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CERE-120: Adeno-Associated Virus Delivery of Neurturin

Phase 1

Idiopathic Parkinson's Disease | Monoclonal antibody | Neurology |Sangamo Therapeutics, Inc.|Last Updated: Apr 16, 2020

Success Probability

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMC
Total Trials1
Total Enrollment57

FDA Designations

No designations recorded

Clinical trial landscape

CERE-120: Adeno-Associated Virus Delivery of Neurturin · 1 trial · 1 indication

Phase 1 1
NCT00985517Safety and Efficacy of CERE-120 in Subjects With Parkinson's DiseaseIdiopathic Parkinson's Disease
COMPLETED57 Analytics
PHASE1COMPLETED
Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease
Idiopathic Parkinson's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants Who Received CERE-120 Treatment
5 years

Number of Participants who received CERE-120 Treatment

Phase 1 and Phase 2: Change From Baseline in Motor Examination Part III Total Score in the "Off" Condition for the CERE-120 Group as Compared to the Sham Surgery Control Group at the Last Double-blind Assessment.
Baseline, Months 15, 18, 21 and 24

UPDRS is a tool to evaluate the impact of symptomatic and potential disease modifying treatments. Part III focuses on 14 motor functions, assessing each on a scale from 0 (normal) to 4 (severe) with total score 0 - 56. The 5 Feb 2003 version of the UPDRS Rating was used in this trial. The total score is the sum of 14 motor functions. Change from baseline in total score is reported. The last double blind assessment for each subject is defined as the most recent assessment at the time when the final randomized subject completes the Month 15 Visit. The length of the double-blind follow-up period for each subject ranged from 15 to 24 months, depending on the enrollment rate.

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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase 1: Cohort 1EXPERIMENTALCERE-120 9.4 x 10\^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 1: Cohort 2EXPERIMENTALCERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 2: CERE-120EXPERIMENTALCERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen
Phase 2: Sham SurgerySHAM_COMPARATORNeurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery.

Interventions

NameTypeDescription
CERE-120: Adeno-Associated Virus Delivery of NeurturinBIOLOGICAL -
Sham SurgeryPROCEDURE -
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Eligibility Criteria

Age Range35 Years to 70 Years
SexALL
Healthy VolunteersNo
Study Sites11

Inclusion Criteria: * Males and females, ages 35 to 70 years old (inclusive) * A diagnosis of idiopathic Parkinson's disease based on UK Brain Bank criteria, including bradykinesia and at least 1 of the following PD features: resting tremor or rigidity * A Hoehn and Yahr score of no greater than 3 ...

Countries:United States
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