ATH434 for Multiple System Atrophy

Overseen By Alterity Clinical Trials

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Alterity Therapeutics

Disqualifiers: Neurological disorder, Unstable illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ATH434 to determine its safety and effectiveness for people with Multiple System Atrophy (MSA), a condition affecting movement, balance, and automatic body functions like blood pressure and bladder control. The trial focuses on individuals experiencing symptoms such as dizziness when standing (orthostatic hypotension), bladder issues, and coordination problems (ataxia). Participants must also have specific test results indicating MSA. The study aims to explore whether ATH434 can better manage these symptoms. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that ATH434 is likely to be safe for humans?

Research has shown that ATH434 is generally safe for people with Multiple System Atrophy (MSA). Studies have found it does not cause serious side effects. The number of unwanted side effects is similar to those in placebo groups, indicating no major safety issues. This suggests ATH434 is safe for humans based on current evidence. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for Multiple System Atrophy (MSA) focus on managing symptoms, like using medications for movement issues or blood pressure problems. But ATH434 works differently, targeting the underlying cause of brain cell damage. Researchers are excited about ATH434 because it specifically reduces the buildup of a protein called alpha-synuclein, which is thought to contribute to the progression of MSA. By addressing this root cause, ATH434 has the potential to slow down disease progression rather than just alleviate symptoms.

What evidence suggests that ATH434 might be an effective treatment for Multiple System Atrophy?

Research has shown that ATH434, the investigational treatment in this trial, may help treat Multiple System Atrophy (MSA). Recent studies found that ATH434 slowed the shrinking of brain areas affected by MSA. Patients taking ATH434 demonstrated noticeable improvement in their symptoms compared to those on a placebo. The treatment positively affected key indicators of the disease's presence or severity. These findings suggest that ATH434 could be a helpful option for people with MSA. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for individuals with Multiple System Atrophy (MSA), showing symptoms like parkinsonism, low blood pressure upon standing, bladder issues, ataxia or abnormal muscle coordination, and certain signs on a neurological exam. They must have MSA biomarkers in their body fluids and MRI scans. People can't join if they can't swallow pills, attend visits, have brain structure issues on MRI, unstable health conditions or other significant neurological disorders.

Inclusion Criteria

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

I show symptoms similar to Parkinson's disease.

I have coordination issues or muscle weakness.

Exclusion Criteria

You have a brain abnormality shown on an MRI.

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

I cannot swallow pills.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ATH434 to assess safety and efficacy

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ATH434

Trial Overview

The study is testing the safety and effectiveness of a drug called ATH434 in people who are clinically diagnosed with Multiple System Atrophy. It aims to see how well this drug works and what effects it has on patients' bodies.

How Is the Trial Designed?

Group I: ATH434 Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Alterity Therapeutics

Lead Sponsor

Trials

3

Recruited

100+

Citations

1.

alteritytherapeutics.com

alteritytherapeutics.com/investor-centre/news/2025/07/28/alterity-therapeutics-reports-positive-topline-data-from-open-label-phase-2-clinical-trial-of-ath434-in-multiple-system-atrophy/

Alterity Therapeutics Reports Positive Topline Data from ...

Neuroimaging outcomes indicate that ATH434 slowed brain atrophy in MSA affected areas, as measured by the MSA Atrophy Index (MSA-AI)4, when ...

2.

alteritytherapeutics.com

alteritytherapeutics.com/investor-centre/news/2025/10/09/alterity-therapeutics-presents-positive-data-from-ath434-phase-2-trial-at-the-2025-international-congress-of-parkinsons-disease-and-movement-disorders/

Alterity Therapeutics Presents Positive Data from ATH434 ...

The data showed that, compared to placebo, ATH434 produced clinically and statistically significant improvement on the modified Unified Multiple ...

3.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000212526

Topline Data from a Randomized, Double Blind, Placebo ...

Objective: Evaluate the efficacy, biomarker response and safety of ATH434 treatment in early Multiple system atrophy (MSA). Background: MSA ...

4.

neurologylive.com

neurologylive.com/view/ath434-continues-demonstrate-positive-efficacy-latest-phase-2-data-readout

ATH434 Continues to Demonstrate Positive Efficacy in ...

New trial data reveals ATH434 significantly improves symptoms and biomarkers in multiple system atrophy patients, paving the way for potential ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05109091

Study of ATH434 in Participants with Multiple System Atrophy

This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy

6.

neurologylive.com

neurologylive.com/view/multiple-system-atrophy-phase-2-findings-reinforce-ath434-disease-modifying-potential

Phase 2 Findings Reinforce ATH434's Disease-Modifying ...

In terms of safety, ATH434 was well tolerated with no safety signals. We saw similar adverse event rates vs placebo and no serious or severe AEs ...

7.

alteritytherapeutics.com

alteritytherapeutics.com/investor-centre/news/2025/09/29/alterity-therapeutics-reports-new-commercial-assessment-for-ath434-in-multiple-system-atrophy/

Alterity Therapeutics Reports New Commercial ...

ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain ...