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Phase 3 N=336 Randomized Triple-blind Treatment

Study of BHV-3241 in Participants With Multiple System Atrophy

Multiple System Atrophy

Enrolled (actual)
336
Serious AEs
21.7%
Results posted
Sep 2023
Primary outcome: Primary: Change From Baseline in the Modified UMSARS Score at Week 48 — 5.20; 4.85 units on a scale — p=0.4656

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Verdiperstat (Drug); Placebo (Drug)
Age
Adult, Older Adult · 40+ yrs
Sex
All
Sponsor
Biohaven Pharmaceuticals, Inc.
Primary completion
Jul 2021

Outcome Measures

OutcomeResultp-value
PRIMARY
Change From Baseline in the Modified UMSARS Score at Week 48
5.20; 4.85 0.4656
PRIMARY
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
160; 156; 95; 112; 44; 29
SECONDARY
Clinical Global Impression of Improvement (CGI-I) Score at Week 48
5.07; 5.14
SECONDARY
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48
13.83; 13.45
SECONDARY
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48
7.52; 5.56
SECONDARY
Change From Baseline in UMSARS Part I and Part II Total Score at Week 48
12.00; 11.34
SECONDARY
Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48
0.33; 0.27
SECONDARY
Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48
0.79; 0.78
SECONDARY
Change From Baseline in UMSARS Part III at Week 48 (Blood Pressure (BP) Only)
-2.18; -3.09; -2.87; -2.54
SECONDARY
Change From Baseline in UMSARS Part III at Week 48 (Heart Rate (HR) Only)
0.20; -0.68
SECONDARY
Change From Baseline in UMSARS Part IV at Week 48
0.82; 0.85

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

  • 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.

Exclusion Criteria

  • 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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03952806). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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