Trial Outcomes & Findings for Phase I Deep Brain Stimulation (DBS) vs. Best Medical Therapy (BMT) Trial (NCT NCT00056563)
NCT ID: NCT00056563
Last Updated: 2014-05-09
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
255 participants
Primary outcome timeframe
at six months
Results posted on
2014-05-09
Participant Flow
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.
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.
Participant milestones
| Measure |
1: Bilateral Deep Brain Stimulation
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.
|
2: Best Medical Therapy
Best Medical Therapy
best medical therapy : Participants will initially be randomized to DBS or to 6 months of
|
|---|---|---|
|
Overall Study
STARTED
|
121
|
134
|
|
Overall Study
COMPLETED
|
121
|
134
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase I Deep Brain Stimulation (DBS) vs. Best Medical Therapy (BMT) Trial
Baseline characteristics by cohort
| Measure |
1: Bilateral Deep Brain Stimulation
n=121 Participants
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.
|
2: Best Medical Therapy
n=134 Participants
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
n=255 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
90 Participants
n=5 Participants
|
97 Participants
n=7 Participants
|
187 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
31 Participants
n=5 Participants
|
37 Participants
n=7 Participants
|
68 Participants
n=5 Participants
|
|
Age, Continuous
|
62.4 years
STANDARD_DEVIATION 8.8 • n=5 Participants
|
62.3 years
STANDARD_DEVIATION 9.0 • n=7 Participants
|
62.3 years
STANDARD_DEVIATION 8.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
23 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
47 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
98 Participants
n=5 Participants
|
110 Participants
n=7 Participants
|
208 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
121 participants
n=5 Participants
|
134 participants
n=7 Participants
|
255 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: at six monthsOutcome measures
| Measure |
1: Bilateral Deep Brain Stimulation
n=121 Participants
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.
|
2: Best Medical Therapy
n=134 Participants
Best Medical Therapy
best medical therapy : Participants will initially be randomized to DBS or to 6 months of
|
|---|---|---|
|
The Difference of Time Spent in the 'on' State Without Troublesome Dyskinesia Based on Patient Motor Diaries as Compared to the Baseline.
|
4.6 Hours per day
Interval 3.8 to 5.3
|
0 Hours per day
Interval -0.5 to 0.5
|
SECONDARY outcome
Timeframe: at six monthsUnified 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.
Outcome measures
| Measure |
1: Bilateral Deep Brain Stimulation
n=121 Participants
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.
|
2: Best Medical Therapy
n=134 Participants
Best Medical Therapy
best medical therapy : Participants will initially be randomized to DBS or to 6 months of
|
|---|---|---|
|
The Change of Scores on the UPDRS for Blinded Assessed Motor Function 'Off' Medication and 'on' Stimulation
|
-12.3 units
Interval -14.3 to -10.3
|
-1.7 units
Interval -3.3 to -0.1
|
Adverse Events
1: Bilateral Deep Brain Stimulation
Serious events: 49 serious events
Other events: 116 other events
Deaths: 0 deaths
2: Best Medical Therapy
Serious events: 15 serious events
Other events: 97 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
1: Bilateral Deep Brain Stimulation
n=121 participants at risk
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.
|
2: Best Medical Therapy
n=134 participants at risk
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.
|
|---|---|---|
|
Surgical and medical procedures
Surgical Site Infection
|
9.9%
12/121 • Number of events 16
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Fall
|
5.0%
6/121 • Number of events 6
|
1.5%
2/134 • Number of events 2
|
|
Psychiatric disorders
Confusional State
|
2.5%
3/121 • Number of events 3
|
0.00%
0/134
|
|
Blood and lymphatic system disorders
Anaemia
|
1.7%
2/121 • Number of events 3
|
0.00%
0/134
|
|
Nervous system disorders
Cerebrovascular accident
|
1.7%
2/121 • Number of events 2
|
0.00%
0/134
|
|
Nervous system disorders
Dyskinesia
|
1.7%
2/121 • Number of events 2
|
0.00%
0/134
|
|
Psychiatric disorders
Mental status changes
|
1.7%
2/121 • Number of events 3
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Procedural complications
|
1.7%
2/121 • Number of events 2
|
0.00%
0/134
|
|
General disorders
Adverse drug reaction
|
0.00%
0/121
|
0.75%
1/134 • Number of events 1
|
|
Cardiac disorders
Angina Unstable
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Surgical and medical procedures
Angioplasty, Bone Graft, transurethral prostatectomy
|
1.7%
2/121 • Number of events 2
|
0.75%
1/134 • Number of events 1
|
|
Psychiatric disorders
Anxiety
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Infections and infestations
Arthritis infective
|
0.00%
0/121
|
0.75%
1/134 • Number of events 1
|
|
Reproductive system and breast disorders
Benign prostatic hyperplasia
|
0.00%
0/121
|
0.75%
1/134 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary disease, Pleuritic Pain
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Vascular disorders
Deep Vein Thrombosis, Orthostatic hypertension
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Gastrointestinal disorders
Dysphagia
|
0.00%
0/121
|
0.75%
1/134 • Number of events 2
|
|
Psychiatric disorders
Hallucination
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Infections and infestations
Pneumonia
|
0.83%
1/121 • Number of events 1
|
0.75%
1/134 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
|
0.00%
0/121
|
0.75%
1/134 • Number of events 1
|
|
Cardiac disorders
Coronary Artery Insufficiency
|
0.00%
0/121
|
0.75%
1/134 • Number of events 1
|
|
Cardiac disorders
Cardiac Failure Congestive
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Cardiac disorders
Coronary artery disease
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Cardiac disorders
Coronary artery occlusion
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Gastrointestinal disorders
Gastrooesopageal Reflux disease
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Gastrointestinal disorders
Inguinal hernia
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
General disorders
Fatigue
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
General disorders
Lethargy
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
General disorders
Mechanical complication of implant
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
General disorders
Pyrexia
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Immune system disorders
Drug Hypersensitivity
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Infections and infestations
Urinary Tract infection
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Complication of device removal
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Device migration
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Medical Device complication
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Medical device discomfort
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Injury, poisoning and procedural complications
Wound dehiscence
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Glioma, Lung Neoplasm malignant, Prostate Cancer
|
3.3%
4/121 • Number of events 4
|
0.00%
0/134
|
|
Nervous system disorders
Nervous system disorders
|
12.4%
15/121 • Number of events 15
|
2.2%
3/134 • Number of events 3
|
|
Psychiatric disorders
Agitation
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Psychiatric disorders
Depression
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Psychiatric disorders
Sexual Dysfunction
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
|
Social circumstances
Activities of daily living impaired
|
0.83%
1/121 • Number of events 1
|
0.00%
0/134
|
Other adverse events
| Measure |
1: Bilateral Deep Brain Stimulation
n=121 participants at risk
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.
|
2: Best Medical Therapy
n=134 participants at risk
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.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Fall
|
40.5%
49/121 • Number of events 66
|
16.4%
22/134 • Number of events 25
|
|
Nervous system disorders
Motor Dysfunction
|
21.5%
26/121 • Number of events 29
|
18.7%
25/134 • Number of events 25
|
|
Nervous system disorders
Dyskinesia
|
30.6%
37/121 • Number of events 44
|
17.2%
23/134 • Number of events 23
|
|
Nervous system disorders
Tremor
|
24.8%
30/121 • Number of events 37
|
16.4%
22/134 • Number of events 23
|
|
Nervous system disorders
Gait disturbance
|
30.6%
37/121 • Number of events 41
|
15.7%
21/134 • Number of events 21
|
|
Nervous system disorders
Bradykinesia
|
20.7%
25/121 • Number of events 29
|
14.9%
20/134 • Number of events 20
|
|
Nervous system disorders
Balance disorder
|
18.2%
22/121 • Number of events 24
|
14.2%
19/134 • Number of events 19
|
|
Gastrointestinal disorders
Dysphagia
|
26.4%
32/121 • Number of events 35
|
12.7%
17/134 • Number of events 19
|
|
Nervous system disorders
Muscle rigidity
|
19.8%
24/121 • Number of events 27
|
12.7%
17/134 • Number of events 17
|
|
Nervous system disorders
Speech disorder
|
28.1%
34/121 • Number of events 40
|
11.9%
16/134 • Number of events 16
|
|
Vascular disorders
Orthostatic hypotension
|
22.3%
27/121 • Number of events 33
|
11.9%
16/134 • Number of events 17
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
19.0%
23/121 • Number of events 28
|
11.9%
16/134 • Number of events 18
|
|
Psychiatric disorders
Confusional state
|
27.3%
33/121 • Number of events 41
|
11.2%
15/134 • Number of events 15
|
|
Nervous system disorders
Dystonia
|
25.6%
31/121 • Number of events 37
|
11.2%
15/134 • Number of events 16
|
|
Psychiatric disorders
Depression
|
23.1%
28/121 • Number of events 32
|
11.2%
15/134 • Number of events 16
|
|
General disorders
Pain
|
22.3%
27/121 • Number of events 35
|
11.2%
15/134 • Number of events 15
|
|
Nervous system disorders
Freezing phenomenon
|
25.6%
31/121 • Number of events 33
|
10.4%
14/134 • Number of events 14
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
14.9%
18/121 • Number of events 20
|
10.4%
14/134 • Number of events 14
|
|
Nervous system disorders
Akinesia
|
11.6%
14/121 • Number of events 18
|
9.0%
12/134 • Number of events 13
|
|
Nervous system disorders
Headache
|
33.1%
40/121 • Number of events 49
|
1.5%
2/134 • Number of events 2
|
|
Gastrointestinal disorders
Constipation
|
19.0%
23/121 • Number of events 25
|
6.7%
9/134 • Number of events 9
|
|
General disorders
Incision site pain
|
18.2%
22/121 • Number of events 23
|
0.00%
0/134
|
|
Psychiatric disorders
Insomnia
|
16.5%
20/121 • Number of events 20
|
6.0%
8/134 • Number of events 8
|
|
Injury, poisoning and procedural complications
Procedural pain
|
15.7%
19/121 • Number of events 22
|
0.00%
0/134
|
|
Psychiatric disorders
Anxiety
|
13.2%
16/121 • Number of events 18
|
6.7%
9/134 • Number of events 9
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
13.2%
16/121 • Number of events 16
|
4.5%
6/134 • Number of events 6
|
|
Nervous system disorders
Drooling
|
11.6%
14/121 • Number of events 14
|
4.5%
6/134 • Number of events 6
|
|
Gastrointestinal disorders
Nausea
|
11.6%
14/121 • Number of events 15
|
2.2%
3/134 • Number of events 3
|
|
Infections and infestations
Implant site infection
|
10.7%
13/121 • Number of events 18
|
0.00%
0/134
|
|
General disorders
Fatigue
|
9.1%
11/121 • Number of events 12
|
1.5%
2/134 • Number of events 2
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
8.3%
10/121 • Number of events 10
|
1.5%
2/134 • Number of events 2
|
Additional Information
Frances M. Weaver, Ph.D, Study Investigator
Center for Management of Complex Chronic Care
Phone: (708) 202-2414
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER