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

Biohaven

Status and phase: Completed; Phase 3

Conditions: Multiple System Atrophy

Treatments: Drug: Verdiperstat; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03952806

BHV3241-301

Details and patient eligibility

About

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

Enrollment: 421 patients

Sex: All

Ages: 40 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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421 participants in 2 patient groups, including a placebo group

Verdiperstat

Experimental group

Description:

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.

Treatment:

Drug: Verdiperstat

Placebo

Placebo Comparator group

Description:

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:

Drug: Placebo