A Study to Evaluate Feasibility, Safety, and Clinical Responses of Implanting Autologous Peripheral Nerve Tissue Into the Brain for Non-motor or Motor Symptoms in Patients With Parkinson's Disease Undergoing DBS Surgery

NCT06683378 | Recruiting Phase 1 DMC

• 2 other identifiers: 900661R01AG081356-01A1
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The investigators propose a Phase I single surgical-center, double-blinded randomized parallel clinical trial involving bilateral autologous peripheral nerve tissue (PNT) delivery into the NBM or the alternate target also affecting cognition in this population, the substantia nigra (SN), to address "repair cell" support of these areas. Twenty-four participants with idiopathic Parkinson's Disease (PD) who have selected, qualified and agreed to receive as standard of care deep brain stimulation (DBS) will be enrolled and randomly allocated to receive bilateral PNT deployment to either the NBM or SN at the time of DBS surgery. Participants will be allocated equally among both assignments over the course of three years (8 Year 1, 10 Year 2, 6 Year 3). Participants will be evaluated for neurocognitive, motoric function, activities of daily living, and quality of life at enrollment before surgery, two-weeks after surgery, and 6, 12, and 24 months after surgery.

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; Deep Brain Stimulation; DBS; Cell and Tissue Based Therapy

Outcome Measures

Primary Outcomes (4)

• Successful deployment of bilateral peripheral nerve tissue (PNT) into the brain

  Number of participants in each study arm who successfully receive bilateral PNT delivery.

  Intraoperative

• Number of participants completing 12 month study visit

  Total number of participants to complete study visit by arm assignment

  12-month study visit

• Study-related adverse events as assessed by MedDRA v27

  Total number of study-related adverse events experienced by participants.

  Enrollment to 24-month study visit

• Study-related serious adverse events as assessed by MedDRA v.27

  Total number of serious adverse events experienced by participants

  Enrollment to 24-month study visit

Secondary Outcomes (14)

• PNT deployment attempts

  During surgery

• Mean change in Montreal Cognitive Assessment (MoCA) scores

  Baseline 6, 12 and 24 months after surgery

• Mean change in Neuropsychological assessment scores

  Baseline, 12 and 24 months

• Change in Neuropsychological diagnosis

  Baseline, 12 and 24 months

• Change in Neuropsychological domains with impairment

  At 12, 24 months compared to baseline

Study Arms (2)

Peripheral nerve tissue (PNT) deployment to the Substantia Nigra

ACTIVE COMPARATOR

Procedure: Reparative Autologous peripheral nerve tissue

Peripheral nerve tissue (PNT) deployment to the nucleus basalis of Meynert

ACTIVE COMPARATOR

Procedure: Reparative Autologous peripheral nerve tissue

Interventions

Reparative Autologous peripheral nerve tissue PROCEDURE

At the time participants are receiving the standard of care deep brain stimulation (DBS) surgery, a standard incision on the lateral aspect near the ankle is made, the sural nerve is identified, an about 3 cm biopsy of the sural nerve is obtained and the incision is closed. From the biopsied section, the epineurium is removed, fascicles are cut, and (\~5 pieces per side; \~ 5mm length x 1.5 mm diameter: approximately 10 cubic millimeters) implanted bilaterally into the nucleus basalis of Meynert (NBM) or substantia nigra (SN).

Peripheral nerve tissue (PNT) deployment to the Substantia Nigra; Peripheral nerve tissue (PNT) deployment to the nucleus basalis of Meynert

Eligibility Criteria

• Age: 45 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Undergoing DBS
• Diagnosis of clinically established or clinically probably PD as defined by MDS criteria
• Age 45-75, inclusive
• Able to tolerate the surgical procedure
• Able to undergo all planned assessments
• Available access to the sural nerve

You may not qualify if:

• Any condition that would not make the subject a candidate for DBS
• Dementia diagnosis
• Previous PD surgery or intracranial surgery
• Unable to undergo an MRI
• An obstructed trajectory path to the substantia nigra and nucleus basalis of Meynert

Sponsors & Collaborators

• Craig van Horne, MD, PhD: lead
• National Institute on Aging (NIA): collaborator

Study Sites (1)

University of Kentucky

Lexington, Kentucky, 40536, United States

RECRUITING

Related Publications (3)

• Quintero JE, Chau MJ, Slevin JT, Koehl L, Gurwell JA, Wallace E, Kryscio RJ, El Khouli R, Anderson-Mooney AJ, Schmitt FA, Gerhardt GA, van Horne CG. Two-year feasibility and safety of open-label autologous peripheral nerve tissue implantation during deep brain stimulation in patients with Parkinson's disease. J Parkinsons Dis. 2025 Mar;15(2):397-408. doi: 10.1177/1877718X241312409. Epub 2025 Feb 25.

  PMID: 40007169 BACKGROUND

• van Horne CG, Quintero JE, Slevin JT, Anderson-Mooney A, Gurwell JA, Welleford AS, Lamm JR, Wagner RP, Gerhardt GA. Peripheral nerve grafts implanted into the substantia nigra in patients with Parkinson's disease during deep brain stimulation surgery: 1-year follow-up study of safety, feasibility, and clinical outcome. J Neurosurg. 2018 Dec 1;129(6):1550-1561. doi: 10.3171/2017.8.JNS163222. Epub 2018 Feb 18.

  PMID: 29451447 BACKGROUND

• Quintero JE, Slevin JT, Gurwell JA, McLouth CJ, El Khouli R, Chau MJ, Guduru Z, Gerhardt GA, van Horne CG. Direct delivery of an investigational cell therapy in patients with Parkinson's disease: an interim analysis of feasibility and safety of an open-label study using DBS-Plus clinical trial design. BMJ Neurol Open. 2022 Jul 14;4(2):e000301. doi: 10.1136/bmjno-2022-000301. eCollection 2022.

  PMID: 35949912 BACKGROUND

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Craig G van Horne, MD, PhD

  University of Kentucky

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jaimie Hixson

CONTACT

8593231908; jaimie.henderson@uky.edu

Group Monitored Email

CONTACT

nervegraft@uky.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Department of Neurosurgery Chair

Study Record Dates

• First Submitted: November 7, 2024
• First Posted: November 12, 2024
• Study Start: July 21, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): February 28, 2028
• Last Updated: August 14, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share

Locations

US (1)