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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ceregene
ClinicalTrials.gov Identifier:
NCT00400634
First received: November 15, 2006
Last updated: July 6, 2012
Last verified: July 2012
Results First Received: May 24, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, 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

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
58 subjects were recruited over a 10.5 month period at 11 centers in the US

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
CERE-120 Treatment Group Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Sham Surgery Control Group Subjects who were randomized to undergo sham surgery (partial burr holes)

Participant Flow:   Overall Study
    CERE-120 Treatment Group     Sham Surgery Control Group  
STARTED     38     20  
COMPLETED     34     19  
NOT COMPLETED     4     1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
CERE-120 Treatment Group Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Sham Surgery Control Group Subjects who were randomized to undergo sham surgery (partial burr holes)
Total Total of all reporting groups

Baseline Measures
    CERE-120 Treatment Group     Sham Surgery Control Group     Total  
Number of Participants  
[units: participants]
  38     20     58  
Age  
[units: years]
Mean ± Standard Deviation
  60.1  ± 7.56     57.3  ± 8.3     59.1  ± 8.0  
Gender  
[units: participants]
     
Female     10     5     15  
Male     28     15     43  
Region of Enrollment  
[units: participants]
     
United States     38     20     58  



  Outcome Measures
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1.  Primary:   UPDRS Part III OFF   [ Time Frame: Change from Baseline to 12 Month Visit ]

2.  Other Pre-specified:   UPDRS Part III OFF   [ Time Frame: Change from Baseline to 18 Month Visit ]
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Measure Type Other Pre-specified
Measure Title UPDRS Part III OFF
Measure 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  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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.

Reporting Groups
  Description
CERE-120 Treatment Group Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Sham Surgery Control Group Subjects who were randomized to undergo sham surgery (partial burr holes)

Measured Values
    CERE-120 Treatment Group     Sham Surgery Control Group  
Number of Participants Analyzed  
[units: participants]
  19     11  
UPDRS Part III OFF  
[units: units on a scale]
Least Squares Mean ± Standard Error
  -11.96  ± -7.068     -4.34  ± -2.479  

No statistical analysis provided for UPDRS Part III OFF




  Serious Adverse Events


  Other Adverse Events
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Time Frame 23 months
Additional Description No text entered.

Frequency Threshold
Threshold above which other adverse events are reported   5%  

Reporting Groups
  Description
CERE-120 Treatment Group Subjects who were randomized to receive bilateral intraputaminal administration of CERE-120 (5.4 x 10^11 vg)
Sham Surgery Control Group Subjects who were randomized to undergo sham surgery (partial burr holes)

Other Adverse Events
    CERE-120 Treatment Group     Sham Surgery Control Group  
Total, other (not including serious) adverse events      
# participants affected / at risk     38/38     20/20  
Eye disorders      
Vision Blurred † 1    
# participants affected / at risk     1/38 (2.63%)     2/20 (10.00%)  
Gastrointestinal disorders      
Nausea † 1    
# participants affected / at risk     13/38 (34.21%)     6/20 (30.00%)  
Constipation † 1    
# participants affected / at risk     4/38 (10.53%)     3/20 (15.00%)  
Diarrhoea † 1    
# participants affected / at risk     3/38 (7.89%)     1/20 (5.00%)  
Vomiting † 1    
# participants affected / at risk     3/38 (7.89%)     1/20 (5.00%)  
Dyspepsia † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
General disorders      
Fatigue † 1    
# participants affected / at risk     3/38 (7.89%)     1/20 (5.00%)  
Face Edema † 1    
# participants affected / at risk     2/38 (5.26%)     1/20 (5.00%)  
Gait Disturbance † 1    
# participants affected / at risk     2/38 (5.26%)     1/20 (5.00%)  
Edema † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Edema Peripheral † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Infections and infestations      
Urinary Tract Infection † 1    
# participants affected / at risk     4/38 (10.53%)     2/20 (10.00%)  
Nasopharyngitis † 1    
# participants affected / at risk     3/38 (7.89%)     1/20 (5.00%)  
Upper Respiratory Tract Infection † 1    
# participants affected / at risk     2/38 (5.26%)     2/20 (10.00%)  
Bronchitis † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Sinusitis † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Gastroenteritis † 1    
# participants affected / at risk     0/38 (0.00%)     2/20 (10.00%)  
Injury, poisoning and procedural complications      
Post Procedural Pain † 1    
# participants affected / at risk     11/38 (28.95%)     7/20 (35.00%)  
Headache Postoperative † 1    
# participants affected / at risk     5/38 (13.16%)     5/20 (25.00%)  
Incision Site Complication † 1    
# participants affected / at risk     6/38 (15.79%)     2/20 (10.00%)  
Fall † 1    
# participants affected / at risk     4/38 (10.53%)     2/20 (10.00%)  
Procedural Hypotension † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Investigations      
Blood Testosterone Decreased † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Musculoskeletal and connective tissue disorders      
Back Pain † 1    
# participants affected / at risk     5/38 (13.16%)     2/20 (10.00%)  
Arthralgia † 1    
# participants affected / at risk     3/38 (7.89%)     3/20 (15.00%)  
Pain in Extremity † 1    
# participants affected / at risk     5/38 (13.16%)     0/20 (0.00%)  
Shoulder Pain † 1    
# participants affected / at risk     2/38 (5.26%)     2/20 (10.00%)  
Osteoarthritis † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Nervous system disorders      
Headache † 1    
# participants affected / at risk     28/38 (73.68%)     10/20 (50.00%)  
Dyskinesia † 1    
# participants affected / at risk     9/38 (23.68%)     6/20 (30.00%)  
Parkinson's Disease † 1    
# participants affected / at risk     8/38 (21.05%)     4/20 (20.00%)  
Dizziness † 1    
# participants affected / at risk     4/38 (10.53%)     4/20 (20.00%)  
Hypoaesthesia † 1    
# participants affected / at risk     1/38 (2.63%)     4/20 (20.00%)  
Tremor † 1    
# participants affected / at risk     2/38 (5.26%)     2/20 (10.00%)  
Freezing Phenomena † 1    
# participants affected / at risk     2/38 (5.26%)     1/20 (5.00%)  
Balance Disorder † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Brain Edema † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Cognitive Disorder † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Psychiatric disorders      
Insomnia † 1    
# participants affected / at risk     8/38 (21.05%)     2/20 (10.00%)  
Depression † 1    
# participants affected / at risk     4/38 (10.53%)     3/20 (15.00%)  
Anxiety † 1    
# participants affected / at risk     2/38 (5.26%)     4/20 (20.00%)  
Confusional State † 1    
# participants affected / at risk     1/38 (2.63%)     2/20 (10.00%)  
Hallucinations † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Renal and urinary disorders      
Micturation Urgency † 1    
# participants affected / at risk     4/38 (10.53%)     1/20 (5.00%)  
Hematuria † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Pollakiuria † 1    
# participants affected / at risk     2/38 (5.26%)     1/20 (5.00%)  
Urinary Incontinence † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Urinary Retention † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Renal Failure † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Respiratory, thoracic and mediastinal disorders      
Rhinorrhea † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Sleep Apnea Syndrome † 1    
# participants affected / at risk     2/38 (5.26%)     0/20 (0.00%)  
Skin and subcutaneous tissue disorders      
Rash † 1    
# participants affected / at risk     5/38 (13.16%)     0/20 (0.00%)  
Periorbital Edema † 1    
# participants affected / at risk     3/38 (7.89%)     1/20 (5.00%)  
Pruritis † 1    
# participants affected / at risk     3/38 (7.89%)     0/20 (0.00%)  
Vascular disorders      
Hypertension † 1    
# participants affected / at risk     3/38 (7.89%)     2/20 (10.00%)  
Hypotension † 1    
# participants affected / at risk     2/38 (5.26%)     1/20 (5.00%)  
Events were collected by systematic assessment
1 Term from vocabulary, MedDRA (10.0)



  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Raymond T. Bartus, Executive Vice President and Cheif Scientific Officer
Organization: Ceregene, Inc.
phone: 858-458-8823
e-mail: rtbartus@ceregene.com


Publications of Results:

Responsible Party: Ceregene
ClinicalTrials.gov Identifier: NCT00400634     History of Changes
Other Study ID Numbers: CERE-120-02
Study First Received: November 15, 2006
Results First Received: May 24, 2012
Last Updated: July 6, 2012
Health Authority: United States: Food and Drug Administration