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CERE-120

Phase 2

Idiopathic Parkinson's Disease | Gene Therapies | Neurology |Sangamo Therapeutics, Inc.|Trials Updated: Nov 10, 2022

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMC
Total Trials1
Total Enrollment58

FDA Designations

No designations recorded

CERE-120 clinical trials

CERE-120 · 1 trial · 1 indication

Phase 2 1
NCT00400634Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's DiseaseIdiopathic Parkinson's Disease
COMPLETED58 Analytics
PHASE2COMPLETED
Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease
Idiopathic Parkinson's DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

UPDRS Part III OFF
Change from Baseline to 12 Month Visit

The UPDRS (Unified Parkinson's Disease Rating Scale) is a clinical rating scale that assesses the symptomatic burden of Parkinson's Disease. The scale has four main sections, and each item is scored from a 0 to a 4 (higher number is more severe manifestation). Part III is a subsection devoted to motor function, has 14 questions, resulting in a score range of 0 (unaffected) to 56 (severely affected). The scale is administered by a trained clinician, and patients were assessed in a practically defined "off" condition, 12 hours or more after the last administration of medication.

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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
1EXPERIMENTALIntracerebral administration of CERE-120
2SHAM_COMPARATORSham Neurosurgery

Interventions

NameTypeDescription
CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])DRUGCERE-120 5.4 x 10\^11 vg
Sham SurgeryPROCEDUREBilateral partial thickness burr holes placed, no intraparenchymal injections
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Eligibility Criteria

Age Range35 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites9

Inclusion Criteria: * Diagnosis of bilateral, idiopathic Parkinson's Disease (PD) based on UK Brain Bank criteria with motor complications despite adequate oral antiparkinsonian therapy. * At least 5 years disease duration, relative to the anticipated date of surgery, since diagnosis of PD. * Males...

Countries:United States
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Frequently asked questions about CERE-120

What is CERE-120?

CERE-120 is an investigational gene therapy developed for idiopathic Parkinson's disease. It is also known as AAV2-neurturin, or AAV2-NTN, reflecting its adeno-associated virus serotype 2 delivery of the neurturin gene. It has been studied in a completed Phase 2 clinical trial in the United States.

What is CERE-120 used for in Parkinson's disease?

CERE-120 was developed for idiopathic Parkinson's disease, the most common form of Parkinson's. It is a gene therapy designed to deliver the neurturin gene to the brain. Its clinical development has focused on this single neurology indication, and it is not approved for any use.

Who is developing CERE-120?

CERE-120 was developed by Sangamo Therapeutics, Inc., which trades under the ticker SGMOQ. The company sponsored the clinical program evaluating the gene therapy in idiopathic Parkinson's disease.

What phase is CERE-120 in?

CERE-120 reached Phase 2 clinical development. Its Phase 2 trial has been completed, and no trials are currently active. CERE-120 is investigational and has not been approved by the FDA for idiopathic Parkinson's disease or any other indication.

What clinical trials is CERE-120 in?

CERE-120 has been studied in one clinical trial, NCT00400634, a Phase 2 double-blind, multicenter, sham surgery controlled study in subjects with idiopathic Parkinson's disease. The trial enrolled 58 participants in the United States, included both sexes, and required a minimum age of 35 years. It is completed.

Is CERE-120 the same as AAV2-neurturin?

Yes. CERE-120 is also known as AAV2-neurturin, or AAV2-NTN, which stands for adeno-associated virus serotype 2 delivering the neurturin gene. Searches for either name refer to the same investigational gene therapy for idiopathic Parkinson's disease developed by Sangamo Therapeutics.