Recent Updates
Recently added Catalysts

AAV-hAADC-2

Phase 1

Parkinson's Disease | Gene therapy | Neurology |Sanofi|Last Updated: Dec 4, 2013

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment10
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00229736A Study of AAV-hAADC-2 in Subjects With Parkinson's DiseasePHASE1 COMPLETED 10Nov 1, 2004Mar 1, 2013Dec 4, 20131 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
The safety and tolerability of intrastriatal administration of AAV-hAADC-2 as measured by Adverse Events in subjects with mid- to late-stage Parkinson's Disease.
Time of treatment through Month 60.
Secondary Endpoints
The effect of AAV-hAADC-2 on clinical status as recorded in subject diaries, by clinical assessment, and daily levodopa requirement.
Time of treatment through Month 60.
The relationship between the dose of AAV-hAADC-2 vector infused and the resulting level of striatal AADC expression by FMT-PET imaging.
Time of treatment through Month 60
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AAV-hAADC-2 (9x10^10 vector genomes)EXPERIMENTAL -
AAV-hAADC-2 (3x10^11 vector genomes)EXPERIMENTAL -
Interventions
NameTypeDescription
AAV-hAADC-2GENETIC9 x 10\^10 vector genomes (vg) of AAV-hAADC-2 in a single dose of 200 µL bilaterally infused over 4 striatal targets
Unlock Study Design Details
Eligibility Criteria
Age Range40 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Documented informed consent. * Diagnosis of idiopathic Parkinson's disease (two of four cardinal features - tremor, bradykinesia, rigidity and postural instability - plus early dopaminergic responsiveness of symptoms and absence of any cardinal signs of more widespread neurolo...

Countries:United States
Unlock Eligibility Criteria