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CERE-120 · 1 trial · 1 indication
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.
| Arm | Type | Description |
|---|---|---|
| 1 | EXPERIMENTAL | Intracerebral administration of CERE-120 |
| 2 | SHAM_COMPARATOR | Sham Neurosurgery |
| Name | Type | Description |
|---|---|---|
| CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) | DRUG | CERE-120 5.4 x 10\^11 vg |
| Sham Surgery | PROCEDURE | Bilateral partial thickness burr holes placed, no intraparenchymal injections |
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...
CERE-120 is an investigational gene therapy being developed for idiopathic Parkinson's disease. It is designed to deliver a gene to brain cells to support the survival and function of dopamine-producing neurons affected by the disease. It has been studied in a Phase 2 clinical trial and is not approved for any use.
CERE-120 is being developed by Sangamo Therapeutics, Inc., which trades on the Nasdaq under the ticker SGMO. The company is responsible for the clinical development program for the therapy in idiopathic Parkinson's disease.
CERE-120 has completed a Phase 2 clinical trial in idiopathic Parkinson's disease. It is an investigational therapy and has not been approved by the FDA. No active trials are currently listed for CERE-120, and further development steps have not been announced.
CERE-120 has been studied in one completed Phase 2 trial, NCT00400634, titled a double-blind, multicenter, sham surgery controlled study in subjects with idiopathic Parkinson's disease. The trial enrolled 58 participants in the United States and was randomized, double-blind, and sham-controlled.
Yes, CERE-120 is also known as AAV2-neurturin, which stands for adeno-associated virus serotype 2 delivering the neurturin gene. This is the full scientific name for the gene therapy construct used in the clinical program.