The main goal of this trial is to evaluate the efficacy and safety of amlenetug for the treatment of participants with Multiple System Atrophy (MSA).
Eligibility
Sex
ALL
Min age
40 Years
Max age
75 Years
Healthy volunteers
No
Key Inclusion Criteria:
* The participant has a diagnosis of clinically established multiple system atrophy parkinsonian type (MSA-P) or multiple system atrophy cerebellar type (MSA-C), or clinically probable MSA-P or MSA-C, according to the 2022 Movement Disorders Society (MDS) criteria for the diagnosis of MSA at the Screening Visit.
* The participant had onset of motor MSA symptoms (that is, parkinsonian and/or cerebellar) within 5 years prior to the Screening Visit in the judgement of the investigator.
* The participant has an anticipated survival of \>3 years, in the opinion of the investigator, at the Screening Visit.
* The participant has suitable peripheral venous access for investigational medicinal product (IMP) administration and blood sampling.
* The participant has an UMSARS Part I score ≤16 (omitting item 11 on sexual function) at the Screening Visit.
Exclusion Criteria:
* The participant has previously been dosed with amlenetug.
* The participant has taken any active IMP within 3 months or 5 half lives of that product, whichever is longer, prior to the first dose of IMP.
* The participant has 2 or more first degree relatives with a history of MSA.
* The participant, if of MSA-P subtype, has unexplained anosmia (not explained by other common causes such as allergic rhinitis or smoking, nasal structural lesions, or nasal surgery) on olfactory testing at the Screening Visit.
* The participant has evidence (clinically or on magnetic resonance imaging (MRI)) and/or history of any clinically significant disease or condition other than MSA, that is, in the investigator's opinion, likely to affect CNS functioning, e.g., serious neurological disorder, other intracranial or systemic disease.
* The participant has a current diagnosis of movement disorders that could mimic MSA, e.g., Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, pharmacological, or post-encephalitic parkinsonism, per investigator discretion. Participants who have previously been incorrectly diagnosed with Parkinson's disease will not be excluded.
Other protocol-defined inclusion and exclusion criteria apply.
Primary outcome measure(s)
Rest of the World (RoW; All Countries Except European Union [EU] and Japan [JP]) Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression — Baseline up to Week 72 Mortality-adjusted clinical progression will be assessed by the composite endpoint modified Unified Multiple System Atrophy Rating Scale (mUMSARS) score and time-to-death (any cause).
EU and JP Regional-specific Outcome Measure: Mortality-adjusted Clinical Progression — Baseline up to Week 72 Mortality-adjusted clinical progression will be assessed by the composite endpoint Unified Multiple System Atrophy Rating Scale (UMSARS) Total Score (TS) and time-to-death (any cause).
Trial sites (74)
Facility
City
Region
Status
Mayo Clinic
Scottsdale
Arizona
University of Arkansas for Medical Sciences
Little Rock
Arkansas
University of California, San Francisco Neurosciences Clinical Research Unit
San Francisco
California
CenExel Rocky Mountain Clinical Research, LLC
Englewood
Colorado
Yale New Haven Health
North Haven
Connecticut
Parkinson's Disease And Movement Disorder Center Of Boca Raton
Boca Raton
Florida
University of Florida Norman Fixel Institute for Neurological Diseases
Gainesville
Florida
Indiana Health University
Indianapolis
Indiana
Johns Hopkins University School of Medicine
Baltimore
Maryland
Brigham and Women's Hospital
Boston
Massachusetts
Beth Israel Deaconess Medical Center
Boston
Massachusetts
QUEST Research Institute
Farmington Hills
Michigan
Mayo Clinic
Rochester
Minnesota
University Nebraska Medical Center
Omaha
Nebraska
NYU Medical Center - Dysautonomia center
New York
New York
Columbia University Medical Center - The Neurological Institute of New York
New York
New York
Cleveland Clinic - Neurological Institute
Cleveland
Ohio
University of Pennsylvania
Philadelphia
Pennsylvania
University Of Pittsburgh
Pittsburgh
Pennsylvania
Vanderbilt University Medical Center-Cognitive and Movement Disorders Group
Nashville
Tennessee
University of Texas Southwestern Medical Center ¿ Multiple System Atrophy Clinic
Dallas
Texas
Baylor College Of Medicine
Houston
Texas
Inland Northwest Research
Spokane
Washington
Liverpool Hospital - South Western Sydney Local Health District
Liverpool
New South Wales
Westmead Hospital
Westmead
New South Wales
Gold Coast Hospital
Southport
Queensland
The Alfred Hospital
Melbourne
Victoria
The Ottawa Hospital Research Institute
Ottawa
Ontario
Toronto Western Hospital (TWH)
Toronto
Ontario
Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
Bordeaux
France
Centre Hospitalier Regional Universitaire de Lille - Hopital Roger Salengro
Lille
France
CHU - Hospital de la Timone
Marseille
France
Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Saint-Eloi Guy de Chauliac
Montpellier
France
Chu De La Pitie-Salpetriere
Paris
France
Unite d'investigation clinique de Neurologie Rez-de-jardin-Bloc Hopital CHU Pontchaillou
Rennes
France
Centre Hospitalier Universitaire de Toulouse - Hopital Purpan
Toulouse
France
Movement Disorders Clinic
Beelitz-Heilstätten
Germany
Universitaetsklinikum Erlangen
Erlangen
Germany
Curiositas-ad-Sanum GmbH
Haag in Oberbayern
Germany
Ludwig-Maximilians-Universitat Munchen Klinikum der Universitat - Grosshadern
München
Germany
+ 34 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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