Clinical trial NCT03952806: Study of BHV-3241 in Participants With Multiple System Atrophy
Official title: Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of BHV-3241 in Subjects With Multiple System Atrophy (M-STAR Study)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Multiple System Atrophy
Keywords: Multiple System Atrophy (MSA)
Lead sponsor: Biohaven Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-05-16
Enrollment: 421
Sex: ALL
Minimum age (years): 40
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to compare the efficacy of verdiperstat (BHV-3241) versus placebo in participants with Multiple System Atrophy


Eligibility criteria: Inclusion Criteria:

1. Diagnosis of probable or possible MSA according to consensus clinical criteria (Gilman et al 2008), including participants with MSA of either subtype (MSA-P or MSA-C).
2. Able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
3. Anticipated survival of at least 3 years at the time of Screening, as judged by the Investigator.

Exclusion Criteria:

1. Any condition that would interfere with the participant's ability to comply with study instructions, place the participant at unacceptable risk, and/or confound the interpretation of safety or efficacy data from the study, as judged by the Investigator.
2. Diagnosis of neurological disorders, other than MSA.

Primary outcomes:
measure: Change From Baseline in the Modified UMSARS Score at Week 48
timeFrame: Baseline and Week 48
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
timeFrame: Up to 100 weeks
Secondary outcomes:
measure: Clinical Global Impression of Improvement (CGI-I) Score at Week 48
timeFrame: Week 48
measure: Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
timeFrame: Baseline and Week 48
measure: Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
timeFrame: Baseline and Week 48
measure: Change From Baseline in UMSARS Part I and Part II Total Score at Week 48
timeFrame: Baseline and Week 48
measure: Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48
timeFrame: Baseline and Week 48
measure: Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48
timeFrame: Baseline and Week 48
measure: Change From Baseline in UMSARS Part III at Week 48 (Blood Pressure (BP) Only)
timeFrame: Baseline and Week 48
measure: Change From Baseline in UMSARS Part III at Week 48 (Heart Rate (HR) Only)
timeFrame: Baseline and Week 48
measure: Change From Baseline in UMSARS Part IV at Week 48
timeFrame: Baseline and Week 48
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: La Jolla
country: United States
facility: UC San Diego Department of Neurosciences
state: California
city: Los Angeles
country: United States
facility: UCLA Medical Center / Neurological Services
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Francisco
country: United States
facility: UCSF Memory and Aging Center
state: California
city: Englewood
country: United States
facility: Rocky Mountain Movement Disorders Center
state: Colorado
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton
state: Florida
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Chicago
country: United States
facility: University of Chicago
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: John Hopkins University
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Burlington
country: United States
facility: Lahey Hospital & Medical Center
state: Massachusetts
city: Farmington Hills
country: United States
facility: QUEST Research Institute
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: New York
country: United States
facility: NYU School of Medicine, NYU Dysautonomia Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center, Neurological Institute
state: New York
city: Hershey
country: United States
facility: Pennsylvania State University Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Parkinson's Disease and Movement Disorders Center at the University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Kerwin Research Center
state: Texas
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Seattle
country: United States
facility: Swedish Medical Center
state: Washington
city: Wien
country: Austria
facility: Confraternitaet Privatklinik Josefstadt in Wien
state: Vienna
city: Innsbruck
country: Austria
facility: University Clinic Innsbruck
city: Bordeaux
country: France
facility: CHU de Bordeaux, Service de Neurologie
city: Marseille Cedex 5
country: France
facility: CHU - Hospital de la Timone
city: Rennes
country: France
facility: Unité d'investigation clinique de Neurologie Rez-de-jardin, Bloc Hopital CHU Pontchaillou
city: Strasbourg
country: France
facility: Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes
city: Toulouse cedex 9
country: France
facility: CHU Purpan
city: Leipzig
country: Germany
facility: University Hospital of Liepzig
state: Sachsen
city: Bochum
country: Germany
facility: St. Josef - Hospital Bochum, Kardiologische Studienambulanz
city: Bonn
country: Germany
facility: Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)
city: Dusseldorf
country: Germany
facility: University Hospital Duesseldorf
city: Hannover
country: Germany
facility: CRC Core Facility Medizinische Hochschule Hannover (MHH)
city: Kassel
country: Germany
facility: Paracelsus-Elena-Klinik
city: Kiel
country: Germany
facility: Klinik für Neurologie - UKSH - Campus Kiel
city: Marburg
country: Germany
facility: Universitaetsklinikum Giessen und Marburg GmbH - Parkinson-Studienzentrum, Klinik für Neurologie
city: Muenster
country: Germany
facility: Universitaetsklinikum Muenster
city: Milano
country: Italy
facility: Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
state: Milan
city: Salerno
country: Italy
facility: A.O.U. San Giovanni di Dio e Ruggi d'Aragona
city: Newcastle Upon Tyne
country: United Kingdom
facility: The Newcastle upon Tyne Hospitals NHS Foundation Trust - Campus for Ageing and Vitality (NGH)
Study officials:
References:
citation: Schmahmann JD, Pierce S, MacMore J, L'Italien GJ. Development and Validation of a Patient-Reported Outcome Measure of Ataxia. Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11.