AMX0035 and Progressive Supranuclear Palsy

Last updated: December 19, 2024
Sponsor: Amylyx Pharmaceuticals Inc.
Overall Status: Active - Not Recruiting

Phase

2/3

Condition

Dyskinesias

Progressive Supranuclear Palsy

Cataplexy

Treatment

Placebo

AMX0035

Clinical Study ID

NCT06122662
A35-009
  • Ages 40-80
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 timeof screening. Residence in an assisted living facility is allowed

  • Must have a study partner willing to attend study visits and provide information onparticipant's status

  • Capable of providing informed consent

  • Capable and willing to comply with trial procedures including visits to the trialclinic, visit requirements and treatment schedule, including MRI scans

  • Female participants of childbearing potential must agree to use effective birthcontrol for the duration of the study and for 6 months after last dose of studydrug.

  • Males must agree to use effective birth control method for the duration of the studyand for 6 months after the last dose of study drug. Men must not plan to donatesperm.

Exclusion

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 neurondisease 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 LobarDegeneration (FTLD)

  • Prior or current diagnosis of schizophrenia, schizoaffective disorder, or bipolardisorder

  • Presence of unstable psychiatric disease, cognitive impairment (e.g., majorcognitive dysfunction), dementia, major depression, or substance abuse that wouldimpair ability of the participant to provide informed consent and followinstructions

  • Abnormal liver function

  • Renal insufficiency

  • Ongoing anemia

  • History of Class III/IV heart failure per New York Heart Association (NYHA)

Study Design

Total Participants: 110
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
December 21, 2023
Estimated Completion Date:
November 30, 2029

Study Description

AMX0035 is a fixed dose combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the endoplasmic reticulum (ER) and mitochondria. This clinical trial is designed to demonstrate that AMX0035 is safe and tolerable, and to assess its effect on disease progression as measured by the Progressive Supranuclear Palsy (PSP) Rating Scale (PSPRS) over a 52-week double-blind phase. The Phase 2b and Phase 3 study portions are planned to feature an identical design: a randomized, double-blind, placebo-controlled phase that is followed by an optional open-label extension (OLE) phase. The phase 3 portion of ORION may be initiated based on results of the phase 2b Interim Analysis and/or the Primary Analysis and the totality of data from the Phase 2b study portion.

Connect with a study center

  • Hôpital de la Timone

    Marseille cedex 05, Bouches-du-Rhône 13385
    France

    Site Not Available

  • Hopital Caremeau

    Nimes Cedex 09, Gard 30029
    France

    Site Not Available

  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin

    Bordeaux cedex, Gironde 30076
    France

    Site Not Available

  • CHU de Limoges - Hôpital Dupuytren

    Limoges, Haute Vienne 87042
    France

    Site Not Available

  • CHU Rennes - Hopital Pontchaillou

    Rennes cedex 09, Ille Et Vilaine 35033
    France

    Site Not Available

  • Hopital Roger Salengro - CHU Lille

    Lille Cedex, Nord 59037
    France

    Site Not Available

  • Groupe Hospitalier Pitie-Salpetriere

    Paris, 75013
    France

    Site Not Available

  • Universitaetsklinikum Duesseldorf AoeR

    Ulm, Baden Wuerttemberg 89081
    Germany

    Site Not Available

  • Universitaetsklinikum Ulm

    Ulm, Baden Wuerttemberg 89081
    Germany

    Site Not Available

  • Krankenhaus Agatharied

    Hausham, Bayern 83734
    Germany

    Site Not Available

  • Klinikum der Universität München

    Muenchen, Bayern
    Germany

    Site Not Available

  • Paracelsus-Elena-Klinik Kassel

    Kassel, Hessen 34128
    Germany

    Site Not Available

  • Universitaetsklinikum Carl Gustav Carus TU Dresden

    Dresden, Sachsen 1307
    Germany

    Site Not Available

  • Asklepios Fachklinikum Stadtroda

    Stadtroda, Thueringen 7646
    Germany

    Site Not Available

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milano, 20122
    Italy

    Site Not Available

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milano, 20133
    Italy

    Site Not Available

  • Azienda Ospedale-Università di Padova

    Padova, 35128
    Italy

    Site Not Available

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

    Roma, 185
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168
    Italy

    Site Not Available

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

    Salerno, 84131
    Italy

    Site Not Available

  • Hospital de Cruces

    Barakaldo, Bizkaia 48903
    Spain

    Site Not Available

  • Clinica Universidad de Navarra

    Pamplona, Navarra 31008
    Spain

    Site Not Available

  • Hospital Universitari i Politecnic La Fe

    Valencia, València 46026
    Spain

    Site Not Available

  • Hospital Clinic de Barcelona

    Barcelona, 8036
    Spain

    Site Not Available

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034
    Spain

    Site Not Available

  • Hospital Universitario de La Princesa

    Madrid, 28006
    Spain

    Site Not Available

  • Hospital Universitario Virgen del Rocio

    Seville, 41013
    Spain

    Site Not Available

  • Mayo Clinic

    Scottsdale, Arizona 85259
    United States

    Site Not Available

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas 72205
    United States

    Site Not Available

  • Parkinson's & Movement Disorder Institute

    Fountain Valley, California 92708
    United States

    Site Not Available

  • Cedars Sinai Medical Center

    Los Angeles, California 90048
    United States

    Site Not Available

  • University of Southern California

    Los Angeles, California 90033
    United States

    Site Not Available

  • Stanford Movement Disorder Center

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of California, San Francisco (UCSF)

    San Francisco, California 94158
    United States

    Site Not Available

  • UC Health Anschutz Outpatient Pavilion Anschutz Medical Campus

    Aurora, Colorado 80045
    United States

    Site Not Available

  • Parkinson's Disease and Movement Disorders Center of Boca Raton

    Boca Raton, Florida 33486
    United States

    Site Not Available

  • UF Health Dorothy Mangurian Neuroimaging Suite

    Gainesville, Florida 32608
    United States

    Site Not Available

  • Mayo Clinic Florida

    Jacksonville, Florida 32224
    United States

    Site Not Available

  • Augusta University

    Augusta, Georgia 30912
    United States

    Site Not Available

  • Parkinsons and Movement Disorder Center at the Queens Medical Center

    Honolulu, Hawaii 96813
    United States

    Site Not Available

  • Northwestern University PD and Movement Disorders Center

    Chicago, Illinois 60611
    United States

    Site Not Available

  • University of Kansas Medical Center Research Institute, Inc.

    Kansas City, Kansas 66160
    United States

    Site Not Available

  • University of Kentucky Medical Center

    Lexington, Kentucky 40536-0274
    United States

    Site Not Available

  • Ochsner Medical Center

    New Orleans, Louisiana 70121
    United States

    Site Not Available

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Lahey Hospital and Medical Center

    Burlington, Massachusetts 01805
    United States

    Site Not Available

  • Quest Research Institute

    Farmington Hills, Michigan 48334
    United States

    Site Not Available

  • University of Minnesota, CTSI

    Minneapolis, Minnesota 55414
    United States

    Site Not Available

  • Mayo Clinic - Rochester

    Rochester, Missouri 55905
    United States

    Site Not Available

  • Cleveland Clinic Lou Ruvo Center for Brain Health

    Las Vegas, Nevada 89106
    United States

    Site Not Available

  • University of Cincinnati

    Cincinnati, Ohio 45219
    United States

    Site Not Available

  • The Cleveland Clinic Foundation

    Cleveland, Ohio 44195
    United States

    Site Not Available

  • Oregon Health & Science University

    Portland, Oregon 97239
    United States

    Site Not Available

  • Pennsylvania Hospital

    Philadelphia, Pennsylvania 19107
    United States

    Site Not Available

  • Medical University of South Carolina (MUSC)

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • Veracity Neuroscience, LLC

    Memphis, Tennessee 38157
    United States

    Site Not Available

  • Vanderbilt University Medical Center

    Nashville, Tennessee 37232
    United States

    Site Not Available

  • Kerwin Medical Center

    Dallas, Texas 75231
    United States

    Site Not Available

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Site Not Available

  • Central Texas Neurology Consultants

    Round Rock, Texas 78681
    United States

    Site Not Available

  • University of Utah Movement Disorders Center

    Salt Lake City, Utah 84108
    United States

    Site Not Available

  • Virginia Commonwealth University Department of Neurology

    Richmond, Virginia 23298-0059
    United States

    Site Not Available

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