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Phase I Deep Brain Stimulation (DBS) vs. Best Medical Therapy (BMT) Trial

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ClinicalTrials.gov Identifier: NCT00056563
Recruitment Status : Completed
First Posted : March 19, 2003
Results First Posted : July 30, 2013
Last Update Posted : May 9, 2014
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
VA Office of Research and Development ( US Department of Veterans Affairs )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Parkinson's Disease
Interventions Device: Bilateral Deep Brain Stimulation
Other: best medical therapy
Enrollment 255
Recruitment Details Patient recruitment begun in May 2002 and the discontinuation of randomization to the BMT arm was approved in August 2005 and participating sites received their IRB approval of this change through October 2005. A total of 255 patients were randomized to either BMT or DBS from 7 VA medical centers and 6 affiliated university sites.
Pre-assignment Details An additional 23 patients were screened after signing the informed consent form but not randomized. 11 of them weren’t willing to participate in the follow-up visits after intervention, 7 of them had neuropsychological dysfunction, 2 of them were not responsive to levodopa, and 3 had medical contraindication to surgery and/or DBS stimulation.
Arm/Group Title 1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Hide Arm/Group Description

Bilateral Deep Brain Stimulation

Bilateral Deep Brain Stimulation : The DBS site (STN or GPi) will be assigned on a random basis at the time the patient enters the surgical phase of the trial.

Best Medical Therapy

best medical therapy : Participants will initially be randomized to DBS or to 6 months of

Period Title: Overall Study
Started 121 134
Completed 121 134
Not Completed 0 0
Arm/Group Title 1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy Total
Hide Arm/Group Description

Bilateral Deep Brain Stimulation

Bilateral Deep Brain Stimulation : The DBS site (STN or GPi) will be assigned on a random basis at the time the patient enters the surgical phase of the trial.

Best Medical Therapy

best medical therapy : Participants will initially be randomized to DBS or to 6 months of "best medical therapy." BMT participants will then proceed into the surgical phase of the trial. Effective 08/05/05, randomization to the BMT arm has been discontinued since the study has sufficient information to compare the outcomes of DBS and BMT patients at 6 months.

Total of all reporting groups
Overall Number of Baseline Participants 121 134 255
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 121 participants 134 participants 255 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
90
  74.4%
97
  72.4%
187
  73.3%
>=65 years
31
  25.6%
37
  27.6%
68
  26.7%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 121 participants 134 participants 255 participants
62.4  (8.8) 62.3  (9.0) 62.3  (8.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 121 participants 134 participants 255 participants
Female
23
  19.0%
24
  17.9%
47
  18.4%
Male
98
  81.0%
110
  82.1%
208
  81.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 121 participants 134 participants 255 participants
121 134 255
1.Primary Outcome
Title The Difference of Time Spent in the 'on' State Without Troublesome Dyskinesia Based on Patient Motor Diaries as Compared to the Baseline.
Hide Description [Not Specified]
Time Frame at six months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Hide Arm/Group Description:

Bilateral Deep Brain Stimulation

Bilateral Deep Brain Stimulation : The DBS site (STN or GPi) will be assigned on a random basis at the time the patient enters the surgical phase of the trial.

Best Medical Therapy

best medical therapy : Participants will initially be randomized to DBS or to 6 months of

Overall Number of Participants Analyzed 121 134
Mean (95% Confidence Interval)
Unit of Measure: Hours per day
4.6
(3.8 to 5.3)
0
(-0.5 to 0.5)
2.Secondary Outcome
Title The Change of Scores on the UPDRS for Blinded Assessed Motor Function 'Off' Medication and 'on' Stimulation
Hide Description Unified Parkinson’s Disease Rating Scale (UPDRS Part III) measured while the patient is off medications and on stimulation at follow-up visits post surgery. UPDRS Part III has 14 items assessing motor skills including facial expression and speech, tremors, rigidity, posture, gait, and bradykinesia. Left and right sides (arms, legs, and hands) are assessed separately for seven of the functions. A summary score ranging from 0 to 108 is generated by adding the 14 specific motor function responses. The motor function (UPDRS part III) assessments are done by turning on the stimulation with and without taking PD medications (on/off) at each in-person visit. The higher score indicates that the condition is worse.
Time Frame at six months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title 1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Hide Arm/Group Description:

Bilateral Deep Brain Stimulation

Bilateral Deep Brain Stimulation : The DBS site (STN or GPi) will be assigned on a random basis at the time the patient enters the surgical phase of the trial.

Best Medical Therapy

best medical therapy : Participants will initially be randomized to DBS or to 6 months of

Overall Number of Participants Analyzed 121 134
Mean (95% Confidence Interval)
Unit of Measure: units
-12.3
(-14.3 to -10.3)
-1.7
(-3.3 to -0.1)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Hide Arm/Group Description

Bilateral Deep Brain Stimulation

Bilateral Deep Brain Stimulation : The DBS site (STN or GPi) will be assigned on a random basis at the time the patient enters the surgical phase of the trial.

Best Medical Therapy

best medical therapy : Participants will initially be randomized to DBS or to 6 months of "best medical therapy." BMT participants will then proceed into the surgical phase of the trial. Effective 08/05/05, randomization to the BMT arm has been discontinued since the study has sufficient information to compare the outcomes of DBS and BMT patients at 6 months.

All-Cause Mortality
1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   49/121 (40.50%)      15/134 (11.19%)    
Blood and lymphatic system disorders     
Anaemia *  2/121 (1.65%)  3 0/134 (0.00%)  0
Cardiac disorders     
Angina Unstable *  1/121 (0.83%)  1 1/134 (0.75%)  1
Coronary Artery Insufficiency *  0/121 (0.00%)  0 1/134 (0.75%)  1
Cardiac Failure Congestive *  1/121 (0.83%)  1 0/134 (0.00%)  0
Coronary artery disease *  1/121 (0.83%)  1 0/134 (0.00%)  0
Coronary artery occlusion *  1/121 (0.83%)  1 0/134 (0.00%)  0
Gastrointestinal disorders     
Dysphagia *  0/121 (0.00%)  0 1/134 (0.75%)  2
Gastrooesopageal Reflux disease *  1/121 (0.83%)  1 0/134 (0.00%)  0
Inguinal hernia *  1/121 (0.83%)  1 0/134 (0.00%)  0
General disorders     
Adverse drug reaction *  0/121 (0.00%)  0 1/134 (0.75%)  1
Fatigue *  1/121 (0.83%)  1 0/134 (0.00%)  0
Lethargy *  1/121 (0.83%)  1 0/134 (0.00%)  0
Mechanical complication of implant *  1/121 (0.83%)  1 0/134 (0.00%)  0
Pyrexia *  1/121 (0.83%)  1 0/134 (0.00%)  0
Immune system disorders     
Drug Hypersensitivity *  1/121 (0.83%)  1 0/134 (0.00%)  0
Infections and infestations     
Arthritis infective *  0/121 (0.00%)  0 1/134 (0.75%)  1
Pneumonia *  1/121 (0.83%)  1 1/134 (0.75%)  1
Urinary Tract infection *  1/121 (0.83%)  1 0/134 (0.00%)  0
Injury, poisoning and procedural complications     
Fall *  6/121 (4.96%)  6 2/134 (1.49%)  2
Procedural complications *  2/121 (1.65%)  2 0/134 (0.00%)  0
Complication of device removal *  1/121 (0.83%)  1 0/134 (0.00%)  0
Device migration *  1/121 (0.83%)  1 0/134 (0.00%)  0
Medical Device complication *  1/121 (0.83%)  1 0/134 (0.00%)  0
Medical device discomfort *  1/121 (0.83%)  1 0/134 (0.00%)  0
Subdural haematoma *  1/121 (0.83%)  1 0/134 (0.00%)  0
Wound dehiscence *  1/121 (0.83%)  1 0/134 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Spinal osteoarthritis *  0/121 (0.00%)  0 1/134 (0.75%)  1
Musculoskeletal chest pain *  1/121 (0.83%)  1 0/134 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Glioma, Lung Neoplasm malignant, Prostate Cancer *  4/121 (3.31%)  4 0/134 (0.00%)  0
Nervous system disorders     
Cerebrovascular accident *  2/121 (1.65%)  2 0/134 (0.00%)  0
Dyskinesia *  2/121 (1.65%)  2 0/134 (0.00%)  0
Nervous system disorders *  15/121 (12.40%)  15 3/134 (2.24%)  3
Psychiatric disorders     
Confusional State *  3/121 (2.48%)  3 0/134 (0.00%)  0
Mental status changes *  2/121 (1.65%)  3 0/134 (0.00%)  0
Anxiety *  1/121 (0.83%)  1 1/134 (0.75%)  1
Hallucination *  1/121 (0.83%)  1 1/134 (0.75%)  1
Agitation *  1/121 (0.83%)  1 0/134 (0.00%)  0
Depression *  1/121 (0.83%)  1 0/134 (0.00%)  0
Sexual Dysfunction *  1/121 (0.83%)  1 0/134 (0.00%)  0
Reproductive system and breast disorders     
Benign prostatic hyperplasia *  0/121 (0.00%)  0 1/134 (0.75%)  1
Respiratory, thoracic and mediastinal disorders     
Chronic Obstructive Pulmonary disease, Pleuritic Pain *  1/121 (0.83%)  1 1/134 (0.75%)  1
Social circumstances     
Activities of daily living impaired *  1/121 (0.83%)  1 0/134 (0.00%)  0
Surgical and medical procedures     
Surgical Site Infection *  12/121 (9.92%)  16 0/134 (0.00%)  0
Angioplasty, Bone Graft, transurethral prostatectomy *  2/121 (1.65%)  2 1/134 (0.75%)  1
Vascular disorders     
Deep Vein Thrombosis, Orthostatic hypertension *  1/121 (0.83%)  1 1/134 (0.75%)  1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 4.7%
1: Bilateral Deep Brain Stimulation 2: Best Medical Therapy
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   116/121 (95.87%)      97/134 (72.39%)    
Gastrointestinal disorders     
Dysphagia *  32/121 (26.45%)  35 17/134 (12.69%)  19
Constipation *  23/121 (19.01%)  25 9/134 (6.72%)  9
Nausea *  14/121 (11.57%)  15 3/134 (2.24%)  3
General disorders     
Pain *  27/121 (22.31%)  35 15/134 (11.19%)  15
Incision site pain *  22/121 (18.18%)  23 0/134 (0.00%)  0
Fatigue *  11/121 (9.09%)  12 2/134 (1.49%)  2
Infections and infestations     
Implant site infection *  13/121 (10.74%)  18 0/134 (0.00%)  0
Injury, poisoning and procedural complications     
Fall *  49/121 (40.50%)  66 22/134 (16.42%)  25
Procedural pain *  19/121 (15.70%)  22 0/134 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Musculoskeletal stiffness *  23/121 (19.01%)  28 16/134 (11.94%)  18
Back pain *  16/121 (13.22%)  16 6/134 (4.48%)  6
Musculoskeletal pain *  10/121 (8.26%)  10 2/134 (1.49%)  2
Nervous system disorders     
Motor Dysfunction *  26/121 (21.49%)  29 25/134 (18.66%)  25
Dyskinesia *  37/121 (30.58%)  44 23/134 (17.16%)  23
Tremor *  30/121 (24.79%)  37 22/134 (16.42%)  23
Gait disturbance *  37/121 (30.58%)  41 21/134 (15.67%)  21
Bradykinesia *  25/121 (20.66%)  29 20/134 (14.93%)  20
Balance disorder *  22/121 (18.18%)  24 19/134 (14.18%)  19
Muscle rigidity *  24/121 (19.83%)  27 17/134 (12.69%)  17
Speech disorder *  34/121 (28.10%)  40 16/134 (11.94%)  16
Dystonia *  31/121 (25.62%)  37 15/134 (11.19%)  16
Freezing phenomenon *  31/121 (25.62%)  33 14/134 (10.45%)  14
Akinesia *  14/121 (11.57%)  18 12/134 (8.96%)  13
Headache *  40/121 (33.06%)  49 2/134 (1.49%)  2
Drooling *  14/121 (11.57%)  14 6/134 (4.48%)  6
Psychiatric disorders     
Confusional state *  33/121 (27.27%)  41 15/134 (11.19%)  15
Depression *  28/121 (23.14%)  32 15/134 (11.19%)  16
Insomnia *  20/121 (16.53%)  20 8/134 (5.97%)  8
Anxiety *  16/121 (13.22%)  18 9/134 (6.72%)  9
Skin and subcutaneous tissue disorders     
Hyperhidrosis *  18/121 (14.88%)  20 14/134 (10.45%)  14
Vascular disorders     
Orthostatic hypotension *  27/121 (22.31%)  33 16/134 (11.94%)  17
*
Indicates events were collected by non-systematic assessment
A limitation of this study is that the STN and GPi cases are pooled into a single DBS group. When the study was planned, it was thought that differences between STN and GPi at six months would be small, which was tested during the study analysis.
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: Frances M. Weaver, Ph.D, Study Investigator
Organization: Center for Management of Complex Chronic Care
Phone: (708) 202-2414
Publications of Results:
Weaver FM, Rothlind J, Stern M. Deep Brain Stimulation for Patients with Advanced Parkinson Disease Reply. [Letter to the Editor]. JAMA : the journal of the American Medical Association. 2009 May 20; 301(19):1985.
Follett K, Weaver FM, Stern M. Comment: Deep-brain stimulation for Parkinson's Disease. The New England journal of medicine. 2010 Sep 3; 363(10):988.
Responsible Party: VA Office of Research and Development ( US Department of Veterans Affairs )
ClinicalTrials.gov Identifier: NCT00056563     History of Changes
Obsolete Identifiers: NCT00101556
Other Study ID Numbers: 468 Phase I
First Submitted: March 18, 2003
First Posted: March 19, 2003
Results First Submitted: May 20, 2013
Results First Posted: July 30, 2013
Last Update Posted: May 9, 2014