Study of BHV-3241 in Participants With Multiple System Atrophy

NCT ID: NCT03952806

Last Updated: 2023-09-29

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

421 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-07-29

Study Completion Date

2022-06-30

Brief Summary

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

Detailed Description

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Conditions

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Multiple System Atrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
Double-blind to Sponsor, Investigator and Subject

Study Groups

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Verdiperstat

Participants received verdiperstat 300 mg tablet orally once daily for 1 week, followed by 300 mg twice daily for 1 week, and then 600 mg twice daily for the remaining 46 weeks of the double-blind phase.

Participants who completed the double-blind phase were offered the opportunity to enroll in an open-label extension (OLE) phase to continue verdiperstat 600 mg twice daily for 48 weeks.

Group Type EXPERIMENTAL

Verdiperstat

Intervention Type DRUG

300mg 2 oral tablets, twice daily

Placebo

Participants received placebo matching with verdiperstat for 48 weeks. Participants who completed the double-blind phase were offered the opportunity to enroll in an OLE phase to receive verdiperstat 600 mg tablet orally twice daily for 48 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Matching placebo

Interventions

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Verdiperstat

300mg 2 oral tablets, twice daily

Intervention Type DRUG

Placebo

Matching placebo

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

40 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Biohaven Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Barrow Neurological Institute

Phoenix, Arizona, United States

Site Status

UC San Diego Department of Neurosciences

La Jolla, California, United States

Site Status

UCLA Medical Center / Neurological Services

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

UCSF Memory and Aging Center

San Francisco, California, United States

Site Status

Rocky Mountain Movement Disorders Center

Englewood, Colorado, United States

Site Status

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Mayo Clinic Florida

Jacksonville, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

John Hopkins University

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

Lahey Hospital & Medical Center

Burlington, Massachusetts, United States

Site Status

QUEST Research Institute

Farmington Hills, Michigan, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

NYU School of Medicine, NYU Dysautonomia Center

New York, New York, United States

Site Status

Columbia University Medical Center, Neurological Institute

New York, New York, United States

Site Status

Pennsylvania State University Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

Parkinson's Disease and Movement Disorders Center at the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Kerwin Research Center

Dallas, Texas, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Swedish Medical Center

Seattle, Washington, United States

Site Status

Confraternitaet Privatklinik Josefstadt in Wien

Vienna, Vienna, Austria

Site Status

University Clinic Innsbruck

Innsbruck, , Austria

Site Status

CHU de Bordeaux, Service de Neurologie

Bordeaux, , France

Site Status

CHU - Hospital de la Timone

Marseille, , France

Site Status

Unité d'investigation clinique de Neurologie Rez-de-jardin, Bloc Hopital CHU Pontchaillou

Rennes, , France

Site Status

Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes

Strasbourg, , France

Site Status

CHU Purpan

Toulouse, , France

Site Status

University Hospital of Liepzig

Leipzig, Saxony, Germany

Site Status

St. Josef - Hospital Bochum, Kardiologische Studienambulanz

Bochum, , Germany

Site Status

Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)

Bonn, , Germany

Site Status

University Hospital Duesseldorf

Düsseldorf, , Germany

Site Status

CRC Core Facility Medizinische Hochschule Hannover (MHH)

Hanover, , Germany

Site Status

Paracelsus-Elena-Klinik

Kassel, , Germany

Site Status

Klinik für Neurologie - UKSH - Campus Kiel

Kiel, , Germany

Site Status

Universitaetsklinikum Giessen und Marburg GmbH - Parkinson-Studienzentrum, Klinik für Neurologie

Marburg, , Germany

Site Status

Universitaetsklinikum Muenster

Münster, , Germany

Site Status

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, Milan, Italy

Site Status

A.O.U. San Giovanni di Dio e Ruggi d'Aragona

Salerno, , Italy

Site Status

The Newcastle upon Tyne Hospitals NHS Foundation Trust - Campus for Ageing and Vitality (NGH)

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Austria France Germany Italy United Kingdom

References

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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.

Reference Type DERIVED
PMID: 34115419 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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BHV3241-301

Identifier Type: -

Identifier Source: org_study_id

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