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Can a failed ALS drug help PSP patients? new trial results are in

NCT ID NCT06122662

Disease control Sponsor: Amylyx Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This completed Phase 2b/3 trial tested the drug AMX0035 in 139 people with progressive supranuclear palsy (PSP), a rare brain disease that causes problems with balance, movement, and thinking. The study compared the drug to a placebo to see if it could slow the disease. The results will help determine if AMX0035 is safe and effective enough to pursue as a treatment for PSP.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AMX0035 (a combination of sodium phenylbutyrate and taurursodiol)
What this could lead to
If successful, AMX0035 could slow the progression of PSP, a rare and devastating brain disease, giving patients more time with better function.
What could go wrong
This is an early-to-mid-stage trial with only 139 participants. The drug may not show a meaningful benefit, and side effects could limit its use. Previous trials of AMX0035 for ALS failed to confirm benefit.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

139 people

The number who actually took part.

Started

Dec 2023

Finished

Oct 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

40 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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)

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Conditions

The condition(s) this trial relates to.

familial spontaneous pneumothorax neurodegenerative disease progressive supranuclear palsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Atypical parkinsonism Neurodegenerative diseases Progressive supranuclear palsy PSP

Contacts and locations

Locations

  • Asklepios Fachklinikum Stadtroda

    Stadtroda, Thuringia, 7646, Germany

  • Augusta University

    Augusta, Georgia, 30912, United States

  • Azienda Ospedale-Università di Padova

    Padova, 35128, Italy

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

    Salerno, 84131, Italy

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

    Roma, 185, Italy

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • CHU Rennes - Hopital Pontchaillou

    Rennes, Ille Et Vilaine, 35033, France

  • CHU de Limoges - Hôpital Dupuytren

    Limoges, Haute Vienne, 87042, France

  • Cedars Sinai Medical Center

    Los Angeles, California, 90048, United States

  • Central Texas Neurology Consultants

    Round Rock, Texas, 78681, United States

  • Cleveland Clinic Lou Ruvo Center for Brain Health

    Las Vegas, Nevada, 89106, United States

  • Clinica Universidad de Navarra

    Pamplona, Navarre, 31008, Spain

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168, Italy

  • Groupe Hospitalier Pellegrin - Hôpital Pellegrin

    Bordeaux, Gironde, 30076, France

  • Groupe Hospitalier Pitie-Salpetriere

    Paris, 75013, France

  • Hopital Caremeau

    Nîmes, Gard, 30029, France

  • Hopital Roger Salengro - CHU Lille

    Lille, Nord, 59037, France

  • Hospital Clinic de Barcelona

    Barcelona, 8036, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, València, 46026, Spain

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • Hospital Universitario de La Princesa

    Madrid, 28006, Spain

  • Hospital de Cruces

    Barakaldo, Bizkaia, 48903, Spain

  • Hôpital de la Timone

    Marseille, Bouches-du-Rhône, 13385, France

  • Kerwin Medical Center

    Dallas, Texas, 75231, United States

  • Klinikum der Universität München

    Munich, Bavaria, Germany

  • Krankenhaus Agatharied

    Hausham, Bavaria, 83734, Germany

  • Lahey Hospital and Medical Center

    Burlington, Massachusetts, 01805, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    Scottsdale, Arizona, 85259, United States

  • Mayo Clinic - Rochester

    Rochester, Missouri, 55905, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Medical University of South Carolina (MUSC)

    Charleston, South Carolina, 29425, United States

  • Northwestern University PD and Movement Disorders Center

    Chicago, Illinois, 60611, United States

  • Ochsner Medical Center

    New Orleans, Louisiana, 70121, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Paracelsus-Elena-Klinik Kassel

    Kassel, Hesse, 34128, Germany

  • Parkinson's & Movement Disorder Institute

    Fountain Valley, California, 92708, United States

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

    Boca Raton, Florida, 33486, United States

  • Parkinsons and Movement Disorder Center at the Queens Medical Center

    Honolulu, Hawaii, 96813, United States

  • Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19107, United States

  • Quest Research Institute

    Farmington Hills, Michigan, 48334, United States

  • Stanford Movement Disorder Center

    Palo Alto, California, 94304, United States

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • UC Health Anschutz Outpatient Pavilion Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • UF Health Dorothy Mangurian Neuroimaging Suite

    Gainesville, Florida, 32608, United States

  • Universitaetsklinikum Carl Gustav Carus TU Dresden

    Dresden, Saxony, 1307, Germany

  • Universitaetsklinikum Duesseldorf AoeR

    Ulm, Baden-Wurttemberg, 89081, Germany

  • Universitaetsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205, United States

  • University of California, San Francisco (UCSF)

    San Francisco, California, 94158, United States

  • University of Cincinnati

    Cincinnati, Ohio, 45219, United States

  • University of Kansas Medical Center Research Institute, Inc.

    Kansas City, Kansas, 66160, United States

  • University of Kentucky Medical Center

    Lexington, Kentucky, 40536-0274, United States

  • University of Minnesota, CTSI

    Minneapolis, Minnesota, 55414, United States

  • University of Southern California

    Los Angeles, California, 90033, United States

  • University of Utah Movement Disorders Center

    Salt Lake City, Utah, 84108, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Veracity Neuroscience, LLC

    Memphis, Tennessee, 38157, United States

  • Virginia Commonwealth University Department of Neurology

    Richmond, Virginia, 23298-0059, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.