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

NCT00400634 | Completed Phase 2 Results DMC

• 1 other identifier: CERE-120-02
• Study Type: interventional
• Target: 58
• Locations: 1 country
• Sites: 9

Brief 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).

Conditions

Idiopathic Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

• UPDRS Part III OFF

  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.

  Change from Baseline to 12 Month Visit

Other Outcomes (1)

• UPDRS Part III OFF

  Change from Baseline to 18 Month Visit

Study Arms (2)

1

EXPERIMENTAL

Intracerebral administration of CERE-120

Drug: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])

2

SHAM COMPARATOR

Sham Neurosurgery

Procedure: Sham Surgery

Interventions

CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) DRUG

CERE-120 5.4 x 10\^11 vg

1

Sham Surgery PROCEDURE

Bilateral partial thickness burr holes placed, no intraparenchymal injections

2

Eligibility Criteria

• Age: 35 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 or nonpregnant females 35-75 years of age, inclusive.
• A UPDRS motor scale score of 30 or greater in the practically defined off condition during the 30-day eligibility evaluation period.
• Stable doses of antiparkinsonian medications and parkinsonian features for the 60-day period preceding the surgical procedure.
• No conditions that would render the subject unsuitable for surgery, or that would interfere with any of the assessments of efficacy or safety in this trial.
• Subject's informed consent prior to the performance of any study-specific procedures.

You may not qualify if:

• Subjects with atypical or secondary parkinsonism.
• Any subject, in the judgment of the investigator, for whom participation in the study would pose a safety risk including, but not limited to, a history of any clinically significant medical, psychiatric, or laboratory abnormality.
• History of treatment of PD by any procedure involving intracranial surgery or implantation of a device.
• MRI of the brain within 12 months before the surgical procedure that indicates the presence of an abnormality that may interfere with the assessments of safety or efficacy or would, in the judgment of the investigator, represent a surgical risk to the subject.
• Any disorder that precludes a surgical procedure (e.g., signs of sepsis or inadequately treated infection) or alters wound healing.
• Receipt of antiplatelet agents for at least 10 days prior to the surgical procedure.
• A score of less than or equal to 27 on the Folstein Mini-Mental examination performed during the eligibility evaluation period or clinical evidence of cognitive impairment that would affect the subject's ability to sign the informed consent or perform any of the protocol required assessments.
• Chemotherapy, cytotoxic therapy, or immunotherapy within 6 weeks prior to the surgical procedure.
• Vaccinations within 30 days prior to the surgical procedure.
• History, within 2 years before the surgical procedure, of drug or alcohol abuse.
• Treatment with neuroleptics within 1 year before the surgical procedure.
• Any medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) that would interfere with the assessment of efficacy and safety in this trial or would compromise the ability of the subject to undergo study procedures (e.g., MRI, PET), or give informed consent.
• History of prior gene transfer therapy.
• Treatment with an investigational agent within 60 days before the surgical procedure.

Sponsors & Collaborators

• Sangamo Therapeutics: lead
• Ceregene: collaborator

Study Sites (9)

University of Alabama, Birmingham

Birmingham, Alabama, 35233, United States

Location

University of California, San Francisco

San Francisco, California, 94143-0138, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Pennsylvania Hospital

Philadelphia, Pennsylvania, 19107, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (1)

• 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.

  PMID: 20970382 RESULT

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Results Point of Contact

• Title: Raymond T. Bartus, Executive Vice President and Cheif Scientific Officer
• Organization: Ceregene, Inc.

rtbartus@ceregene.com

858-458-8823

Study Officials

• Joao Siffert, M.D.

  Ceregene

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 15, 2006
• First Posted: November 17, 2006
• Study Start: November 1, 2006
• Primary Completion: November 1, 2008
• Study Completion: November 1, 2008
• Last Updated: November 10, 2022
• Results First Posted: August 14, 2012

Record last verified: 2019-08

Locations

US (9)