AMX0035 and Progressive Supranuclear Palsy

Completed · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Progressive Supranuclear Palsy, PSP, Neurodegenerative Diseases, Atypical Parkinsonism

In brief

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.

Key facts

Study ID

NCT06122662

Run by

Amylyx Pharmaceuticals Inc.

People needed

139

Starts

2023-12-21

Expected to finish

2025-10-21

Last updated by the study team

2026-04-30

Who can join

Age: 40 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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)

Where it is running

• 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
• Pennsylvania Hospital — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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