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AZD3241

Phase 2

Multiple System Atrophy, MSA | Small molecule | Other |AstraZeneca PLC|Last Updated: Sep 25, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment59
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02388295AZD3241 PET MSA Trial, Phase 2, Randomized,12 Week Safety and Tolerability Trial With PET in MSA PatientsPHASE2 COMPLETED 59Apr 27, 2015Sep 19, 2016Sep 25, 201717 United States, Austria +5
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Study Endpoints
Primary Endpoints
Striatum Brain Region: Change From Baseline in Microglia Activation Via Positron Emission Tomography(PET)
Baseline (pre randomization) and Week 12

Striatum Brain region: Change from baseline in microglia activation via PET By \[11C\]PBR28 binding to translocator protein

Secondary Endpoints
Myeloperoxidase (MPO) Inhibition in Plasma (Change From Baseline), Specific Activity
Baseline (Day -1) and week 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AZD3241EXPERIMENTALSubjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.
Placebo to match AZD3241PLACEBO_COMPARATORSubjects will be randomized to one of the two doses of AZD3241 or placebo in a 1:1:1 ratio.
Interventions
NameTypeDescription
AZD3241DRUGDrug: AZD3241 administered for 12 weeks orally as a tablet.
PlaceboDRUGPlacebo to match AZD3241 administered for 12 weeks orally as a tablet.
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Eligibility Criteria
Age Range30 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: 1. Male or female, age 30-80 years, inclusive, at screen. 2. Meet criteria for diagnosis of probable or possible MSA according to the consensus criteria (Gilman et al. 2008 ). 3. "High-affinity binder" or "mixed-affinity binder" for TSPO, as confirmed by prospective genotyping o...

Countries:United StatesAustriaFinlandFranceItalySwedenUnited Kingdom
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