Double-Blind, Multicenter, Sham Surgery Controlled Study of CERE-120 in Subjects With Idiopathic Parkinson's Disease

Summary

The purpose of this double blind study is to determine whether CERE-120 (adeno-associated virus serotype 2 \[AAV2\]-neurturin \[NTN\]) is effective and safe in the treatment of patients with idiopathic Parkinson's Disease. CERE-120 is administered via bilateral stereotactic injections targeting the putaminal region of the brain. The design of this study involves approximately 34 patients receiving CERE-120 treatment via stereotactic surgery and approximately 17 patients receiving sham stereotactic surgery (no CERE-120 administered).

Timeline

Start

2006-11

Primary completion

2008-11

Completion

2008-11

Outcome

Missed primary endpoint

paper There was no significant difference in the primary endpoint in patients treated with AAV2-neurturin compared with control individuals PMID 20970382 ↗

paper Intraputaminal AAV2-neurturin is not superior to sham surgery when assessed using the UPDRS motor score at 12 months. PMID 20970382 ↗

Publications

• Results Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW. Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	CERE-120	Gene therapy (AAV / viral vector)	5.4 vg	Other

Indications