Effect of Verdiperstat on Microglial Activation in Well-characterized MSA Patients
Status:
Active, not recruiting
Trial end date:
2022-06-01
Target enrollment:
Participant gender:
Summary
This study will comprise of two phases, an observational phase and a treatment phase.
In the observational phase the specific aims are: 1. To determine the presence and regional
distribution of microglial activation, as assessed by 18F-PBR06 PET, in subjects with MSA as
compared to healthy controls, at baseline and at 6-9 months' follow-up.
2. To assess the relationship between microglial activation and clinical progression at
baseline and follow-up.
In the treatment phase the specific aims of the study are:
The specific aims of the study are:
1. To assess whether verdiperstat (BHV-3241) reduces 18F-PBR06 PET signal, and thus
microglial activation and inflammation, in well-characterized MSA patients.
2. To assess the relationship between PET changes and clinical progression at baseline and
follow-up in patients treated with verdiperstat.
3. To assess the relationship between PET changes and volumetric brain MRI at baseline and
follow-up in patients treated with verdiperstat.
Currently there is no known disease modifying therapy for MSA. Recently, the drug
verdiperstat (BHV-3241) has appeared in the investigational arena specifically for the
indication of Multiple System Atrophy. Verdiperstat (BHV-3241) is currently being used in a
phase 3 active drug trial at Massachusetts Hospital. Verdiperstat (BHV-3241) is known to
target Myeloperoxidase, an enzyme implicated in neuroinflammation, a major driver in disease
pathogenesis. Our previous study (IRB protocol #2016P002373) demonstrated that applying TSPO
(translator protein) PET imaging enabled us to track changes in neuroinflammation and thus
provide a viable biomarker for disease progression.
In this pilot study, the investigators aim to assess the effect of an investigational drug,
verdiperstat (BHV-3241) on microglial activation in MSA patients using [F-18]PBR06 and to
link it with clinical and morphometric MRI brain changes following treatment.