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CERE-110: Adeno-Associated Virus Surgery

Phase 2

Alzheimer's Disease | Small molecule | Neurology |Sangamo Therapeutics, Inc.|Last Updated: Dec 21, 2020

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-BlindSHAM_CONTROLLEDDMC
Total Trials1
Total Enrollment49
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00876863Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's DiseasePHASE2 COMPLETED 49Nov 1, 2008Aug 13, 2015Dec 21, 202010 United States
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Study Endpoints
Primary Endpoints
Alzheimer's Disease Assessment Scale - Cognitive Subscale (ADAS-Cog)
24 Months
Secondary Endpoints
Neuropsychological Test Battery
24 Months
Mini-Mental State Examination (MMSE)
24 Months
Neuropsychiatric Inventory (NPI)
24 Months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
CERE-110EXPERIMENTALCERE-110: Adeno-Associated Virus Delivery of NGF
PlaceboSHAM_COMPARATORPlacebo Surgery
Interventions
NameTypeDescription
CERE-110: Adeno-Associated Virus injection SurgeryDRUGInjecting CERE-110 2.0 X 10\^11 vg into brain during surgical procedure.
Placebo SurgeryPROCEDUREPlacebo Surgery
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Eligibility Criteria
Age Range55 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Diagnosis of mild to moderate Alzheimer's disease * Approved medications for Alzheimer's disease may be taken if the dose has been stable for 3 months * A study partner who can attend all study visits * Good general health * Medically able to undergo neurosurgery Exclusion Cr...

Countries:United States
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