Convection Enhanced Delivery of CSF in DBS for Parkinson's

Not Applicable

Completed

• Conditions: Parkinson Disease

• Registration Number: NCT03540134
• Lead Sponsor: Jeff Elias, MD

Brief Summary

This study will evaluate safety foremost but also the distribution and initial effectiveness of infusion-enhanced, MRI-guided DBS for patients with medication-refractory, Parkinson's disease

Detailed Description

This is an open-label, single-arm, pilot study investigating the safety and feasibility of infusion-enhanced, MRI-guided DBS electrode placement. The investigators intend to enroll patients with Parkinson's disease and medically-refractory motor symptoms, who are already planned for MRI-guided DBS electrodes under general anesthesia. The hypothesis of the study is that a convective micro-infusion of autologous CSF will enhance the T2-weighted MRI visualization of the targeted nucleus during image-guided DBS surgery for Parkinson's disease. The investigators will record standard clinical measures of PD at baseline and 6 months following DBS surgery. The study will recruit patients at a rate of approximately one a month and will take less than two years to complete.

Study Timeline

• Study Start: 2018-02-13
• Study First Submitted: 2018-04-11
• Study First Submitted QC: 2018-05-25
• Study First Posted: 2018-05-30
• Primary Completion: 2018-12-18
• Study Completion: 2018-12-18
• Results First Submitted: 2020-11-18
• Status Verified: 2021-03
• Results First Submitted QC: 2021-03-16
• Last Update Submitted: 2021-03-16
• Results First Posted: 2021-04-08
• Last Update Posted: 2021-04-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

1. Men and women, age 30 years and older
2. Subjects with advanced PD who are scheduled for MRI-guided DBS surgery under general anesthesia
3. Subjects who are able and willing to give informed consent and able to attend clinic visits through 6 months
4. The target nucleus, GPi or STN, is visible on MRI so that it can be targeted for the study infusion and then for MRI-guided DBS

Exclusion Criteria

1. DBS surgery planned in the awake condition with microelectrode recordings and clinical testing
2. Spinal pathology not amenable to lumbar puncture
3. Subjects who have had deep brain stimulation or a prior stereotactic ablation of the basal ganglia
4. Legal incapacity or limited legal capacity as determined by the neuropsychologist
5. Are participating or have participated in another clinical trial in the last 30 days
6. Any illness that in the investigator's opinion preclude participation in this study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events That Are Related to Treatment	6 months postoperatively	Safety will be determined by an evaluation of the incidence and severity of infusion-related side effects and complications from the first treatment day visit through the 6-month post-treatment time point. All AEs will be reported and categorized by investigators as definitely, probably, possibly, unlikely, or unrelated to the CSF infusion, and/or Parkinson's disease progression. Safety will be assessed by incidence and severity of AEs, including changes in physical examinations and neurological examinations, and the number of discontinuations due to AEs.

Secondary Outcome Measures

Name	Time	Method
% Change in Un-medicated UPDRS, Motor Subsection (Part III) From Baseline to 6 Months	6 months postoperatively in comparison to baseline	The efficacy endpoint will be focused on determining the degree of change in motor symptoms as measured from the in-medicated UPDRS, motor subsection (Part III).
Change in Quality of Life Assessment With PDQ-39 Questionnaire	6 months postoperatively in comparison to baseline	The PDQ-39 questionnaire includes 39 quality of life questions rated on a scale of 0-4. 0 indicating least frequent (never) and 4 indicating most frequent (always). The mean change in the sum of the PDQ-39 questionnaire is being reported with a overall score of 0 being the best quality of life and a maximum score of 156 being the worst possible quality of life.
Vd/Vi Ratio	Day of Surgery	The ratio of the volume of distribution (Vd) immediately following the infusion (Vi) with intraoperative 1.5T MRI, T2-weighted sequences to the volume of the infusion (Vi) will be calculated. Each volume is measured in microliters (uL). The distribution of the infusion by convective properties in a deep brain human nucleus (GPi or STN) will be determined by calculating the Vd/Vi ratio.
Baseline Levodopa Medication Equivalents (Milligrams)	Baseline

Trial Locations

Locations (1)

University of Virginia; 🇺🇸 Charlottesville, Virginia, United States