Objective: To evaluate the feasibility, safety, and clinical responses of bilateral delivery, at the time of deep brain stimulation (DBS) surgery, of autologous peripheral nerve tissue (PNT) to the nucleus basalis of Meynert (NBM) or substantia nigra (SN) in individuals with Parkinson’s disease (PD).

Background: Over the last decade, our group has been carrying out a series of open-label, single-center Phase I clinical trials to examine the capability of using regenerative peripheral nerve tissue (PNT – taken from a participant’s own sural nerve) to help in the repair of damaged central nervous system cells and possibly alter or slow the progression of PD in patients undergoing DBS surgery. We are beginning a feasibility and safety double-blind, single center Phase I trial of bilaterally implanting autologous PNT into the NBM or SN in participants with a diagnosis of PD. The primary outcome is to determine our ability to recruit, enroll, execute a randomized study intervention, and follow participants with PD for 2 years under masked conditions.

Method: Twenty-four participants with idiopathic PD are being enrolled. Participants will be randomized to receive bilateral delivery of PNT to the NBM or SN immediately after DBS electrodes are implanted as part of the standard of care. Participants will be followed for 24 months to monitor safety and clinical responses including quality of life and activities of daily living.

Results: The Primary Endpoint is determining the number of participants who received bilateral, unilateral or no implantation of PNT. Additional endpoints include determining the number of participants who complete 12-month study visits, the number of adverse events and serious adverse events associated with bilateral PNT deployment, and mean change (with 95% confidence interval) in clinical outcomes (MDS-UPDRS, PDQ-8, Modified Schwab and England scale, neurocognitive exams) at 12 and 24 months compared to baseline.

Conclusion: With the completion of this study, we will better understand our capabilities and limitations of executing a larger Phase II trial designed to assess a disease-modifying strategy in patients with PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/phase-i-randomized-double-blind-study-to-evaluate-feasibility-safety-and-clinical-responses-of-implanting-autologous-peripheral-nerve-tissue-into-the-nucleus-basalis-of-meynert-or-substantia-nigra/