Study of BHV-3241 in Participants With Multiple System Atrophy

NCT03952806 | Completed Phase 3 Results FDA Drug

• 1 other identifier: BHV3241-301
• Study Type: interventional
• Target: 421
• Locations: 6 countries
• Sites: 48

Brief Summary

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

Conditions

Multiple System Atrophy

Keywords

Multiple System Atrophy (MSA)

Outcome Measures

Primary Outcomes (2)

• Change From Baseline in the Modified UMSARS Score at Week 48

  UMSARS - clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination), Part IV (Global Disability Scale). Modified UMSARS is composed of subset of 9 items from original UMSARS Part I and Part II. Responses are measured on 4-point scale ranged from 0-3, where 0= no/mild impairment, 1= moderate impairment, 2= severe impairment, 3=complete impairment. Total modified UMSARS score is sum of these 9 items, score range from 0 to 27. Higher scores indicate greater impairment.

  Baseline and Week 48

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs

  An adverse event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in participants or clinical investigation participants administered an investigational (medicinal) product and that does not necessarily have a causal relationship with this treatment. A serious AE (SAE) is defined as any event that met any of the following criteria at any dose: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a participant who received study drug; other important medical events that may not have resulted in death, be life-threatening, or required hospitalization, or, based upon appropriate medical judgment, they may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the other serious outcomes.

  Up to 100 weeks

Secondary Outcomes (9)

• Clinical Global Impression of Improvement (CGI-I) Score at Week 48

  Week 48

• Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48

  Baseline and Week 48

• Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48

  Baseline and Week 48

• Change From Baseline in UMSARS Part I and Part II Total Score at Week 48

  Baseline and Week 48

• Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48

  Baseline and Week 48

Study Arms (2)

Verdiperstat

EXPERIMENTAL

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.

Drug: Verdiperstat

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Verdiperstat DRUG

300mg 2 oral tablets, twice daily

Verdiperstat

Placebo DRUG

Matching placebo

Placebo

Eligibility Criteria

• Age: 40 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).
• 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.
• Anticipated survival of at least 3 years at the time of Screening, as judged by the Investigator.

You may not qualify if:

• 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.
• Diagnosis of neurological disorders, other than MSA.

Sponsors & Collaborators

• Biohaven Pharmaceuticals, Inc.: lead

Study Sites (48)

Barrow Neurological Institute

Phoenix, Arizona, 80013, United States

Location

UC San Diego Department of Neurosciences

La Jolla, California, 92037, United States

Location

UCLA Medical Center / Neurological Services

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 84127, United States

Location

UCSF Memory and Aging Center

San Francisco, California, 95158, United States

Location

Rocky Mountain Movement Disorders Center

Englewood, Colorado, 80113, United States

Location

Parkinson's Disease and Movement Disorders Center of Boca Raton

Boca Raton, Florida, 33486, United States

Location

University of Florida

Gainesville, Florida, 32611, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Emory University

Atlanta, Georgia, 30329, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

John Hopkins University

Baltimore, Maryland, 21287, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Lahey Hospital & Medical Center

Burlington, Massachusetts, 01805, United States

Location

QUEST Research Institute

Farmington Hills, Michigan, 48334, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Albany Medical College

Albany, New York, 12208, United States

Location

NYU School of Medicine, NYU Dysautonomia Center

New York, New York, 10016, United States

Location

Columbia University Medical Center, Neurological Institute

New York, New York, 10032, United States

Location

Pennsylvania State University Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

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

Philadelphia, Pennsylvania, 19107, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Kerwin Research Center

Dallas, Texas, 75231, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Swedish Medical Center

Seattle, Washington, 98122, United States

Location

Confraternitaet Privatklinik Josefstadt in Wien

Vienna, Vienna, 1080, Austria

Location

University Clinic Innsbruck

Innsbruck, 06020, Austria

Location

CHU de Bordeaux, Service de Neurologie

Bordeaux, 33076, France

Location

CHU - Hospital de la Timone

Marseille, 13385, France

Location

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

Rennes, 35033, France

Location

Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes

Strasbourg, 67098, France

Location

CHU Purpan

Toulouse, 31059, France

Location

University Hospital of Liepzig

Leipzig, Saxony, 04103, Germany

Location

St. Josef - Hospital Bochum, Kardiologische Studienambulanz

Bochum, 44791, Germany

Location

Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)

Bonn, 53127, Germany

Location

University Hospital Duesseldorf

Düsseldorf, 40225, Germany

Location

CRC Core Facility Medizinische Hochschule Hannover (MHH)

Hanover, 30625, Germany

Location

Paracelsus-Elena-Klinik

Kassel, 34128, Germany

Location

Klinik für Neurologie - UKSH - Campus Kiel

Kiel, 24105, Germany

Location

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

Marburg, 35043, Germany

Location

Universitaetsklinikum Muenster

Münster, 48149, Germany

Location

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano

Milan, Milan, 20122, Italy

Location

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

Salerno, 84100, Italy

Location

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

Newcastle upon Tyne, NE4 5PL, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 34115419 DERIVED

MeSH Terms

Conditions

Multiple System Atrophy

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Results Point of Contact

• Title: Chief Medical Officer
• Organization: Biohaven Pharmaceuticals, Inc

clinicaltrials@biohavenpharma.com

+1 203-404-0410

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double-blind to Sponsor, Investigator and Subject
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2019
• First Posted: May 16, 2019
• Study Start: July 29, 2019
• Primary Completion: July 29, 2021
• Study Completion: June 30, 2022
• Last Updated: September 29, 2023
• Results First Posted: September 29, 2023

Record last verified: 2023-09

Locations

IT (2); GB (1); DE (9); AU (2); FR (5); US (29)