Parkinson DiseasePhase 1/2NCT00821743
Pedunculo-Pontin Nucleus (PPN) - Deep Brain Stimulation (DBS) in Gait and Balance Disturbance in Parkinson's Disease (PPN-GAB-PARK Study)
A Phase 1/2 study testing Deep brain stimulation electrode in people with Parkinson Disease, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
Trial summary
Disturbance of posture, gait and balance usually appear several years after Parkinson's disease (PD) onset. These axial signs, generally resistant to conventional drug and non-drug treatment, can generate a loss of autonomy and traumatic complications. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- Deep brain stimulation electrode
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2010to Sep 2013
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Severe idiopathic Parkinson's disease (stage of Hoehn and Yahr ≥ 3).
- Disease duration ≥ 5 years.
- Predominant axial motor signs (gait and balance disturbance as freezing of gait, postural instability, falls) with disability in daily life, defined by a score UPDRS II 2 of item 13 or 3 of item 14 or 15.
- Axial motor signs resistant to optimal drug and non-drug treatment, including physical therapy and eventually STN-DBS (if STN-DBS is ongoing for at least 1 year and has shown no efficacy on axial motor signs).
- No significant abnormality on brain MRI.
- Non-inclusion criteria
Show all 11Show fewer
- Patient with non-axial, dopa-responsive motor signs and indication of first line STN-DBS. In case of absence of efficacy of STN-DBS on axial motor signs after one year of stimulation, PPN-DBS may be proposed to these patients.
- Patient with major cognitive disturbances defined by a score Mattis DRS 130 (of 144).
- Pregnant or breast-feeding woman.
- Patient with contraindication for general anaesthesia, a contraindication for surgery (including disorders of haemostasis, or necessity of anticoagulant and antiaggregant drug), or a contraindication for MRI.
- Patient presenting major depression, psychosis or addiction in six months preceding inclusion.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Neurology, Neurosugery of Nice University Hospital
Nice, 06000
Denys Fontaine, PhD, 04 92 03 84 50, fontaine.d@chu-nice.fr
Caroline Bayreuther, PhD, 04 92 03 78 96, bayreuther.c@chu-nice.fr
About this study
Official title: Study of Deep Brain Stimulation of the Pedunculo-pontin Nucleus (PPN) for the Treatment of Gait and Balance Disturbances in Patients With Parkinson's Disease (PPN-GAB-PARK Study)
Disturbance of posture, gait and balance usually appear several years after Parkinson's disease (PD) onset. These axial signs, generally resistant to conventional drug and non-drug treatment, can generate a loss of autonomy and traumatic complications. Improvement of these axial signs by deep brain stimulation (DBS) of the pedunculo-pontin nucleus (PPN) has been described for a small number of patients. The objectives of the present prospective monocentric pilot study are to determine the optimal stimulation parameters of PPN-DBS, to evaluate the long term tolerance and the effect of PPN-DBS on axial signs and quality of life in five patients with severe PD. The use of detailed clinical rating scales and quantitative computer-based gait analysis will allow to identify the optimal stimulation parameters and relevant clinical efficacy parameters, mandatory for the design of further comparative large-scale studies.
Read the detailed description
Parkinson's disease (PD) is a neurodegenerative disorder with an association of akinesia, rigidity and tremor, but also so-called "axial" motor signs (disturbance of posture, gait and balance) that usually appear after several years of disease evolution. These axial signs, generally resistant to drug treatment and deep brain stimulation (DBS) of subthalamic nucleus (STN), can generate a loss of autonomy and traumatic complications due to frequent falls. An improvement of axial signs by DBS of the pedunculo-pontin nucleus (PPN) has been described for a small number of patients. Nevertheless, this effect needs to be confirmed in more patients and there is need for complementary knowledge, including optimal stimulation modalities.
The primary objective of the current prospective monocentric pilot study is to determine the optimal stimulation parameters (site of stimulation, frequency, amplitude) of PPN-DBS in patients with severe PD. Secondary objectives are 1) to confirm the effect of PPN-DBS on disturbance of posture, balance and gait; 2) to evaluate long term tolerance of this technique; 3) to determine the components of gait that are most improved by PPN-DBS; 4) to evaluate unilateral versus bilateral stimulation; 5) to evaluate the effects of PPN-DBS on other PD symptoms; 6) to determine the impact of PPN-DBS on quality of life. Five patients fulfilling the following criteria, will be included: age between 18 and 70 years, idiopathic PD for more than 5 years, predominant axial motor signs (disturbance of gait and balance, falls, freezing of gait) responsible for disability in daily living and resistant to optimal treatment.
The DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic). After the surgery, the study will comprise two phases: a 6-month phase dedicated to optimisation of the stimulation parameters (including systematic evaluation of defined parameters); followed by a phase of evaluation, that will compare, in a double-blind setting, 4 conditions (right unilateral, left unilateral, bilateral and off stimulation). Efficacy of PPN-DBS will be evaluated by clinical rating scales quantifying motor disturbance, cognitive function and quality of life, and by the means of computer-based measure of gait and balance (system Gaitrite and Vicon).
Expected results are 1) an improvement of gait, postural and balance disturbance, due to PPN-DBS. 2) Identification of the optimal PPN-DBS parameters, improving most the axial PD symptoms, will allow to abbreviate the adjustment period, by using as default setting these parameters when turning on the stimulator for the first time; 3) Identification of the axial signs which are the most improved will allow to select patients that could most benefit from PPN-DBS 4) The use of diverse specific rating scales and quantitative gait analysis will allow to determine the most relevant clinical criteria to be evaluated in further studies.
Thus, this project will allow to obtain data (selection criteria, relevant rating scales, optimal stimulation parameters) mandatory for the construction of further comparative large-scale studies, if the efficacy of PPN-DBS was confirmed by the present study.
- Conditions
- Parkinson Disease
- Being tested
- Deep brain stimulation electrode
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deep brain stimulation electrodeDevice
The DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic).
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | The DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic). Receives: Device: Deep brain stimulation electrode |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary endpoint will be the optimal parameters of PPN-DBS, defined as those leading to the best improvement of posture, balance and gait disturbance
Measured: at day 0, day 7, 6 months, year 1 and year 2
Secondary outcomes (6)
Efficacy of PPN-DBS on disturbance of posture, balance and gait
Measured: at day 0, day 7, 6 months, year 1 and year 2
Long term tolerance will be assessed by general and neurological examination, neuro-psychological tests (MMS, Mattis-DRS, Score fontal, BREF, MADRS) and by assessment of adverse events;
Measured: at day 0, day 7, 6 months, year 1 and year 2
the components of gait that are most improved by PPN-DBS will be determined using computer-based systems (GAITRite and VICON);
Measured: at day 0, day 7, 6 months, year 1 and year 2
evaluation of unilateral versus bilateral stimulation will be done during the double-blind assessment of all efficacy criteria;
Measured: at day 0, day 7, 6 months, year 1 and year 2
effects of PPN-DBS on other PD symptom will be assessed using UPDRS and Hoehn and Yahr's staging;
Measured: at day 0, day 7, 6 months, year 1 and year 2
the impact of PPN-DBS on quality of life and activities of daily living will be assessed by UPDRS II, SF36 and PDQ39 questionnaires and by a global satisfaction scale.
Measured: at day 0, day 7, 6 months, year 1 and year 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2009 (estimated)
- Study startMar 2010
- Primary completionSep 2013 (estimated)
- Study completionSep 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Denys FONTAINE, PhD, Principal investigator, Department of neurosurgery, CHU de Nice
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00821743
- Sponsor's protocol ID
- PPN-GAB-PARK study
- Status last verified
- Mar 2010
- First submitted
- 12 Jan 2009
- Last updated
- 24 Apr 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Deep Brain Stimulation of the Pedunculo-pontin Nucleus (PPN) for the Treatment of Gait and Balance Disturbances in Patients With Parkinson's Disease (PPN-GAB-PARK Study)
- ClinicalTrials.gov ID
- NCT00821743
- Acronym / sponsor ID
- PPN-GAB-PARK study
- Conditions
- Parkinson Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Mar 2010
- Primary completion
- Sep 2013 (estimated)
- Study completion
- Sep 2013 (estimated)
- First posted
- 13 Jan 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Disturbance of posture, gait and balance usually appear several years after Parkinson's disease (PD) onset. These axial signs, generally resistant to conventional drug and non-drug treatment, can generate a loss of autonomy and traumatic complications. Improvement of these axial signs by deep brain stimulation (DBS) of the pedunculo-pontin nucleus (PPN) has been described for a small number of patients. The objectives of the present prospective monocentric pilot study are to determine the optimal stimulation parameters of PPN-DBS, to evaluate the long term tolerance and the effect of PPN-DBS on axial signs and quality of life in five patients with severe PD. The use of detailed clinical rating scales and quantitative computer-based gait analysis will allow to identify the optimal stimulation parameters and relevant clinical efficacy parameters, mandatory for the design of further comparative large-scale studies.
Detailed description
Parkinson's disease (PD) is a neurodegenerative disorder with an association of akinesia, rigidity and tremor, but also so-called "axial" motor signs (disturbance of posture, gait and balance) that usually appear after several years of disease evolution. These axial signs, generally resistant to drug treatment and deep brain stimulation (DBS) of subthalamic nucleus (STN), can generate a loss of autonomy and traumatic complications due to frequent falls. An improvement of axial signs by DBS of the pedunculo-pontin nucleus (PPN) has been described for a small number of patients. Nevertheless, this effect needs to be confirmed in more patients and there is need for complementary knowledge, including optimal stimulation modalities.
The primary objective of the current prospective monocentric pilot study is to determine the optimal stimulation parameters (site of stimulation, frequency, amplitude) of PPN-DBS in patients with severe PD. Secondary objectives are 1) to confirm the effect of PPN-DBS on disturbance of posture, balance and gait; 2) to evaluate long term tolerance of this technique; 3) to determine the components of gait that are most improved by PPN-DBS; 4) to evaluate unilateral versus bilateral stimulation; 5) to evaluate the effects of PPN-DBS on other PD symptoms; 6) to determine the impact of PPN-DBS on quality of life. Five patients fulfilling the following criteria, will be included: age between 18 and 70 years, idiopathic PD for more than 5 years, predominant axial motor signs (disturbance of gait and balance, falls, freezing of gait) responsible for disability in daily living and resistant to optimal treatment.
The DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic). After the surgery, the study will comprise two phases: a 6-month phase dedicated to optimisation of the stimulation parameters (including systematic evaluation of defined parameters); followed by a phase of evaluation, that will compare, in a double-blind setting, 4 conditions (right unilateral, left unilateral, bilateral and off stimulation). Efficacy of PPN-DBS will be evaluated by clinical rating scales quantifying motor disturbance, cognitive function and quality of life, and by the means of computer-based measure of gait and balance (system Gaitrite and Vicon).
Expected results are 1) an improvement of gait, postural and balance disturbance, due to PPN-DBS. 2) Identification of the optimal PPN-DBS parameters, improving most the axial PD symptoms, will allow to abbreviate the adjustment period, by using as default setting these parameters when turning on the stimulator for the first time; 3) Identification of the axial signs which are the most improved will allow to select patients that could most benefit from PPN-DBS 4) The use of diverse specific rating scales and quantitative gait analysis will allow to determine the most relevant clinical criteria to be evaluated in further studies.
Thus, this project will allow to obtain data (selection criteria, relevant rating scales, optimal stimulation parameters) mandatory for the construction of further comparative large-scale studies, if the efficacy of PPN-DBS was confirmed by the present study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Device: Deep brain stimulation electrodeThe DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Severe idiopathic Parkinson's disease (stage of Hoehn and Yahr ≥ 3).
- Disease duration ≥ 5 years.
- Predominant axial motor signs (gait and balance disturbance as freezing of gait, postural instability, falls) with disability in daily life, defined by a score UPDRS II 2 of item 13 or 3 of item 14 or 15.
- Axial motor signs resistant to optimal drug and non-drug treatment, including physical therapy and eventually STN-DBS (if STN-DBS is ongoing for at least 1 year and has shown no efficacy on axial motor signs).
- No significant abnormality on brain MRI.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deep brain stimulation electrodeThe DBS electrodes (model 3389, Medtronic) will be stereotactically implanted bilaterally in the PPN, according to the technique usually used for STN-DBS, and connected to the subcutaneously implanted stimulator (Kinetra, Medtronic). | Device | 1 |
Primary outcome
Primary endpoint will be the optimal parameters of PPN-DBS, defined as those leading to the best improvement of posture, balance and gait disturbance
Time frame: at day 0, day 7, 6 months, year 1 and year 2
6 secondary outcomes
Efficacy of PPN-DBS on disturbance of posture, balance and gait
Time frame: at day 0, day 7, 6 months, year 1 and year 2
Long term tolerance will be assessed by general and neurological examination, neuro-psychological tests (MMS, Mattis-DRS, Score fontal, BREF, MADRS) and by assessment of adverse events;
Time frame: at day 0, day 7, 6 months, year 1 and year 2
the components of gait that are most improved by PPN-DBS will be determined using computer-based systems (GAITRite and VICON);
Time frame: at day 0, day 7, 6 months, year 1 and year 2
evaluation of unilateral versus bilateral stimulation will be done during the double-blind assessment of all efficacy criteria;
Time frame: at day 0, day 7, 6 months, year 1 and year 2
effects of PPN-DBS on other PD symptom will be assessed using UPDRS and Hoehn and Yahr's staging;
Time frame: at day 0, day 7, 6 months, year 1 and year 2
the impact of PPN-DBS on quality of life and activities of daily living will be assessed by UPDRS II, SF36 and PDQ39 questionnaires and by a global satisfaction scale.
Time frame: at day 0, day 7, 6 months, year 1 and year 2
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Denys FONTAINE, PhD principal investigator
Department of neurosurgery, CHU de Nice
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Deep brain stimulation electrode in people with Parkinson Disease, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Denys FONTAINE, PhD (04 92 03 84 50, fontaine.d@chu-nice.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary endpoint will be the optimal parameters of PPN-DBS, defined as those leading to the best improvement of posture, balance and gait disturbance (time frame: at day 0, day 7, 6 months, year 1 and year 2). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2013.