Clinical trial NCT05109091: Study of ATH434 in Participants with Multiple System Atrophy
Official title: A Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in Multiple System Atrophy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Multiple System Atrophy
Keywords: Multiple System Atrophy
ATH434
Neurodegenerative disease
Shy-Drager Syndrome
Movement disorders
Autonomic dysfunction
Synucleinopathies
Atypical parkinsonism
Lead sponsor: Alterity Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-11-05
Enrollment: 77
Sex: ALL
Minimum age (years): 30
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: Inclusion Criteria:

1. Participant has clinical features of parkinsonism.
2. Participant has evidence of orthostatic hypotension and/or bladder dysfunction.
3. Participant has ataxia and/or pyramidal signs on neurological examination.
4. Participant is ambulatory.
5. Participant has biomarker evidence of MSA in biologic fluid and on MRI.

Exclusion Criteria:

1. Participant has motor symptoms for \> 4 years.
2. Participant has advanced disease, as indicated by frequent falls or choking.
3. Participant has structural brain abnormality on MRI.
4. Participant has any significant neurological disorder other than MSA.
5. Participant has an unstable medical or psychiatric illness.
6. Participant has a contraindication to, or is unable to tolerate, MRI or lumbar puncture.

Primary outcomes:
measure: Change in iron content as measured by brain MRI
timeFrame: Change from Baseline to Week 52
Secondary outcomes:
measure: Change in Neurofilament Light Chain Levels
timeFrame: Change from Baseline to Week 52
measure: Change in Unified MSA Rating Scale (UMSARS) Score
timeFrame: Change from Baseline to Week 52
measure: Change in SF-36 Score
timeFrame: Change from Baseline to Week 52
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: New York
country: United States
facility: Columbia University Irving Medical Center
state: New York
city: Tulsa
country: United States
facility: The Movement Disorder Clinic of Oklahoma
state: Oklahoma
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Darlinghurst
country: Australia
facility: St Vincent's Hospital (Sydney)
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Toulouse
country: France
facility: CHU de Toulouse/Hôpital Pierre-Paul Riquet
state: Haute-Garonne
city: Paris
country: France
facility: CHU/HU Pitié Salpêtrière
state: Ile-de-France
city: Bordeaux
country: France
facility: CHU de Bordeaux/Groupe Hospitalier Pellegrin
city: Marseille
country: France
facility: CHU de Marseille/Hôpital de la Timone
city: Bologna
country: Italy
facility: IRCCS Istituto Delle Scienze Neurologiche di Bologna
city: Milan
country: Italy
facility: Policlinico di Milano
city: Pisa
country: Italy
facility: Azienda Ospedaliero-Universitaria Pisana
city: Salerno
country: Italy
facility: AOU San Giovanni di Dio Ruggi d'Aragona
city: Christchurch
country: New Zealand
facility: New Zealand Brain Research Institute
city: Grafton
country: New Zealand
facility: Auckland City Hospital
city: London
country: United Kingdom
facility: University College London
state: England
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle University
state: England
city: Salford
country: United Kingdom
facility: Salford Royal Hospital
state: England
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
state: Scotland
Study officials:
References: