MedPath

Clinical Effect of Ampreloxetine (TD-9855) for Treating Symptomatic nOH in Subjects With Primary Autonomic Failure

Phase 3
Completed
Conditions
Symptomatic Neurogenic Orthostatic Hypotension
Interventions
Drug: Placebo
Registration Number
NCT03750552
Lead Sponsor
Theravance Biopharma
Brief Summary

A Phase 3 study to evaluate efficacy, safety, and tolerability of ampreloxetine (TD-9855) in subjects with primary autonomic failures (MSA, PD, or PAF) and symptomatic nOH with up to 4 weeks of treatment.

Detailed Description

A Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multicenter study to evaluate efficacy, safety, and tolerability of ampreloxetine (TD-9855) in subjects with primary autonomic failures (MSA, PD, or PAF) and symptomatic nOH. The study consists of 3 periods: (i) 4-week screening, (ii) 4-week randomized treatment, and (iii) 2-week follow up. The trial utilizes an operational design featuring the ability to conduct protocol required visits as either in clinic or remote visits (except Screening visit).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
195
Inclusion Criteria
  • Subject is male or female and at least 30 years old.
  • Subject must meet the diagnostic criteria of symptomatic nOH, as demonstrated by a sustained reduction in BP of ≥20 mm Hg (systolic) or ≥10 mm Hg (diastolic) within 3 minutes of being tilted-up to ≥60o from a supine position as determined by a tilt-table test.
  • Subject must score at least a 4 on the Orthostatic Hypotension Symptom Assessment Question #1 at randomization visit.
  • For subjects with PD only: Subject has a diagnosis of PD according to the United Kingdom Parkinson's Disease Society (UKPDS) Brain Bank Criteria (1992).
  • For subjects with MSA only: Subject 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).
  • For subjects with PAF only: Subject has documented impaired autonomic reflexes, including the Valsalva maneuver performed within 24 months from the date of randomization.
  • Subject has plasma NE levels >100 pg/mL after being in seated position for 30 minutes.
Exclusion Criteria
  • Subject has a known systemic illness known to produce autonomic neuropathy, including but not limited to amyloidosis, and autoimmune neuropathies.

  • Subject has a known intolerance to other NRIs or SNRIs.

  • Subject currently uses concomitant antihypertensive medication for the treatment of essential hypertension unrelated to autonomic dysfunction.

  • Subject has used strong CYP1A2 inhibitors or inducers within 7 days or 5 half-lives, whichever is longer, prior to randomization or requires concomitant use until the follow-up visit.

  • Subject has changed dose, frequency, or type of prescribed medication for orthostatic hypotension within 7 days prior to V1.

    • Midodrine and droxidopa (if applicable) must be tapered off at least 7 days prior to V1.
  • Subject has a known or suspected alcohol or substance abuse within the past 12 months (DSM-IV-TR® definition of alcohol or substance abuse).

  • Subject has a clinically unstable coronary artery disease, or major cardiovascular or neurological event in the past 6 months.

  • Subject has used any monoamine oxidase inhibitor (MAO-I) within 14 days prior to randomization.

  • Subject 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 subject.

  • Subject has any significant uncontrolled cardiac arrhythmia.

  • Subject has a Montreal Cognitive Assessment (MoCA) ≤23.

  • Subject had a myocardial infarction in the past 6 months or has current unstable angina.

  • Subject has known congestive heart failure (New York Heart Association [NYHA] Class 3 or 4).

  • Subject has a clinically significant abnormal laboratory findings (e.g., alanine aminotransferase [ALT] or aspartate aminotransferase [AST] >3.0 x upper limit of normal [ULN]; blood bilirubin [total] >1.5 x ULN; estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2, or any abnormal laboratory value that could interfere with safety of the subject).

  • Subject has demonstrated a history of lifetime suicidal ideation and/or suicidal behavior, as outlined by the C-SSRS (Columbia Suicide Severity Rating Scale) (Baseline/Screening Version) subject should be assessed by the rater for risk of suicide and the subject's appropriateness for inclusion in the study.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlaceboParticipants randomized to Placebo will receive a single, oral, daily dose of placebo for 4 weeks.
ampreloxetineampreloxetineParticipants randomized to ampreloxetine will receive a single, oral, daily dose of active drug for 4 weeks.
Primary Outcome Measures
NameTimeMethod
Change From Baseline in Orthostatic Hypotension Symptom Assessment (OHSA) Question #1 Score at Week 4Baseline and Week 4

OHSA is an assessment of the severity of symptoms from low blood pressure. OHSA is a 6 question symptom assessment scale where each question uses an 11 point scale from 0 to 10, with 0 indicating no symptoms/no interference and 10 indicating the worst possible symptoms/complete interference.

Question #1 assesses dizziness, lightheadedness, feeling faint, or feeling like you might blackout.

A mean negative change from baseline indicates a better outcome.

Secondary Outcome Measures
NameTimeMethod
Change From Baseline in Orthostatic Hypotension Symptom Assessment (OHSA) Composite Score at Week 4Baseline and Week 4

OHSA is an assessment of the severity of symptoms from low blood pressure. OHSA is a 6 question symptom assessment scale in which the composite score uses an 11 point scale from 0 to 10, with 0 indicating no symptoms/no interference and 10 indicating the worst possible symptoms/complete interference.

A mean negative change from baseline indicates a better outcome.

Change From Baseline in Orthostatic Hypotension Daily Activities Scale (OHDAS) Composite Score at Week 4Baseline and Week 4

OHDAS is an assessment of how low blood pressure symptoms affect daily life. OHDAS is a 4 item assessment in which the composite score uses an 11 point scale from 0 to 10, with 0 indicating no symptoms/no interference and 10 indicating the worst possible symptoms/complete interference.

A mean negative change from baseline indicates a better outcome.

Number of Participants Who Experienced an Improvement From Baseline in Patient Global Impression of Change (PGI-C) Score at Week 4Baseline and Week 4

PGI-C was assessed using a 5-point scale where participants were asked to compare their current condition to their condition at baseline from 1 to 5, with 1 indicating the condition is very much improved and 5 indicating the condition is very much worse. These scores were analyzed in 2 categories: better and no change/worse.

Number of Participants Who Experienced at Least One FallUp to Week 4

Trial Locations

Locations (124)

Parkinson's Disease and Movement Disorders Center

🇺🇸

Boca Raton, Florida, United States

NorthShore University Health System

🇺🇸

Glenview, Illinois, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Royal Brisbane and Women's Hospital

🇦🇺

Herston, Queensland, Australia

University Health Network - Toronto Western Hospital Movement Disorders Clinic

🇨🇦

Toronto, Ontario, Canada

UMHAT Sveti Georgi EAD Clinic of Neurological Diseases

🇧🇬

Plovdiv, Bulgaria

MHATNP Sv.Naum, EAD

🇧🇬

Sofia, Bulgaria

Clinexpert Kft.

🇭🇺

Budapest, Hungary

Instytut Zdrowia dr Boczarska-Jedynak

🇵🇱

Oświęcim, Poland

Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii Klinicznej

🇵🇱

Kraków, Poland

Astra Team Clinic

🇪🇪

Tallinn, Estonia

Hospital Senhora da Oliveira - Guimaraes, EPE

🇵🇹

Guimaraes, Portugal

Hopital Roger Salengro - CHU Lille

🇫🇷

Lille Cedex, Nord, France

Campus Neurologico Senior

🇵🇹

Torres Vedras, Portugal

Bispebjerg og Frederiksberg Hospital

🇩🇰

Copenhagen, Denmark

Odense Universitetshospital

🇩🇰

Odense, Denmark

A.O. Santa Maria

🇮🇹

Terni, Italy

Sapienza University of Rome

🇮🇹

Roma, Italy

Collaborative Neuroscience Network, LLC

🇺🇸

Long Beach, California, United States

Georgetown University Hospital, Dept. of Neurology

🇺🇸

Washington, District of Columbia, United States

Azienda Ospedaliero Universitaria Pisana

🇮🇹

Pisa, Italy

Hospital General Universitario Gregorio Marañon

🇪🇸

Madrid, Spain

Universita di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche Ospedale Bellaria

🇮🇹

Bologna, Italy

CHU Caremeau

🇫🇷

Nîmes, France

Azienda Ospedaliero - Universitaria Policlinico Vittorio Emanuele. - P.O G. Rodolico, Clinica Neurologica

🇮🇹

Catania, Italy

Ospedale San Raffaele I U.O. di Neurologia

🇮🇹

Milano, Italy

Hospital Pierre Paul Rquet, CHU Purpan

🇫🇷

Toulouse, France

Universitaetsklinikum Freiburg - Klinik fur Neurologie und Neurophysiologie

🇩🇪

Freiburg, Baden Wuerttemberg, Germany

Alexianer St. Joseph-Krankenhaus Berlin-Weißensee, Klinik für Neurologie

🇩🇪

Berlin, Germany

Pecsi Tudomanyegyetem, Neurologiai Klinika

🇭🇺

Pécs, Hungary

Fondazione Istituto G.Giglio di Cefalù

🇮🇹

Cefalù, Palermo, Italy

Ospedale San Giovanni Battista

🇮🇹

Roma, Italy

Charite - Campus Virchow-Klinikum, Klinik fur Neurologie

🇩🇪

Berlin, Germany

Istituto Clinico Humanitas - IRCCS

🇮🇹

Rozzano, Milano, Italy

Fondazione PTV - Policlinico Tor Vergata I U.O.C. Neurologia

🇮🇹

Roma, Italy

Universita Gabriele D'Annunzio- Cesi-Met

🇮🇹

Chieti, Italy

Szent Borbala Korhaz

🇭🇺

Tatabanya, Hungary

PRATIA MCM Kraków

🇵🇱

Kraków, Poland

Charite - Campus Virchow-Klinikum

🇩🇪

Berlin, Germany

Ospedale San Giovanni Battista del Sovrano Militare Ordine di Malta

🇮🇹

Roma, Italy

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

🇮🇹

Milano, Italy

NEURO-CARE Sp. z o.o. Sp. Komandytowa

🇵🇱

Siemianowice Śląskie, Poland

Policlinico Umberto I - Universita degli Studi di Roma La Sapienza / Neurologia

🇮🇹

Roma, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS / Istituto di Neurologia - Ambulatorio Disturbi del Movimento

🇮🇹

Roma, Italy

City Neurological Center Sibneiromed

🇷🇺

Novosibirsk, Russian Federation

SBEIHPE Novosibirsk State Medical University

🇷🇺

Novosibirsk, Russian Federation

Specjalistyczna Praktyka Lekarska prof.Grzegorz Opala

🇵🇱

Katowice, Poland

Samodzielny Publiczny Szpital Kliniczny Nr 4 w Lublinie

🇵🇱

Lublin, Poland

Specjalistyczne Gabinety sp. z o.o.

🇵🇱

Warszawa, Poland

Centro Hospitalar e Universitario Lisboa Norte - Hospital de Santa Maria

🇵🇹

Lisboa, Portugal

Communal Institution Dnipropetrovsk I.I.Mechnikov RCH

🇺🇦

Dnipro, Ukraine

Hospital Universitario Mutua de Terrasa

🇪🇸

Barcelona, Spain

Hospital de Cruces

🇪🇸

Barakaldo, Vizcaya, Spain

Complejo Hospitalario de Navarra

🇪🇸

Pamplona, Navarra, Spain

Hospital Universitario de La Princesa

🇪🇸

Madrid, Spain

Hospital Universitario Puerta del Mar

🇪🇸

Cadiz, Spain

Saint Petersburg State Budgetary Institution of Healthcare City Hospital # 40 of Kurortnyi Region

🇷🇺

Saint Petersburg, Russian Federation

Royal Devon and Exeter Hospital

🇬🇧

Exeter, Devon, United Kingdom

Hospital Universitario Virgen del Rocio

🇪🇸

Sevilla, Spain

Re:Cognition Health

🇬🇧

London, United Kingdom

Salford Royal

🇬🇧

Salford, United Kingdom

King's College Hospital

🇬🇧

London, Manchester, United Kingdom

Barts Hospital

🇬🇧

London, Greater London, United Kingdom

State Autonomous Institution of Healthcare Republican Clinical Hospital of the Ministry of Healthcare of Republic Tatarstan

🇷🇺

Kazan, Tatarstan, Russian Federation

Gemeinschaftspraxis Dr. med. J. Springub/ W. Schwarz

🇩🇪

Westerstede, Niedersachsen, Germany

Praxis Dr. Oehlwein

🇩🇪

Gera, Thueringen, Germany

Rabin Medical Center

🇮🇱

Petah Tikva, Israel

Kaplan Medical Center

🇮🇱

Rehovot, Israel

Ziv Medical Center

🇮🇱

Safed, Israel

Tel Aviv Medical Center

🇮🇱

Tel Aviv, Israel

Colorado Springs Neurological Associates, PC

🇺🇸

Colorado Springs, Colorado, United States

Semmelweis Egyetem

🇭🇺

Budapest, Hungary

Banner Sun Health Research Institute

🇺🇸

Sun City, Arizona, United States

Stanford Neuroscience Health Center

🇺🇸

Palo Alto, California, United States

University of Colorado Health

🇺🇸

Loveland, Colorado, United States

Neurostudies, Inc

🇺🇸

Port Charlotte, Florida, United States

SFM Clinical Research

🇺🇸

Boca Raton, Florida, United States

Fixel Institute for Neurological Diseases

🇺🇸

Gainesville, Florida, United States

Rutgers New Jersey Medical School

🇺🇸

Newark, New Jersey, United States

The Ohio State University Wexner Medical Center

🇺🇸

Columbus, Ohio, United States

New York University Langone Health

🇺🇸

New York, New York, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

University of Cincinnati Medical Center

🇺🇸

Cincinnati, Ohio, United States

Concord Hospital

🇦🇺

Concord, New South Wales, Australia

Inland Northwest Research

🇺🇸

Spokane, Washington, United States

Medizinische Universitat Innsbruck Abteilung Fur Neurologie

🇦🇹

Innsbruck, Austria

Monash Health - Clinical Trials Centre

🇦🇺

Clayton, Victoria, Australia

Perron Institute for Neurological and Translational Science, QEII Medical Centre

🇦🇺

Nedlands, Australia

Wilhelminenspital Wien Abteilung fur Neurologie

🇦🇹

Wien, Austria

Universitätsklinikum Tulln

🇦🇹

Tulln, Austria

UMHAT Alexandrovska EAD Clinic of Neurological Diseases

🇧🇬

Sofia, Bulgaria

London Health Sciences Centre-CCIT

🇨🇦

London, Ontario, Canada

Montreal Neurological Institute and Hospital

🇨🇦

Montreal, Quebec, Canada

East Tallinn Central Hospital

🇪🇪

Tallinn, Estonia

Tartu University Hospital

🇪🇪

Tartu, Estonia

Chaim Sheba Medical Center

🇮🇱

Tel HaShomer, Israel

Azienda Ospedaliero Universitaria Ospedali Riuniti Ancona Umberto I - G.M. Lancisi - G. Salesi, SOD Clinica di Neuroriabilitazione

🇮🇹

Ancona, Italy

AOU San Giovanni di Dio e Ruggi d'Aragona

🇮🇹

Salerno, Italy

New Zealand Brain Research Institute

🇳🇿

Christchurch, New Zealand

ETG Warszawa

🇵🇱

Warszawa, Poland

NHI Central Clinical Hospital #2 of JSC Russian Railways N.A. Semashko

🇷🇺

Moscow, Russian Federation

Federal State Budgetary Institution Federal Sibirian Scientific and Clinical Center of Federal Medico-Biological Agency

🇷🇺

Krasnoyarsk, Russian Federation

Hospital Universitario Donostia

🇪🇸

San Sebastián, Guipuzcoa, Spain

Hospital Universitari Vall d'Hebron

🇪🇸

Barcelona, Spain

Hospital del Mar

🇪🇸

Barcelona, Spain

Communal Noncommercial Enterprise of Lviv Regional Council Lviv Regional Clinical Hospital Dept of Neurology

🇺🇦

Lviv, Ukraine

The National Hospital for Neurology & Neurosurgery

🇬🇧

London, Greater London, United Kingdom

Re:Cognition Health Ltd

🇬🇧

Birmingham, West Midlands, United Kingdom

The Royal Melbourne Hospital

🇦🇺

Parkville, Victoria, Australia

Mayo Clinic - Rochester

🇺🇸

Rochester, Minnesota, United States

Georgetown University Hospital

🇺🇸

McLean, Virginia, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

Federal State Budgetary Educational Institution of Additional Professional Education Russian Medical Academy of Continuous Postgraduate Education of the Ministry of Healthcare of the Russian Federation

🇷🇺

Moscow, Russian Federation

Charite Universitatsmedizin Berlin - Campus Benjamin Franklin

🇩🇪

Berlin, Germany

University of Calgary

🇨🇦

Calgary, Alberta, Canada

Federal State Budgetary Institution National Medical Research Centre of psychiatry and neurology named after V.M. Bekhterev of the Ministry of Healthcare of the Russian Federation

🇷🇺

Saint Petersburg, Russian Federation

Human Brain Institute RAMS

🇷🇺

Saint Petersburg, Russian Federation

Regional State Budgetary Institution of Healthcare Smolensk Regional Clinical Hospital

🇷🇺

Smolensk, Russian Federation

Communal Noncommercial Enterprise City Policlinic #9 of Kharkiv City Council

🇺🇦

Kharkiv, Ukraine

CNE Acad O.I. Yushchenko Vinnytsia Reg Psychoneurological Hospital of Vinnytsia Regional Council, Department of Neurology

🇺🇦

Vinnytsia, Ukraine

Communal Institution City Clinical Hospital #6

🇺🇦

Zaporizhzhia, Ukraine

Navarrabiomed Fundacion Miguel Servet

🇪🇸

Pamplona, Navarra, Spain

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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