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ACTIVE NOT RECRUITING

NCT06122662

PHASE2/PHASE3

AMX0035 and Progressive Supranuclear Palsy

Sponsor: Amylyx Pharmaceuticals Inc.

View on ClinicalTrials.gov

Summary

A35-009 (ORION) is a Phase 2b/3 trial to evaluate the efficacy and safety of AMX0035 in participants with Progressive Supranuclear Palsy (PSP), consisting of randomized, double blind placebo controlled phases, followed by an optional open-label extension phase.

Official title: A Phase 2b/3 Study of the Safety and Efficacy of AMX0035 in Progressive Supranuclear Palsy (ORION)

Key Details

Gender

All

Age Range

40 Years - 80 Years

Study Type

INTERVENTIONAL

Enrollment

110

Start Date

2023-12-21

Completion Date

2029-11-30

Last Updated

2024-12-20

Healthy Volunteers

Conditions

Progressive Supranuclear Palsy PSP Neurodegenerative Diseases Atypical Parkinsonism

Interventions

DRUG

AMX0035

Proprietary formulation of sodium phenylbutyrate and taurursodiol

OTHER

Placebo

Matching Placebo Comparator

Inclusion Criteria: * Male or female 40 to 80 years of age, inclusive * Diagnosis of possible or probable PSP Richardson Syndrome * Presence of PSP symptoms for \<5 years * Score of \<40 on the total (28-item) Progressive Supranuclear Palsy Rating Scale (PSPRS) * Able to walk independently or with minimal assistance * Minimum score of 24 on the Mini Mental State Examination (MMSE) * Must reside outside a skilled nursing facility or dementia care facility at the time of screening. Residence in an assisted living facility is allowed * Must have a study partner willing to attend study visits and provide information on participant's status * Capable of providing informed consent * Capable and willing to comply with trial procedures including visits to the trial clinic, visit requirements and treatment schedule, including MRI scans * Female participants of childbearing potential must agree to use effective birth control for the duration of the study and for 6 months after last dose of study drug. * Males must agree to use effective birth control method for the duration of the study and for 6 months after the last dose of study drug. Men must not plan to donate sperm. Exclusion Criteria: * Require use of a feeding tube * Evidence of any neurological disorder that could explain signs of PSP * Evidence of any clinically significant neurological disorder other than PSP, including significant cerebrovascular abnormalities, vascular dementia, motor neuron disease or ALS, Huntington's disease, normal pressure hydrocephalus, brain tumor, seizure disorder, multiple sclerosis, or known structural brain abnormalities. * History of autosomal dominant PSP due to a Microtubule Associated Protein Tau (MAPT) mutation * History of an autosomal dominant mutation associated with Frontotemporal Lobar Degeneration (FTLD) * Prior or current diagnosis of schizophrenia, schizoaffective disorder, or bipolar disorder * Presence of unstable psychiatric disease, cognitive impairment (e.g., major cognitive dysfunction), dementia, major depression, or substance abuse that would impair ability of the participant to provide informed consent and follow instructions * Abnormal liver function * Renal insufficiency * Ongoing anemia * History of Class III/IV heart failure per New York Heart Association (NYHA)

Locations (62)

Mayo Clinic

Scottsdale, Arizona, United States

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Parkinson's & Movement Disorder Institute

Fountain Valley, California, United States

University of Southern California

Los Angeles, California, United States

Cedars Sinai Medical Center

Los Angeles, California, United States

Stanford Movement Disorder Center

Palo Alto, California, United States

University of California, San Francisco (UCSF)

San Francisco, California, United States

UC Health Anschutz Outpatient Pavilion Anschutz Medical Campus

Aurora, Colorado, United States

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

UF Health Dorothy Mangurian Neuroimaging Suite

Gainesville, Florida, United States

Mayo Clinic Florida

Jacksonville, Florida, United States

Augusta University

Augusta, Georgia, United States

Parkinsons and Movement Disorder Center at the Queens Medical Center

Honolulu, Hawaii, United States

Northwestern University PD and Movement Disorders Center

Chicago, Illinois, United States

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, United States

University of Kentucky Medical Center

Lexington, Kentucky, United States

Ochsner Medical Center

New Orleans, Louisiana, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Lahey Hospital and Medical Center

Burlington, Massachusetts, United States

Quest Research Institute

Farmington Hills, Michigan, United States

University of Minnesota, CTSI

Minneapolis, Minnesota, United States

Mayo Clinic - Rochester

Rochester, Missouri, United States

Cleveland Clinic Lou Ruvo Center for Brain Health

Las Vegas, Nevada, United States

University of Cincinnati

Cincinnati, Ohio, United States

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Oregon Health & Science University

Portland, Oregon, United States

Pennsylvania Hospital

Philadelphia, Pennsylvania, United States

Medical University of South Carolina (MUSC)

Charleston, South Carolina, United States

Veracity Neuroscience, LLC

Memphis, Tennessee, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Kerwin Medical Center

Dallas, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

Central Texas Neurology Consultants

Round Rock, Texas, United States

University of Utah Movement Disorders Center

Salt Lake City, Utah, United States

Virginia Commonwealth University Department of Neurology

Richmond, Virginia, United States

Hôpital de la Timone

Marseille, Bouches-du-Rhône, France

Hopital Caremeau

Nîmes, Gard, France

Groupe Hospitalier Pellegrin - Hôpital Pellegrin

Bordeaux, Gironde, France

CHU de Limoges - Hôpital Dupuytren

Limoges, Haute Vienne, France

CHU Rennes - Hopital Pontchaillou

Rennes, Ille Et Vilaine, France

Hopital Roger Salengro - CHU Lille

Lille, Nord, France

Groupe Hospitalier Pitie-Salpetriere

Paris, France

Universitaetsklinikum Duesseldorf AoeR

Ulm, Baden-Wurttemberg, Germany

Universitaetsklinikum Ulm

Ulm, Baden-Wurttemberg, Germany

Krankenhaus Agatharied

Hausham, Bavaria, Germany

Klinikum der Universität München

Munich, Bavaria, Germany

Paracelsus-Elena-Klinik Kassel

Kassel, Hesse, Germany

Universitaetsklinikum Carl Gustav Carus TU Dresden

Dresden, Saxony, Germany

Asklepios Fachklinikum Stadtroda

Stadtroda, Thuringia, Germany

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, Italy

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy

Azienda Ospedale-Università di Padova

Padova, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

Azienda Ospedaliera Universitaria Policlinico Umberto I - Università di Roma La Sapienza

Roma, Italy

Azienda Ospedaliera Universitaria OO. RR. S. Giovanni di Dio e Ruggi D'Aragona

Salerno, Italy

Hospital de Cruces

Barakaldo, Bizkaia, Spain

Clinica Universidad de Navarra

Pamplona, Navarre, Spain

Hospital Universitari i Politecnic La Fe

Valencia, València, Spain

Hospital Clinic de Barcelona

Barcelona, Spain

Hospital Universitario de La Princesa

Madrid, Spain

Hospital Universitario Ramon y Cajal