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Study of BHV-3241 in Participants With Multiple System Atrophy (M-STAR Trial)

Phase 3
Waitlist Available
Research Sponsored by Biohaven Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 100 weeks
Awards & highlights
Pivotal Trial

Summary

This trial is testing a drug called verdiperstat to see if it helps people with Multiple System Atrophy, a condition that affects the nervous system. The drug aims to reduce inflammation and protect nerve cells. Researchers are comparing its effects to a non-active substance to determine its effectiveness.

Eligible Conditions
  • Multiple System Atrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 100 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 100 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline in the Modified UMSARS Score at Week 48
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Secondary study objectives
Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
+6 more

Side effects data

From 2022 Phase 2 & 3 trial • 167 Patients • NCT04436510
28%
Nausea
21%
Headache
20%
Insomnia
19%
Fall
19%
Constipation
17%
Muscular weakness
16%
Fatigue
13%
Neuromyopathy
10%
Tension headache
10%
Dizziness
10%
Decreased appetite
10%
Dysphagia
9%
Blood thyroid stimulating hormone increased
8%
Dyspnoea
8%
Diarrhoea
8%
Urinary tract infection
8%
Salivary hypersecretion
7%
Anxiety
6%
Urine odour abnormal
6%
Cough
6%
Back pain
6%
Arthralgia
6%
Oedema peripheral
3%
Pain in extremity
3%
Dysarthria
2%
Pulmonary embolism
2%
Respiratory failure
2%
Amyotrophic lateral sclerosis
2%
Febrile neutropenia
1%
Bacteraemia
1%
Abdominal pain
1%
Cerebral infarction
1%
Deep vein thrombosis
1%
B-cell lymphoma
1%
Acute respiratory failure
1%
COVID-19
1%
COVID-19 pneumonia
1%
Dehydration
1%
Sudden death
1%
Aphasia
1%
Bacterial sepsis
1%
Failure to thrive
1%
Hip fracture
1%
Ileus
1%
Hepatic enzyme abnormal
100%
80%
60%
40%
20%
0%
Study treatment Arm
Verdiperstat
Matching Placebo

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: VerdiperstatExperimental Treatment1 Intervention
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 II: PlaceboPlacebo Group1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Verdiperstat
Not yet FDA approved

Find a Location

Who is running the clinical trial?

Biohaven Pharmaceuticals, Inc.Lead Sponsor
48 Previous Clinical Trials
37,180 Total Patients Enrolled
2 Trials studying Multiple System Atrophy
19 Patients Enrolled for Multiple System Atrophy
~66 spots leftby Dec 2025