Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 delivers the gene for neurturin, a neurotrophic factor, directly into the brain. It is not approved for any indication and remains in clinical development.
CERE-120 is being developed by Sangamo Therapeutics, Inc., which trades on the Nasdaq under the ticker SGMO. Sangamo is the sponsor of the clinical program evaluating the therapy in idiopathic Parkinson's disease.
CERE-120 is in Phase 1 clinical development. One Phase 1 trial has been completed, and no trials are currently active. The program remains investigational and has not been approved by the FDA for any use.
CERE-120 has been evaluated in a single completed Phase 1 trial, NCT00985517, titled Safety and Efficacy of CERE-120 in Subjects With Parkinson's Disease. The trial enrolled 57 participants in the United States and was randomized, double-blind, and sham-controlled.
CERE-120 is an adeno-associated virus gene therapy that delivers the gene for neurturin, a neurotrophic factor, to the brain. The goal is to support the survival and function of dopaminergic neurons affected by idiopathic Parkinson's disease. It is administered directly to the brain.