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

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ClinicalTrials.gov Identifier: NCT00400634
Recruitment Status : Completed
First Posted : November 17, 2006
Results First Posted : August 14, 2012
Last Update Posted : August 14, 2012
Sponsor:
Information provided by (Responsible Party):
Ceregene

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Idiopathic Parkinson's Disease
Interventions Genetic: CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN])
Procedure: Sham Surgery
Enrollment 58

Recruitment Details 58 subjects were recruited over a 10.5 month period at 11 centers in the US
Pre-assignment Details  
Arm/Group Title CERE-120 Treatment Group Sham Surgery Control Group
Hide Arm/Group Description Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) Subjects who were randomized to undergo sham surgery (partial burr holes)
Period Title: Overall Study
Started 38 20
Completed 34 19
Not Completed 4 1
Arm/Group Title CERE-120 Treatment Group Sham Surgery Control Group Total
Hide Arm/Group Description Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) Subjects who were randomized to undergo sham surgery (partial burr holes) Total of all reporting groups
Overall Number of Baseline Participants 38 20 58
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 38 participants 20 participants 58 participants
60.1  (7.56) 57.3  (8.3) 59.1  (8.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 38 participants 20 participants 58 participants
Female
10
  26.3%
5
  25.0%
15
  25.9%
Male
28
  73.7%
15
  75.0%
43
  74.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 38 participants 20 participants 58 participants
38 20 58
1.Primary Outcome
Title UPDRS Part III OFF
Hide Description 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.
Time Frame Change from Baseline to 12 Month Visit
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who completed the end-of-study visit at month 12 and had no important protocol deviations that potentially could have affected the efficacy assessment of the study drug.
Arm/Group Title CERE-120 Treatment Group Sham Surgery Control Group
Hide Arm/Group Description:
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Subjects who were randomized to undergo sham surgery (partial burr holes)
Overall Number of Participants Analyzed 34 19
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-7.21
(-10.34 to -4.09)
-6.91
(-11.16 to -2.66)
2.Other Pre-specified Outcome
Title UPDRS Part III OFF
Hide Description 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.
Time Frame Change from Baseline to 18 Month Visit
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Subjects who completed the 12-month double-blind posttreatment period of the study continued to undergo double-blind assessments every 3 months until the last subject had completed the end-of-study visit at month 12. The LOCF method was used to impute data at month 18 for the subjects who had blinded data through month 15.
Arm/Group Title CERE-120 Treatment Group Sham Surgery Control Group
Hide Arm/Group Description:
Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Subjects who were randomized to undergo sham surgery (partial burr holes)
Overall Number of Participants Analyzed 19 11
Least Squares Mean (Standard Error)
Unit of Measure: units on a scale
-11.96  (-7.068) -4.34  (-2.479)
Time Frame 23 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title CERE-120 Treatment Group Sham Surgery Control Group
Hide Arm/Group Description Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg) Subjects who were randomized to undergo sham surgery (partial burr holes)
All-Cause Mortality
CERE-120 Treatment Group Sham Surgery Control Group
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
CERE-120 Treatment Group Sham Surgery Control Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   13/38 (34.21%)      4/20 (20.00%)    
Cardiac disorders     
Chest Pain  1  0/38 (0.00%)  0 1/20 (5.00%)  1
Gastrointestinal disorders     
Gastroenteritis  1  0/38 (0.00%)  0 1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders     
spinal compression fracture  1 [1]  1/38 (2.63%)  1 0/20 (0.00%)  0
Lumbar Stenosis  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Nerve Root Compression  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Glioblastoma  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Prostate Cancer  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Esophageal Cancer  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Neoplasm Progression  1  0/38 (0.00%)  0 1/20 (5.00%)  1
Nervous system disorders     
Cerebral Hemorrhage  1 [3]  1/38 (2.63%)  1 0/20 (0.00%)  0
Gait Disturbance  1 [1]  1/38 (2.63%)  1 0/20 (0.00%)  0
Fall  1  0/38 (0.00%)  0 1/20 (5.00%)  1
Renal and urinary disorders     
Urinary Retention  1 [2]  2/38 (5.26%)  2 0/20 (0.00%)  0
Surgical and medical procedures     
Device Failure  1 [2]  1/38 (2.63%)  1 0/20 (0.00%)  0
Vascular disorders     
Death  1 [4]  2/38 (5.26%)  2 0/20 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (10.0)
[1]
Not considered related to CERE-120
[2]
Considered not related to CERE-120
[3]
Considered not related to CERE-120, Related to surgical procedure
[4]
Neither death was considered related to CERE-120
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
CERE-120 Treatment Group Sham Surgery Control Group
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   38/38 (100.00%)      20/20 (100.00%)    
Eye disorders     
Vision Blurred  1  1/38 (2.63%)  2/20 (10.00%) 
Gastrointestinal disorders     
Nausea  1  13/38 (34.21%)  6/20 (30.00%) 
Constipation  1  4/38 (10.53%)  3/20 (15.00%) 
Diarrhoea  1  3/38 (7.89%)  1/20 (5.00%) 
Vomiting  1  3/38 (7.89%)  1/20 (5.00%) 
Dyspepsia  1  3/38 (7.89%)  0/20 (0.00%) 
General disorders     
Fatigue  1  3/38 (7.89%)  1/20 (5.00%) 
Face Edema  1  2/38 (5.26%)  1/20 (5.00%) 
Gait Disturbance  1  2/38 (5.26%)  1/20 (5.00%) 
Edema  1  2/38 (5.26%)  0/20 (0.00%) 
Edema Peripheral  1  2/38 (5.26%)  0/20 (0.00%) 
Infections and infestations     
Urinary Tract Infection  1  4/38 (10.53%)  2/20 (10.00%) 
Nasopharyngitis  1  3/38 (7.89%)  1/20 (5.00%) 
Upper Respiratory Tract Infection  1  2/38 (5.26%)  2/20 (10.00%) 
Bronchitis  1  3/38 (7.89%)  0/20 (0.00%) 
Sinusitis  1  3/38 (7.89%)  0/20 (0.00%) 
Gastroenteritis  1  0/38 (0.00%)  2/20 (10.00%) 
Injury, poisoning and procedural complications     
Post Procedural Pain  1  11/38 (28.95%)  7/20 (35.00%) 
Headache Postoperative  1  5/38 (13.16%)  5/20 (25.00%) 
Incision Site Complication  1  6/38 (15.79%)  2/20 (10.00%) 
Fall  1  4/38 (10.53%)  2/20 (10.00%) 
Procedural Hypotension  1  3/38 (7.89%)  0/20 (0.00%) 
Investigations     
Blood Testosterone Decreased  1  2/38 (5.26%)  0/20 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back Pain  1  5/38 (13.16%)  2/20 (10.00%) 
Arthralgia  1  3/38 (7.89%)  3/20 (15.00%) 
Pain in Extremity  1  5/38 (13.16%)  0/20 (0.00%) 
Shoulder Pain  1  2/38 (5.26%)  2/20 (10.00%) 
Osteoarthritis  1  2/38 (5.26%)  0/20 (0.00%) 
Nervous system disorders     
Headache  1  28/38 (73.68%)  10/20 (50.00%) 
Dyskinesia  1  9/38 (23.68%)  6/20 (30.00%) 
Parkinson's Disease  1  8/38 (21.05%)  4/20 (20.00%) 
Dizziness  1  4/38 (10.53%)  4/20 (20.00%) 
Hypoaesthesia  1  1/38 (2.63%)  4/20 (20.00%) 
Tremor  1  2/38 (5.26%)  2/20 (10.00%) 
Freezing Phenomena  1  2/38 (5.26%)  1/20 (5.00%) 
Balance Disorder  1  2/38 (5.26%)  0/20 (0.00%) 
Brain Edema  1  2/38 (5.26%)  0/20 (0.00%) 
Cognitive Disorder  1  2/38 (5.26%)  0/20 (0.00%) 
Psychiatric disorders     
Insomnia  1  8/38 (21.05%)  2/20 (10.00%) 
Depression  1  4/38 (10.53%)  3/20 (15.00%) 
Anxiety  1  2/38 (5.26%)  4/20 (20.00%) 
Confusional State  1  1/38 (2.63%)  2/20 (10.00%) 
Hallucinations  1  3/38 (7.89%)  0/20 (0.00%) 
Renal and urinary disorders     
Micturation Urgency  1  4/38 (10.53%)  1/20 (5.00%) 
Hematuria  1  3/38 (7.89%)  0/20 (0.00%) 
Pollakiuria  1  2/38 (5.26%)  1/20 (5.00%) 
Urinary Incontinence  1  3/38 (7.89%)  0/20 (0.00%) 
Urinary Retention  1  3/38 (7.89%)  0/20 (0.00%) 
Renal Failure  1  2/38 (5.26%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Rhinorrhea  1  2/38 (5.26%)  0/20 (0.00%) 
Sleep Apnea Syndrome  1  2/38 (5.26%)  0/20 (0.00%) 
Skin and subcutaneous tissue disorders     
Rash  1  5/38 (13.16%)  0/20 (0.00%) 
Periorbital Edema  1  3/38 (7.89%)  1/20 (5.00%) 
Pruritis  1  3/38 (7.89%)  0/20 (0.00%) 
Vascular disorders     
Hypertension  1  3/38 (7.89%)  2/20 (10.00%) 
Hypotension  1  2/38 (5.26%)  1/20 (5.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (10.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Raymond T. Bartus, Executive Vice President and Cheif Scientific Officer
Organization: Ceregene, Inc.
Phone: 858-458-8823
Responsible Party: Ceregene
ClinicalTrials.gov Identifier: NCT00400634     History of Changes
Other Study ID Numbers: CERE-120-02
First Submitted: November 15, 2006
First Posted: November 17, 2006
Results First Submitted: May 24, 2012
Results First Posted: August 14, 2012
Last Update Posted: August 14, 2012