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

Idiopathic Parkinson's Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00400634
• Other study ID #: CERE-120-02
• Status: Completed
• Phase: Phase 2
• Start date: November 2006
• Est. completion date: November 2008

Study information

• Verified date: August 2019
• Source: Sangamo Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 58
• Est. completion date: November 2008
• Est. primary completion date: November 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 75 Years

Eligibility

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 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.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Idiopathic Parkinson's Disease
• Parkinson Disease

Intervention

Drug:
• CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
CERE-120 5.4 x 10^11 vg

Procedure:
• Sham Surgery
Bilateral partial thickness burr holes placed, no intraparenchymal injections

Locations

Country	Name	City	State
United States	University of Alabama, Birmingham	Birmingham	Alabama
United States	Rush University Medical Center	Chicago	Illinois
United States	Cleveland Clinic	Cleveland	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	Baylor College of Medicine	Houston	Texas
United States	Mount Sinai School of Medicine	New York	New York
United States	Pennsylvania Hospital	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	University of California, San Francisco	San Francisco	California

Sponsors (2)

Lead Sponsor	Collaborator
Sangamo Therapeutics	Ceregene

Country where clinical trial is conducted

United States, 

References & 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, Ko — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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 18 Month Visit
Primary	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