Multiple System AtrophyPhase 3M-STARNCT03952806
Study of BHV-3241 in Participants With Multiple System Atrophy
A Phase 3 study testing Verdiperstat in people with Multiple System Atrophy, run by Biohaven Pharmaceuticals, Inc. at 48 sites in 6 countries.
Trial summary
The purpose of this study is to compare the efficacy of verdiperstat (BHV-3241) versus placebo in participants with Multiple System Atrophy Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Multiple System Atrophy
- What is being tested?
- Verdiperstat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 421 participants
- Where
- 48 sitesin 6 countries
- Study dates
- 29 Jul 2019to 30 Jun 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
48 sites in 6 countries.
48 sites in 6 countries
United States29 sites
Albany Medical College
Albany, New York, 12208
Emory University
Atlanta, Georgia, 30329
John Hopkins University
Baltimore, Maryland, 21287
Parkinson's Disease and Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486
Massachusetts General Hospital
Boston, Massachusetts, 02114
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Lahey Hospital & Medical Center
Burlington, Massachusetts, 01805
Rush University Medical Center
Chicago, Illinois, 60612
University of Chicago
Chicago, Illinois, 60637
Kerwin Research Center
Dallas, Texas, 75231
UT Southwestern Medical Center
Dallas, Texas, 75390
Rocky Mountain Movement Disorders Center
Englewood, Colorado, 80113
QUEST Research Institute
Farmington Hills, Michigan, 48334
University of Florida
Gainesville, Florida, 32611
Pennsylvania State University Hershey Medical Center
Hershey, Pennsylvania, 17033
Mayo Clinic Florida
Jacksonville, Florida, 32224
University of Kansas Medical Center
Kansas City, Kansas, 66160
UC San Diego Department of Neurosciences
La Jolla, California, 92037
UCLA Medical Center / Neurological Services
Los Angeles, California, 90095
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
NYU School of Medicine, NYU Dysautonomia Center
New York, New York, 10016
Columbia University Medical Center, Neurological Institute
New York, New York, 10032
Stanford University
Palo Alto, California, 84127
Parkinson's Disease and Movement Disorders Center at the University of Pennsylvania
Philadelphia, Pennsylvania, 19107
Barrow Neurological Institute
Phoenix, Arizona, 80013
Mayo Clinic
Rochester, Minnesota, 55905
UCSF Memory and Aging Center
San Francisco, California, 95158
Swedish Medical Center
Seattle, Washington, 98122
University of South Florida
Tampa, Florida, 33612
Germany9 sites
St. Josef - Hospital Bochum, Kardiologische Studienambulanz
Bochum, 44791
Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE)
Bonn, 53127
University Hospital Duesseldorf
Düsseldorf, 40225
CRC Core Facility Medizinische Hochschule Hannover (MHH)
Hanover, 30625
Paracelsus-Elena-Klinik
Kassel, 34128
Klinik für Neurologie - UKSH - Campus Kiel
Kiel, 24105
University Hospital of Liepzig
Leipzig, Saxony, 04103
Universitaetsklinikum Giessen und Marburg GmbH - Parkinson-Studienzentrum, Klinik für Neurologie
Marburg, 35043
Universitaetsklinikum Muenster
Münster, 48149
France5 sites
CHU de Bordeaux, Service de Neurologie
Bordeaux, 33076
CHU - Hospital de la Timone
Marseille, 13385
Unité d'investigation clinique de Neurologie Rez-de-jardin, Bloc Hopital CHU Pontchaillou
Rennes, 35033
Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes
Strasbourg, 67098
CHU Purpan
Toulouse, 31059
Austria2 sites
University Clinic Innsbruck
Innsbruck, 06020
Confraternitaet Privatklinik Josefstadt in Wien
Vienna, State of Vienna, 1080
Italy2 sites
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano
Milan, Milan, 20122
A.O.U. San Giovanni di Dio e Ruggi d'Aragona
Salerno, 84100
United Kingdom1 site
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Campus for Ageing and Vitality (NGH)
Newcastle upon Tyne, NE4 5PL
About this study
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)
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
- Being tested
- Verdiperstat, Placebo
- Keywords
- Multiple System Atrophy (MSA)
- Medical subject headings
- Multiple System Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VerdiperstatDrug
300mg 2 oral tablets, twice daily
PlaceboDrug
Matching placebo
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 100 weeks
Secondary outcomes (9)
Clinical Global Impression of Improvement (CGI-I) Score at Week 48
The CGI-I is a clinician-rated scale measuring the change in the participant's clinical status from a specific point in time. It is scored on a 7- point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change. Higher scores indicate greater impairment.
Measured: Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
The MSA-QoL is a participant-rated scale that was designed to measure health-related quality of life specifically in MSA. It assesses activities of daily living and has subscales for motor, nonmotor, and emotional/social domains. The MSA-QoL motor subscale includes 14 items. The response to each question ranges from 0= no problem, to 4= extreme problem, with a total scale ranging from 0-56. Higher scores indicates higher impact of the disease on the aspect measured by each subscale.
Measured: Baseline and Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
The MSA-QoL is a participant-rated scale that was designed to measure health-related quality of life specifically in MSA. It assesses activities of daily living and has subscales for motor, nonmotor, and emotional/social domains. The MSA-QoL nonmotor subscale includes 12 items. The response to each question ranges from 0= no problem, to 4= extreme problem, with a total scale ranging from 0-48. Higher scores indicates higher impact of the disease on the aspect measured by each subscale.
Measured: Baseline and Week 48
Change From Baseline in UMSARS Part I and Part II Total Score at Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review with 12 questions), Part II (Motor Examination with 14 questions), Part III (Autonomic Examination), and Part IV (Global Disability Scale). UMSARS Part I and Part II responses are measured on a 5-point scale ranging from 0 to 4, where 0= no impairment, 1= mild impairment, 2= moderate impairment, 3= severe impairment, 4=complete impairment. Each subscale score is a sum of its items and total score is sum of all 26 items, score range from 0 to 104. Higher scores indicates greater impairment.
Measured: Baseline and Week 48
Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48
The PGI-S is a participant-rated scale that measures how participants perceive the severity of their illness. The PGI-S is a 1-item questionnaire on a 4-point scale ranging from 1 to 4, where, 1 = normal, 2 = mild, 3 = moderate, 4 = severe. Higher scores indicate greater impairment.
Measured: Baseline and Week 48
Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48
The CGI-S is a clinician-rated scale measuring the severity of the participant's illness. It is scored on a 7- point scale ranging from 1 (normal, not ill at all) to 7 (among the most extremely ill patients). Higher scores indicate greater impairment.
Measured: Baseline and Week 48
Change From Baseline in UMSARS Part III at Week 48 (Blood Pressure (BP) Only)
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part III of UMSARS was analyzed. Part III of the UMSARS is an Autonomic Exam, consisting of Supine and Standing Systolic and Diastolic Blood Pressure, Heart rate and presence Orthostatic Symptoms. Only change in Orthostatic Blood Pressure reported.
Measured: Baseline and Week 48
Change From Baseline in UMSARS Part III at Week 48 (Heart Rate (HR) Only)
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part III of UMSARS was analyzed. Part III of the UMSARS is an Autonomic Exam, consisting of Supine and Standing Systolic and Diastolic Blood Pressure, Heart rate and presence Orthostatic Symptoms. Only change in Orthostatic Heart Rate reported.
Measured: Baseline and Week 48
Change From Baseline in UMSARS Part IV at Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part IV of UMSARS was analyzed. Part IV was measured on a 5-point scale ranging from 1= Completely independent. Able to do all chores with minimal difficulty or impairment. Essentially normal. Unaware of any difficulty, to 5= Totally dependent and helpless. Bedridden. Higher scores indicate greater impairment.
Measured: Baseline and Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2019
- Study start29 Jul 2019
- Primary completion29 Jul 2021
- Study completion30 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple System AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 421 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03952806
- Sponsor's protocol ID
- BHV3241-301
- Acronym
- M-STAR
- Status last verified
- Sep 2023
- First submitted
- 15 May 2019
- Last updated
- 29 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 34115419
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03952806
- Acronym / sponsor ID
- M-STAR / BHV3241-301
- Conditions
- Multiple System Atrophy
- Keywords
- Multiple System Atrophy (MSA)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 421 participants (actual)
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 29 Jul 2019 (actual)
- Primary completion
- 29 Jul 2021 (actual)
- Study completion
- 30 Jun 2022 (actual)
- First posted
- 16 May 2019 (actual)
- Results
- Posted 29 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to compare the efficacy of verdiperstat (BHV-3241) versus placebo in participants with Multiple System Atrophy
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Verdiperstat | Experimental | Drug: VerdiperstatParticipants 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. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants 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. |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Verdiperstat300mg 2 oral tablets, twice daily | Drug | Verdiperstat |
| PlaceboMatching placebo | Drug | Placebo |
Primary outcome
Change From Baseline in the Modified UMSARS Score at Week 48
Time frame: Baseline and 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.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Up to 100 weeks
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.
9 secondary outcomes
Clinical Global Impression of Improvement (CGI-I) Score at Week 48
Time frame: Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
Time frame: Baseline and Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
Time frame: Baseline and Week 48
Change From Baseline in UMSARS Part I and Part II Total Score at Week 48
Time frame: Baseline and Week 48
Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48
Time frame: Baseline and Week 48
Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48
Time frame: Baseline and Week 48
Change From Baseline in UMSARS Part III at Week 48 (Blood Pressure (BP) Only)
Time frame: Baseline and Week 48
Change From Baseline in UMSARS Part III at Week 48 (Heart Rate (HR) Only)
Time frame: Baseline and Week 48
Change From Baseline in UMSARS Part IV at Week 48
Time frame: Baseline and Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34115419Schmahmann 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
48 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Verdiperstat in people with Multiple System Atrophy, run by Biohaven Pharmaceuticals, Inc. at 48 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Germany, France, Austria, Italy and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in the Modified UMSARS Score at Week 48 (time frame: Baseline and Week 48). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03952806, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2023.