New pill aims to stop dangerous blood pressure drops in rare brain disease

NCT ID NCT05696717

Symptom relief Sponsor: Theravance Biopharma Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 3 study tests a drug called ampreloxetine for people with multiple system atrophy (MSA) who experience dizziness and fainting due to low blood pressure when standing. About 102 adults with MSA will take the drug for 20 weeks, then some will switch to placebo to see if symptoms return. The goal is to see if ampreloxetine safely improves daily activities and reduces symptoms.

What this could mean

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

Active substance: ampreloxetine (oral tablet)
What this could lead to: If it works, this could provide a new treatment option to reduce dizziness and fainting in people with multiple system atrophy, making daily activities easier.
What could go wrong: This is a mid-stage trial with only 102 participants. The drug may not work better than placebo, and side effects could occur. Results may not apply to all MSA patients.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 102 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2023

Expected to finish

Jan 2028

An estimate. End dates often move.

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

30 years and older

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: * Participant is male or female and at least 30 years old. * Participant has a diagnosis of possible or probable MSA of the Parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) according to The Gilman Criteria (2008). * Participant has a diagnosis of possible or probable MSA of the Parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) confirmed by the Enrollment Steering Committee (ESC). * Participant must meet the diagnostic criteria of nOH, as demonstrated by a sustained reduction in BP of ≥20 mmHg (systolic) or ≥10 mmHg (diastolic) within 3 min of standing as part of orthostatic standing test or being tilted up ≥60o from a supine position as determined by a tilt-table test. * Participant must score ≤4 on UMSARS Part IV at Visit 1 (Screening). * Participant must score at least a 4 on the OHSA item 1 at Visit 2 (Day 1). * Participant must be willing to not take any prohibited medications during the study. * If participant is female, the participant must not be pregnant, breastfeeding, or planning a pregnancy during the course of the study. A woman of childbearing potential must have a documented negative pregnancy test at screening. * During the study and for 30 days after receiving the last dose of the study drug, females of childbearing potential or males capable of fathering children must agree to use highly effective birth control measures (failure rate \<1% when used consistently and correctly) or agree to abstain from sexual intercourse. * Participant is willing and able to provide signed and dated written informed consent to -participate prior to initiation of any study related procedures. Participant is able to communicate well with the Investigator and clinic staff, understands the expectations of the study and is able to comply with the study procedures, requirements, and restrictions. Exclusion Criteria: * Participant has a systemic illness known to produce autonomic neuropathy, including, but not limited to, amyloidosis and autoimmune neuropathies. Participant with diabetes mellitus (DM) will be evaluated on a case-by-case basis by the medical monitor and considered ineligible unless they meet all of the following criteria: * Well controlled type-2 DM in treatment with only oral medications and diet * HbA1C of ≤7.5% performed during screening or up to 12 weeks before screening * No clinically evident peripheral neuropathy (e.g., normal sensory examination on peripheral extremities) * No known retinopathy (e.g., annual ophthalmic exam is sufficient) * No nephropathy (e.g., absence of albuminuria and GFR \>60). * Participant has a known intolerance to other NRIs or SNRIs. * Participant currently uses concomitant antihypertensive medication for the treatment of essential hypertension. * Participant has used strong CYP1A2 inhibitors or inducers within 7 days or 5 half lives, whichever is longer, prior to Visit 2 (Day 1) or requires concomitant use until the Safety follow-up Visit. * Participant has changed dose, frequency, or type of prescribed medication for orthostatic hypotension within 7 days prior to Visit 2 (Day 1). * Midodrine and droxidopa (if applicable) must be tapered off and stopped at least 7 days prior to Visit 2 (Day 1). * Participant has known or suspected alcohol or substance abuse within the past 12 months (Diagnostic and Statistical Manual of Mental Disorders, Fourth Edition, Text Revision \[DSM IV TR®\] definition of alcohol or substance abuse). * Participant has clinically unstable coronary artery disease or had a major cardiovascular event (e.g., myocardial infarction) in the past 6 months. * Participant has significant uncontrolled cardiac arrhythmia, history of complete heart block, or significant QTc prolongation (≥450 msec for males and ≥470 msec for females). * Participant has a new onset of a neurological event (i.e., seizures, confusion, altered levels of consciousness, etc.) in the past 6 months. * Participant has used any monoamine oxidase inhibitor (MAOI) within 14 days prior to Visit 2 (Day 1). * Participant has a history of untreated closed angle glaucoma, or treated closed angle glaucoma that, in the opinion of an ophthalmologist, might result in an increased risk to the participant. * Participant has a Montreal Cognitive Assessment (MoCA) \<21. * Participant is unable or unwilling to complete all protocol specified procedures including questionnaires. * Participant has known congestive heart failure (New York Heart Association \[NYHA\] Class 3 or 4). * Participant has had any malignant disease, other than carcinoma in situ of the cervix or basal cell carcinoma, within the past 2 years prior to Screening. * Participant has a known gastrointestinal (GI) condition, which in the Investigator's judgment, may affect the absorption of study medication (e.g., ulcerative colitis, gastric bypass). * Participant has psychiatric, neurological, or behavioral disorders that may interfere with the cognitive ability of the participant to give informed consent, understand and comply with study procedures, or interfere with the conduct of the study. * Participant is currently receiving any investigational drug or has received an investigational drug within 30 days of dosing. An investigational drug is defined as a drug that is not approved by a regulatory agency (e.g., Food and Drug Administration \[FDA\]). * Participant has a clinically significant abnormal laboratory finding(s) (e.g., alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\] ≥3.0 x upper limit of normal \[ULN\]; blood bilirubin \[total\] ≥3.0 x ULN; estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m2, or any abnormal laboratory value that could interfere with safety of the participant). * Participant has demonstrated lifetime suicidal ideation and/or suicidal behavior, as outlined by the C-SSRS (Baseline/Screening Version). Participant should be assessed by the rater for risk of suicide and the participant's appropriateness for inclusion in the study. * Participant has a concurrent disease or condition (e.g., COVID-19), or recent surgery, that in the opinion of the Investigator, would confound or interfere with study participation or evaluation of safety, tolerability, or absorption of the study drug. * Participant has known hypersensitivity to ampreloxetine (ampreloxetine hydrochloride), or any excipients in the formulation. * Major surgery (i.e., procedures involving higher risk for infection and extended recovery period, such as, joint replacement, gastric bypass, open heart surgery, organ transplant, etc.) occurring less than 4 weeks prior to enrollment.

Get updates

Get notified about this study

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

multiple system atrophy primary orthostatic hypotension Shy-Drager Syndrome

As listed by the trial registrant

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

Msa - multiple system atrophy Symptomatic neurogenic orthostatic hypotension

Contacts and locations

Locations

AOU San Giovanni di Dio e Ruggi d'Aragona, Salerno

Salerno, 84131, Italy
Aqualane Clinical Research

Naples, Florida, 34105, United States
Astra Clinic (Clinic4U)

Tallinn, 11315, Estonia
Auckland City Hospital

Grafton, Auckland, 1023, New Zealand
Autonomic Unit, National Hospital for Neurology & Neurosurgery

London, WC1N 3BG, United Kingdom
Azienda Ospedaliera Santa Maria di Terni

Terni, I-05100, Italy
Azienda Ospedaliera Universitaria Policlinico Tor Vergata

Rome, 00133, Italy
Barts Health

London, EC1M 6BQ, United Kingdom
Baylor College of Medicine

Houston, Texas, 77030, United States
Bispebjerg Hospital

Copenhagen, 2400-NV, Denmark
Brigham and Women's Hospital (Neuromuscular Division)

Boston, Massachusetts, 02115, United States
CNS-Campus Neurologico Senior

Torres Vedras, 2560-280, Portugal
Centre Hospitalier Universitaire de Bordeaux Health

Bordeaux, 33076, France
Centre d'Investigation Clinique Hôpital Pierre Paul Rique

Toulouse, 31059 Cedex, France
Centro de Pesquisa Clínica (CPC) do Hospital das Clínicas de Porto Alegre (HCPA)

Porto Alegre, 90035-903, Brazil
Charite Universitaetsmedizin Berlin

Berlin, 12203, Germany
Charité - Universitätsmedizin Berlin- Campus Mitte

Berlin, 10117, Germany
David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States
Emory University

Atlanta, Georgia, 30329, United States
Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, 00168, Italy
Fundació Assistencial Mutua de Terrassa

Terrassa, Barcelona, 08222, Spain
Fundación Scherbovsky

Mendoza, M5500, Argentina
Hawaii Pacific Neuroscience

Honolulu, Hawaii, 96817, United States
Hopital Caremeau

Nîmes, Occitanie, Cedex 9, France
Hospital Británico de Buenos Aires

CABA, C1280AEB, Argentina
Hospital General de Agudos Jose Maria Ramos Mejía

CABA, C1221ADC, Argentina
Hospital Germans Trias i Pujol, Department of Neurology

Barakaldo, Bizkaia, 48903, Spain
Hospital São Lucas - PUCRS

Porto Alegre, 90610-000, Brazil
Hospital Universitario Infante Sofia Paseo Europa

Madrid, 28702, Spain
Hospital Universitario de La Princesa

Madrid, 28006, Spain
Hospital de Clínicas - Universidade Federal de Minas Gerais (HC - UFMG)

Belo Horizonte, 30130-100, Brazil
INEBA Instituto Neurociencias Buenos Aires

CABA, Buenos Aires F.D., C1192AAW, Argentina
IRCCS Istituto de Scienze Neurologiche di Bologna (ISNB)

Bologna, 40139, Italy
Instituto Fleni

CABA, Buenos Aires F.D., C1428AQK, Argentina
Instituto Investigación Sanitaria Biocruces

Barakaldo, Bizkaia, 48903, Spain
Instituto de Neurologia de Curitiba S\C LTDA

Curitiba, Paraná, 81210-310, Brazil
Instytut Zdrowia dr Boczarska-Jedynak Sp. z o.o., Sp.k.

Oświęcim, 32-600, Poland
Krakowska Akademia Neurologii Sp.zo.o.

Krakow, 31-505, Poland
Massachusetts Chan Medical School

Worcester, Massachusetts, 01655, United States
Medical University of Innsbruck

Innsbruck, A-6020, Austria
Medstar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Monash Health

Clayton, Victoria, 3168, Australia
Movement Disorders Center of Arizona

Scottsdale, Arizona, 85258, United States
Movement Disorders and Autonomic Disorders Clinic; University of Utah

Salt Lake City, Utah, 84108, United States
Médico/Hospital de la Policía Federal Churruca Visca

CABA, Buenos Aires F.D., C1437JCP, Argentina
NYU Langone Health NYU Dysautonomia Center

New York, New York, 10016, United States
National Taiwan University Cancer Center

Taipei, Taiwan, 106, Taiwan
Neuro-Care Sp. z o.o. Sp. Komandytowa

Katowice, 40-749, Poland
Neurocentrum-Miwomed

Gdansk, 80-207, Poland
Neurostudies, Inc

Port Charlotte, Florida, 33952, United States
Northshore University Health System

Glenview, Illinois, 60026-1339, United States
Parkinson's Centre of Ospedale CTO

Milan, 20126, Italy
Parkinson's Disease And Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States
Pia Fond. Cardinale Giovanni Panico Azienda Ospedaliera

Tricase, 73039, Italy
Praxis Dr. Oehlwein, Outpatient Clinic

Gera, Thuringia, 07551, Germany
Quest Research Institute

Farmington Hills, Michigan, 48334, United States
Rush University Medical Center

Chicago, Illinois, 60612, United States
SFM Clinical Research, LLC

Boca Raton, Florida, 33487, United States
Salford Royal Hospital

Salford, M6 8HD, United Kingdom
Santa Chiara Hospital

Trento, 38122, Italy
Semmelweis Egyetem

Budapest, 1085, Hungary
Stanford Neuroscience Health Center

Palo Alto, California, 94304, United States
Tartu University Hospital

Tartu, 30029, Estonia
The Alfred Hospital

Melbourne, Victoria, 3168, Australia
The Neurological Institute at Columbia University Medical Center

New York, New York, 10032, United States
The Parkinson's and Movement Disorder Institute

Fountain Valley, California, 92708, United States
Theravance Biopharma Investigative Site

Parkville, Victoria, 3050, Australia
Theravance Biopharma Investigative Site

Tel Aviv, 64239, Israel
Theravance Biopharma Investigative Site

Christchurch, 8011, New Zealand
UC San Diego Movement Disorder Center

La Jolla, California, 92037, United States
Universitatsklinikum Tulln

Tulln, Austria
University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2GW, United Kingdom
University of Belgrade Neurology Clinic

Belgrade, 11000, Serbia
University of Calgary - Health Sciences Centre

Calgary, Alberta, T2N 4Z6, Canada
University of Cincinnati Medical Center

Cincinnati, Ohio, 45219, United States
University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, 66160, United States
University of South Florida Ataxia Research Center

Tampa, Florida, 33612, United States
University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States
Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

More trials for these conditions

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