a study on Parkinson's Disease
The purpose of this study is to evaluate the safety and potential benefits of CERE-120 in the treatment of Parkinson's disease. CERE-120 is an experimental drug that consists of an adeno-associated virus (AAV) that was engineered to carry the human gene for neurturin, a neurotrophic (growth) factor. Similar to other growth factors (such as GDNF), neurturin is capable of restoring function and protecting brain cells from further damage. The virus used in CERE-120 is not known to cause disease in people. CERE-120 is delivered directly to the brain cells most affected in Parkinson's disease - the dopamine producing neurons. CERE-120 is injected during brain surgery. Once in place, CERE-120 continuously produces neurturin.
A Phase 1/2 Trial Assessing the Safety and Efficacy of Bilateral Intraputaminal and Intranigral Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) in Subjects With Idiopathic Parkinson's Disease
Approximately sixty patients with Parkinson's disease will participate in this study. The first part of the study (Phase 1) is now complete. Six people with Parkinson's disease received CERE-120 in Phase 1 without complications.
The second part of the study (Phase 2) will provide more information about the safety of CERE-120 and also evaluate if it is beneficial in the treatment of Parkinson's disease. In Phase 2, approximately 52 people with Parkinson's disease will be enrolled. Half of the subjects will receive CERE-120 and the other half will undergo a "placebo" surgery where no medication will be injected. Treatment assignment will be random (like the flip of a coin). Participants in both phases of the study will be followed for three years after surgery.
In this study, CERE-120 will be injected by a neurosurgeon directly in the substantia nigra (where dopamine producing cells are located) and in the putamen (where the dopamine cells project).
CERE-120 has been carefully studied in laboratory animals without any toxicity despite very high doses. In addition, in animals with symptoms similar to Parkinson's disease, CERE-120 was shown to protect brain cells and restore their function.
CERE-120 has been previously studied in 50 people with Parkinson's enrolled in two clinical studies. These patients have been followed for over 2 years (some for almost 5 years) and so far CERE-120 has been well tolerated. Whereas CERE-120 was not better than placebo on the primary efficacy measure in a completed study, several measures of improvement suggested a modest benefit. In order to build upon these findings and increase the chances for a stronger clinical benefit, a dose increase and modifications in the delivery targets have been implemented in the current study.
Idiopathic Parkinson's Disease Parkinson's disease Movement Disorders Gene Therapy Neurotrophic Factors GDNF Neurturin DBS Dopamine
Open to people ages 35–70
UPDRS Part III: Motor Examination
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