Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as AAV2-Neurturin
CERE-120: Adeno-Associated Virus Delivery of Neurturin · 1 trial · 1 indication
Number of Participants who received CERE-120 Treatment
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.
| Arm | Type | Description |
|---|---|---|
| Phase 1: Cohort 1 | EXPERIMENTAL | CERE-120 9.4 x 10\^11 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 1: Cohort 2 | EXPERIMENTAL | CERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 2: CERE-120 | EXPERIMENTAL | CERE-120 2.4 x 10\^12 vg, injected by a neurosurgeon directly in the substantia nigra and in the putamen |
| Phase 2: Sham Surgery | SHAM_COMPARATOR | Neurosurgical procedure that mimics the procedure for CERE-120 delivery. No injections were administered during sham surgery. |
| Name | Type | Description |
|---|---|---|
| CERE-120: Adeno-Associated Virus Delivery of Neurturin | BIOLOGICAL | - |
| Sham Surgery | PROCEDURE | - |
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 ...
CERE-120 is an investigational gene therapy being developed for idiopathic Parkinson's disease. It was studied in a Phase 1/2 clinical trial in the United States that enrolled 57 participants aged 35 years and older. It is not an approved treatment.
CERE-120 is being developed by Sangamo Therapeutics, Inc., which trades under the ticker SGMOQ. The company is the sponsor associated with the CERE-120 gene therapy program for idiopathic Parkinson's disease.
CERE-120 has been studied in a Phase 1/2 clinical trial for idiopathic Parkinson's disease. That trial, NCT00985517, is completed, and no CERE-120 trials are currently active. CERE-120 remains an investigational gene therapy rather than an approved product.
CERE-120 was evaluated in trial NCT00985517, titled Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease. The Phase 1/2 study was completed in the United States, enrolled 57 participants with idiopathic Parkinson's disease, and was randomized, double-blind, and sham-controlled.
CERE-120 is designed to deliver neurturin, a neurotrophic factor, using an adeno-associated virus vector. The gene therapy approach aims to provide sustained neurturin expression in the brain of patients with idiopathic Parkinson's disease through viral vector-mediated gene delivery.