Feasibility and Safety of autoloGous UncondItioneD pEripheral Nerve Tissue Delivery to the Substantia Nigra (GUIDE)

Craig van Horne, MD, PhD

Status and phase

Active, not recruiting

Phase 1

Conditions

Parkinson's Disease

Treatments

Procedure: Peripheral Nerve Tissue Implantation

Study type

Interventional

Funder types

Other

Identifiers

NCT05377281

76436

Details and patient eligibility

About

This is an open-label, non-randomized, single-arm trial design to actively follow participants for 12 months. Ten participants will be enrolled to receive bilateral delivery of Peripheral Nerve Tissue (PNT) to the Substantia Nigra at the time of Deep Brain Stimulation (DBS) surgery. After 12 months, participants will be followed long term through annual visits for the rest of their lives. Participants will serve as their own donor for the tissue.

Full description

We recently designed open-label safety studies to investigate the use of an autologous source of cellular tissue from the peripheral nervous system (van Horne et al., 2017, 2018; Quintero et al. 2022), reparative peripheral (sural) nerve tissue containing a variety of cells and neuroprotective factors (Chau et al. 2022). While our previous studies have used injury-activated peripheral nerve tissue (PNT), where the sural nerve is transected and allowed to regenerate in situ for up to about 2 weeks before implantation, here we propose to use naïve, previously uninjured, nerve tissue delivered bilaterally to the substantia nigra at the time participants are undergoing deep brain stimulation (DBS) surgery. Participants will be assessed preoperatively, then undergo DBS surgery and delivery of autologous PNT, and then assessed postoperatively at two weeks after surgery, 6 months, 12 months and annually thereafter for adverse events and clinical outcomes.

Enrollment

10 estimated patients

Sex

All

Ages

40 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Undergoing DBS
Diagnosis of clinically established or clinically probably PD (Parkinson's disease) as defined by MDS criteria
Age 40-75, inclusive
Able and willing to undergo ioflupane/SPECT
Able to tolerate the surgical procedure
Able to undergo all planned assessments
Available access to the sural nerve

Exclusion criteria

Any condition that would not make the subject a candidate for DBS
Previous PD surgery or intracranial surgery
Typical, nonparkinsonian syndrome ioflupane/SPECT signal
Female who is pregnant, lactating, or of child-bearing potential unwilling to use an adequate birth control method during the period of the study
Unable to undergo an MRI
An obstructed trajectory path to the substantia nigra