Open Actively Recruiting

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


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.

Study Type
Expanded Access


Minimum Age
Maximum Age

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.

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Study Stats
Protocol No.
Principal Investigator
Aaron Fisher
  • UCLA Westwood
For Providers
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