Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2/PHASE3
110 participants
INTERVENTIONAL
2023-12-21
2029-11-30
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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AMX0035
AMX0035 administered by mouth for 52 weeks: once daily for first 2 weeks and then twice daily for remainder of study
For participants electing to continue into the open-label phase at Week 52; AMX0035 will be administered once daily for first 2 weeks and then twice daily for remainder of open-label phase
AMX0035
Proprietary formulation of sodium phenylbutyrate and taurursodiol
Placebo
Placebo administered by mouth for 52 weeks: once daily for first 2 weeks and then twice daily for remainder of study
Placebo
Matching Placebo Comparator
Interventions
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AMX0035
Proprietary formulation of sodium phenylbutyrate and taurursodiol
Placebo
Matching Placebo Comparator
Eligibility Criteria
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Inclusion Criteria
* 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
* 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)
40 Years
80 Years
ALL
No
Sponsors
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Amylyx Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Amylyx Pharmaceuticals
Locations
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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
Padua, , 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
Madrid, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Countries
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Other Identifiers
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A35-009
Identifier Type: -
Identifier Source: org_study_id