What side effects are associated with this type of intervention?

Common treatments for Multiple System Atrophy (MSA) include medications to manage parkinsonism, such as levodopa, which can cause side effects like nausea, dizziness, and dyskinesia. Autonomic dysfunction is often treated with medications like fludrocortisone and midodrine, which can lead to hypertension, headaches, and urinary retention. Additionally, alpha-synuclein aggregation inhibitors like ATH434 are being studied, but specific side effects for these are not well-documented yet.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically targeting alpha-synuclein aggregation for Multiple System Atrophy (MSA). The management of MSA primarily focuses on symptomatic treatment, including the use of medications like levodopa for motor symptoms, although the response is often poor or unsustained. Other treatments address non-motor symptoms such as orthostatic hypotension, depression, and dystonia. Research and clinical trials, such as those investigating ATH434, are ongoing to find more effective disease-modifying therapies for MSA.

What other types of research exist?

Promising novel alternatives to ATH434 for Multiple System Atrophy patients include antisense oligonucleotide therapies, which have shown potential in neurodegenerative diseases like ALS, and monoclonal antibodies targeting alpha-synuclein. Additionally, multitarget therapies that address various pathological mechanisms in MSA are being explored and may offer new treatment avenues.

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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 ataxia and/or pyramidal signs on neurological examination.

Participant has clinical features of parkinsonism.

Must not have

Participant has advanced disease, as indicated by frequent falls or choking.

Participant has an unstable medical or psychiatric illness.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial will test a new drug called ATH434 to see if it is safe and can help people with Multiple System Atrophy by possibly slowing down the disease or reducing symptoms.

Who is the study for?

This trial is for individuals with Multiple System Atrophy (MSA) who can walk and show specific signs of ataxia, parkinsonism, orthostatic hypotension, or bladder issues. They must have MSA biomarkers in their fluids and MRI evidence. Those with motor symptoms over 4 years, other significant neurological disorders, unstable illnesses, MRI or lumbar puncture contraindications, advanced disease with falls/choking, or brain abnormalities on MRI cannot join.

What is being tested?

The study tests the safety and effectiveness of a drug called ATH434 in two different doses compared to a placebo in people with MSA. Participants will be randomly assigned to receive either the drug at one of two dose levels or a placebo to see if there's an improvement in their condition.

What are the potential side effects?

While the specific side effects of ATH434 are not listed here, common types of side effects from drugs like this may include gastrointestinal discomforts such as nausea or diarrhea; potential allergic reactions; fatigue; dizziness due to blood pressure changes; and possibly headaches.

Eligibility Criteria

Inclusion Criteria

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

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I have coordination issues or stiffness and weakness in my limbs.

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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 signs of MSA in my body fluids and MRI.

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I can walk on my own.

Exclusion Criteria

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

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I often fall or choke due to my advanced disease.

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

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I cannot undergo MRI or lumbar puncture due to health reasons.

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I have had motor symptoms for more than 4 years.

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

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: ATH434 Arm 2Experimental Treatment1 Intervention

Group II: ATH434 Arm 1Experimental Treatment1 Intervention

Group III: PlaceboPlacebo Group1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ATH434 dose level 2

2022

Completed Phase 2

~80

ATH434 dose level 1

2022

Completed Phase 2

~80

0 / 10Eligibility 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

Multiple System Atrophy (MSA) treatments often target the underlying pathophysiological mechanisms, such as alpha-synuclein aggregation. Alpha-synuclein aggregation inhibitors, like ATH434, work by preventing the clumping of alpha-synuclein proteins, which is believed to contribute to the neurodegenerative processes in MSA. This is crucial for MSA patients because reducing alpha-synuclein aggregation can potentially slow disease progression, alleviate symptoms, and improve quality of life. Other treatments may include dopaminergic medications to manage motor symptoms and autonomic agents to address blood pressure and urinary issues, but targeting alpha-synuclein directly addresses a core aspect of the disease pathology.

Investigational drugs in Alzheimer's disease: current progress.Parkinson risk in idiopathic REM sleep behavior disorder: preparing for neuroprotective trials.