 to see if it is safe and effective for people with Multiple System Atrophy (MSA), a rare neurological disorder.

Who is the study for?

This trial is for individuals with Multiple System Atrophy (MSA) who show symptoms like parkinsonism, low blood pressure upon standing, bladder issues, and coordination or movement problems. They must have MSA indicators in their biological fluids and MRI scans. People can't join if they can't swallow pills, attend visits, have other major neurological disorders besides MSA, unstable health conditions, or cannot undergo MRI or lumbar puncture.

What is being tested?

The study is testing the safety and effectiveness of a drug called ATH434 in people diagnosed with Multiple System Atrophy. It aims to see how well this drug works on the condition's symptoms and progression.

What are the potential side effects?

While specific side effects of ATH434 are not provided here, common types of side effects from drugs treating neurological conditions may include nausea, dizziness, headache, fatigue or sleep disturbances.

Eligibility Criteria

Inclusion Criteria

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

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I show symptoms similar to Parkinson's disease.

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I experience dizziness when standing up or have bladder control issues.

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My tests show MSA markers in my body fluids and on my MRI.

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I have coordination problems or muscle weakness.

Exclusion Criteria

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

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I do not have any unstable medical or mental health conditions.

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I have a significant neurological condition, but it's not MSA.

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I cannot swallow pills.

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I cannot attend the study visits or complete its procedures.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ change from baseline to week 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~change from baseline to week 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and change from baseline to week 52 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Change in iron content as measured by brain MRI

Secondary study objectives

Change in Aggregating alpha-Synuclein Levels

Change in Neurofilament Light Chain Levels

Change in SF-36 Score

+1 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: ATH434Experimental Treatment1 Intervention

0 / 8Eligibility 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 Multiple System Atrophy (MSA) often target the symptoms rather than the underlying disease. ATH434, currently under study, works by inhibiting the aggregation of alpha-synuclein, a protein that forms toxic clumps in the brains of MSA patients. This mechanism is crucial because alpha-synuclein aggregation is believed to play a significant role in the neurodegenerative process of MSA. By preventing these toxic clumps, ATH434 aims to slow disease progression and improve neurological function. Other treatments may include dopaminergic medications to manage motor symptoms and autonomic agents to address blood pressure and urinary issues, but they do not modify the disease process itself. Understanding these mechanisms helps in developing targeted therapies that can potentially alter the course of MSA, offering hope for more effective management of the disease.

Integrin CD11b mediates α-synuclein-induced activation of NADPH oxidase through a Rho-dependent pathway.UCM707, an inhibitor of the anandamide uptake, behaves as a symptom control agent in models of Huntington's disease and multiple sclerosis, but fails to delay/arrest the progression of different motor-related disorders.Reversal of parkinsonian symptoms by intrastriatal and systemic manipulations of excitatory amino acid and dopamine transmission in the bilateral 6-OHDA lesioned marmoset.