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OZ1

Phase 2

HIV-1 | Gene therapy | Infectious Disease |Johnson & Johnson|Last Updated: Jun 20, 2016

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 Enrollment1
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00074997An Efficacy and Safety Study of Autologous Cluster of Differentiation 34 (CD34+) Hematopoietic Progenitor Cells Transduced With Placebo or an Anti- Human Immunodeficiency Virus Type 1 (HIV-1) Ribozyme (OZ1) in Participants With HIV-1 InfectionPHASE2 COMPLETED 1Dec 1, 2002Jan 1, 2008Jun 20, 20161 Australia
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Study Endpoints
Primary Endpoints
Difference in viral load between the placebo and OZ1 groups.
Week 47
Secondary Endpoints
CD4+ cell count
Weeks 41 - 48
HIV proviral DNA
Weeks 41 - 48
Thymic function
Weeks 41 - 48
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
001EXPERIMENTALOZ1 Single intravenous infusion of 2-20 x 10 to the power of 7 OZ1 transduced autologous CD34+ cells per kilogram of body weight
002PLACEBO_COMPARATORPlacebo Single intravenous infusion of placebo transduced autologous CD34+ cells per kilogram of body weight
Interventions
NameTypeDescription
PlaceboOTHERSingle intravenous infusion of placebo.
OZ1GENETICSingle intravenous infusion of OZ1.
CD34+ cellsGENETICAutologous CD34+ cells.
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Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * An Human Immunodeficiency Virus 1 (HIV-1) infection for at least 6 months documented by positive HIV serology including confirmation by Western Blot * Receiving either the first or second regimen of antiretroviral therapy (ART) defined as 3 or more antiretroviral drugs in comb...

Countries:Australia
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