Evidence map›Paper›PMID 20970382›Full record

Trial reportThe Lancet. Neurology2010

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.

William J Marks, Raymond T Bartus, Joao Siffert, Charles S Davis, Andres Lozano, Nicholas Boulis, Jerrold Vitek, Mark Stacy, Dennis Turner, Leonard Verhagen and 16 more

Registry-linked trialAbstract readClinical Trial, Phase IIMulticenter StudyRandomized Controlled Trial
PubMed Publisher
In one paragraph

Trial report in The Lancet. Neurology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00400634 (Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120), which is not on this map. Cited by 303 papers, 4 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
303citing papers in PubMed, 4 pooled it
21.7field-weighted citation impact, top 1% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT00400634 phase2completednot on this map

Multicenter, Randomized, Double-Blind, Sham Surgery-Controlled Study of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) to Assess the Efficacy and Safety of Bilateral Intraputaminal (IPu) Delivery in Subjects With Idiopathic Parkinson's Disease

TypeinterventionalSponsorSangamo TherapeuticsRan2006 to 2008Enrolled58ConditionsIdiopathic Parkinson's DiseaseArmsCERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]), Sham Surgery
3 · Its place in the literature

Who cites it

303 citing papers in PubMed, 4 syntheses or guidelines pooled it, 673 citations in OpenAlex.

  1. Pooled it
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  5. Trial
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  8. A Phase 1 Study of Convection-Enhanced Delivery of Intraputaminal AAV2-GDNF in Advanced Parkinson's Disease.Movement disorders : official journal of the Movement Disorder Society · 2026
    Article
  9. Article
  10. Review
  11. Article
  12. Review
  13. Clinical GDNF delivery methods for Parkinson's disease.Journal of Parkinson's disease · 2026
    Review
  14. Review
  15. Article
  16. Review
  17. Review
  18. Review
  19. Temperature-modulated separation of therapeutic cells, viral vectors, and exosomes using functional polymers.Analytical sciences : the international journal of the Japan Society for Analytical Chemistry · 2025
    Review
  20. Article

243 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

26 authors at 11 institutions in 3 countries.

William J MarksDepartment of Neurology, University of California San Francisco, San Francisco, CA, USA.
Raymond T BartusCeregene, San Diego, CA, USA.
Joao SiffertCeregene, San Diego, CA, USA.
Charles S DavisCSD Biostatistics, San Diego, CA, USA.
Andres LozanoDepartment of Neurosurgery, University of Toronto, Toronto, Canada.
Nicholas BoulisDepartment of Neurosurgery, Cleveland Clinic, Cleveland, OH, USA.
Jerrold VitekDepartment of Neurology, Cleveland Clinic, Cleveland, OH, USA.
Mark StacyDepartment of Neurology, Duke University, Durham, NC, USA.
Dennis TurnerDepartment of Neurosurgery, Duke University, Durham, NC, USA.
Leonard VerhagenDepartment of Neurology, Rush Medical University, Chicago, IL, USA.
Roy BakayDepartment of Neurosurgery, Rush Medical University, Chicago, IL, USA.
Raymond WattsDepartment of Neurology, University of Alabama, Birmingham, AL, USA.
Barton GuthrieDepartment of Neurosurgery, University of Alabama, Birmingham, AL, USA.
Joseph JankovicDepartment of Neurology, Baylor University, Houston, TX, USA.
Richard SimpsonDepartment of Neurosurgery, Baylor University, Houston, TX, USA.
Michele TagliatiDepartment of Neurology, Mount Sinai School of Medicine, New York, NY, USA.
Ron AltermanDepartment of Neurosurgery, Mount Sinai School of Medicine, New York, NY, USA.
Matthew SternDepartment of Neurology, University of Pennsylvania, Philadelphia, PA, USA.
Gordon BaltuchDepartment of Neurosurgery, University of Pennsylvania, Philadelphia, PA, USA.
Philip A StarrDepartment of Neurosurgery, University of California San Francisco, San Francisco, CA, USA.
Paul S LarsonDepartment of Neurosurgery, University of California San Francisco, San Francisco, CA, USA.
Jill L OstremDepartment of Neurology, University of California San Francisco, San Francisco, CA, USA.
John NuttDepartment of Neurology, University of Oregon, Portland, OR, USA.
Karl KieburtzDepartment of Neurology, University of Rochester, Rochester, NY, USA.
Jeffrey H KordowerDepartment of Neurology, Rush Medical University, Chicago, IL, USA.
C Warren OlanowDepartment of Neurology, Mount Sinai School of Medicine, New York, NY, USA; Department of Neuroscience, Mount Sinai School of Medicine, New York, NY, USA; Institute of Neurology, IRCCS San Raffaele Pisana, Rome, Italy. Electronic address: warren.olanow@mssm.edu.
University of California, San Francisco · USIcahn School of Medicine at Mount Sinai · USBaylor University · USCleveland Clinic · USDuke University · USUniversity of Alabama at Birmingham · USUniversity of Pennsylvania · USCancer Research And Biostatistics · USUniversity of Oregon · USUniversity of Rochester · USUniversity of Toronto · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundIn an open-label phase 1 trial, gene delivery of the trophic factor neurturin via an adeno-associated type-2 vector (AAV2) was well tolerated and seemed to improve motor function in patients with advanced Parkinson's disease. We aimed to assess the safety and efficacy of AAV2-neurturin in a double-blind, phase 2 randomised trial.

methodsWe did a multicentre, double-blind, sham-surgery controlled trial in patients with advanced Parkinson's disease. Patients were randomly assigned (2:1) by a central, computer generated, randomisation code to receive either AAV2-neurturin (5·4 × 10¹¹ vector genomes) injected bilaterally into the putamen or sham surgery. All patients and study personnel with the exception of the neurosurgical team were masked to treatment assignment. The primary endpoint was change from baseline to 12 months in the motor subscore of the unified Parkinson's disease rating scale in the practically-defined off state. All randomly assigned patients who had at least one assessment after baseline were included in the primary analyses. This trial is registered at ClinicalTrials.gov, NCT00400634.

resultsBetween December, 2006, and November, 2008, 58 patients from nine sites in the USA participated in the trial. There was no significant difference in the primary endpoint in patients treated with AAV2-neurturin compared with control individuals (difference -0·31 [SE 2·63], 95% CI -5·58 to 4·97; p=0·91). Serious adverse events occurred in 13 of 38 patients treated with AAV2-neurturin and four of 20 control individuals. Three patients in the AAV2-neurturin group and two in the sham surgery group developed tumours.

interpretationIntraputaminal AAV2-neurturin is not superior to sham surgery when assessed using the UPDRS motor score at 12 months. However, the possibility of a benefit with additional targeting of the substantia nigra and longer term follow-up should be investigated in further studies.

fundingCeregene and Michael J Fox Foundation for Parkinson's Research.

Indexed as

AdultAgedAnalysis of VarianceDependovirusDouble-Blind MethodFemaleFollow-Up StudiesGenetic TherapyHumansMaleMiddle AgedMotor ActivityNeurturinParkinson DiseasePutamenSeverity of Illness IndexNeurturinNRTN protein, human

Identifiers

PMID20970382
OpenAlexW2149731696

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.