Expanded Access Protocol of Verdiperstat in Patients With Multiple System Atrophy (MSA)

NCT05086094 | Unknown

• 1 other identifier: BHV3241-401
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA). Expanded access allows patients with a serious or a life-threatening disease or condition access to an investigational drug when no satisfactory approved treatment options are available.

Conditions

Multiple System Atrophy (MSA)

Keywords

Multiple System Atrophy; MSA; verdiperstat; BHV3241; MPO inhibitor; Myeloperoxidase inhibitor; MSA-P; MSA-C

Interventions

BHV3241, verdiperstat DRUG

BHV3241/Verdiperstat 600mg BID

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• with a diagnosis of probable or possible MSA documented by a physician according to consensus clinical criteria,5 including patients with MSA of either subtype, MSA-P or MSA-C.
• able to swallow whole tablets without crushing or chewing.
• with established care with a physician at the specialized MSA center involved in the protocol and will maintain this clinical care throughout the duration of the EAP.
• not eligible for an ongoing clinical study with verdiperstat and/or an ongoing EAP for MSA

You may not qualify if:

• Based on the Physician's judgement, any condition (including history of a clinically significant or unstable medical condition or lab abnormality) that would interfere with the patient's ability to comply with the EAP instructions, visit schedule, requirements and/or procedures, place the patient at unacceptable risk, or confound/interfere with the adequate assessment/interpretation of data; including difficulty swallowing verdiperstat tablets or any other sound medical, psychiatric and/or social reason.
• Presence of clinically significant thyroid disease despite treatment and/or TSH \>10 mIU/ L at Screening/Baseline, confirmed by repeat.
• Patient is known to have acute or chronic liver disease that is clinically significant in the Prescriber's judgment
• First or second dose of COVID-19 vaccination within 7 days of first dose of verdiperstat.

Sponsors & Collaborators

• Biohaven Pharmaceuticals, Inc.: lead

Study Sites (1)

PPD

Morrisville, North Carolina, 27560, United States

Location

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

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 8, 2021
• First Posted: October 20, 2021
• Last Updated: October 6, 2022

Record last verified: 2021-10

Locations

US (1)