Multiple System AtrophyPhase 2NCT05109091
Study of ATH434 in Participants with Multiple System Atrophy
A Phase 2 study testing ATH434 dose level 1 and ATH434 dose level 2 in people with Multiple System Atrophy, run by Alterity Therapeutics at 23 sites in 6 countries.
Trial summary
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy Read more about this study
- Who is it for?
- Ages 30 to 75 years
- Conditions the study is for
- Multiple System Atrophy
- What is being tested?
- ATH434 dose level 1 and ATH434 dose level 2
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 23 sitesin 6 countries
- Study dates
- 1 Jul 2022to 28 Nov 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant has clinical features of parkinsonism.
- Participant has evidence of orthostatic hypotension and/or bladder dysfunction.
- Participant has ataxia and/or pyramidal signs on neurological examination.
- Participant is ambulatory.
- Participant has biomarker evidence of MSA in biologic fluid and on MRI.
Who cannot join
- Participant has motor symptoms for > 4 years.
- Participant has advanced disease, as indicated by frequent falls or choking.
- Participant has structural brain abnormality on MRI.
- Participant has any significant neurological disorder other than MSA.
- Participant has an unstable medical or psychiatric illness.
- Participant has a contraindication to, or is unable to tolerate, MRI or lumbar puncture.
Trial locations and contacts
23 sites in 6 countries.
23 sites in 6 countries
United States6 sites
Johns Hopkins University
Baltimore, Maryland, 21287
Rush University Medical Center
Chicago, Illinois, 60612
University of California San Diego
La Jolla, California, 92093
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Columbia University Irving Medical Center
New York, New York, 10032
The Movement Disorder Clinic of Oklahoma
Tulsa, Oklahoma, 74136
France4 sites
CHU de Bordeaux/Groupe Hospitalier Pellegrin
Bordeaux, 33076
CHU de Marseille/Hôpital de la Timone
Marseille, 13005
CHU/HU Pitié Salpêtrière
Paris, Île-de-France Region, 75013
CHU de Toulouse/Hôpital Pierre-Paul Riquet
Toulouse, Haute-Garonne, 31059
Italy4 sites
IRCCS Istituto Delle Scienze Neurologiche di Bologna
Bologna, Bologna
Policlinico di Milano
Milan
Azienda Ospedaliero-Universitaria Pisana
Pisa
AOU San Giovanni di Dio Ruggi d'Aragona
Salerno
United Kingdom4 sites
Queen Elizabeth University Hospital
Glasgow, Scotland
University College London
London, England
Newcastle University
Newcastle upon Tyne, England
Salford Royal Hospital
Salford, England
Australia3 sites
St Vincent's Hospital (Sydney)
Darlinghurst, New South Wales
The Alfred Hospital
Melbourne, Victoria
Westmead Hospital
Westmead, New South Wales
New Zealand2 sites
New Zealand Brain Research Institute
Christchurch
Auckland City Hospital
Grafton
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in Multiple System Atrophy
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy
- Conditions
- Multiple System Atrophy
- Being tested
- ATH434 dose level 1, ATH434 dose level 2, Placebo
- Keywords
- Multiple System Atrophy, ATH434, Neurodegenerative disease, Shy-Drager Syndrome, Movement disorders, Autonomic dysfunction, Synucleinopathies, Atypical parkinsonism
- Medical subject headings
- Multiple System Atrophy, Neurodegenerative Diseases, Shy-Drager Syndrome, Movement Disorders, Primary Dysautonomias, Synucleinopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATH434 dose level 1Drug
Also called PBT434
ATH434 taken BID
ATH434 dose level 2Drug
Also called PBT434
ATH434 taken BID
PlaceboDrug
Placebo taken BID
| Study group | Role | What they receive |
|---|---|---|
| ATH434 Arm 1 | Experimental | Not described Receives: Drug: ATH434 dose level 1 |
| ATH434 Arm 2 | Experimental | Not described Receives: Drug: ATH434 dose level 2 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in iron content as measured by brain MRI
Measured: Change from Baseline to Week 52
Secondary outcomes (3)
Change in Neurofilament Light Chain Levels
Measured: Change from Baseline to Week 52
Change in Unified MSA Rating Scale (UMSARS) Score
Measured: Change from Baseline to Week 52
Change in SF-36 Score
Measured: Change from Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2021
- Study start1 Jul 2022
- Primary completion28 Nov 2024
- Study completion28 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple System AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alterity Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05109091
- Sponsor's protocol ID
- ATH434-201
- Status last verified
- Dec 2024
- First submitted
- 22 Oct 2021
- Last updated
- 13 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in Multiple System Atrophy
- ClinicalTrials.gov ID
- NCT05109091
- Acronym / sponsor ID
- ATH434-201
- Conditions
- Multiple System Atrophy
- Keywords
- Multiple System Atrophy, ATH434, Neurodegenerative disease, Shy-Drager Syndrome, Movement disorders, Autonomic dysfunction, Synucleinopathies, Atypical parkinsonism
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Alterity TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2022 (actual)
- Primary completion
- 28 Nov 2024 (actual)
- Study completion
- 28 Nov 2024 (actual)
- First posted
- 5 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will assess the safety and efficacy of ATH434 in participants with Multiple System Atrophy
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ATH434 Arm 1 | Experimental | Drug: ATH434 dose level 1 |
| ATH434 Arm 2 | Experimental | Drug: ATH434 dose level 2 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 30 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Participant has clinical features of parkinsonism.
- Participant has evidence of orthostatic hypotension and/or bladder dysfunction.
- Participant has ataxia and/or pyramidal signs on neurological examination.
- Participant is ambulatory.
- Participant has biomarker evidence of MSA in biologic fluid and on MRI.
Key exclusion criteria (6 listed)
- Participant has motor symptoms for > 4 years.
- Participant has advanced disease, as indicated by frequent falls or choking.
- Participant has structural brain abnormality on MRI.
- Participant has any significant neurological disorder other than MSA.
- Participant has an unstable medical or psychiatric illness.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATH434 dose level 1Also: PBT434ATH434 taken BID | Drug | ATH434 Arm 1 |
| ATH434 dose level 2Also: PBT434ATH434 taken BID | Drug | ATH434 Arm 2 |
| PlaceboPlacebo taken BID | Drug | Placebo |
Primary outcome
Change in iron content as measured by brain MRI
Time frame: Change from Baseline to Week 52
3 secondary outcomes
Change in Neurofilament Light Chain Levels
Time frame: Change from Baseline to Week 52
Change in Unified MSA Rating Scale (UMSARS) Score
Time frame: Change from Baseline to Week 52
Change in SF-36 Score
Time frame: Change from Baseline to Week 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Dec 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ATH434 dose level 1 and ATH434 dose level 2 in people with Multiple System Atrophy, run by Alterity Therapeutics at 23 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, France, Italy, United Kingdom, Australia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in iron content as measured by brain MRI (time frame: Change from Baseline to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Dec 2024.