Nerve Tissue Implantation for Parkinson's Disease

(GUIDE Trial)

Overseen By Morgan Yazell, MBA

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Craig van Horne, MD, PhD

Disqualifiers: Previous PD surgery, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 5

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is nerve tissue implantation safe for humans?

Studies on nerve tissue implantation for Parkinson's disease show it is generally safe, with adverse events similar to standard procedures. Some participants experienced numbness at the nerve harvest site, and one had a minor infection, but no serious complications were reported. ^{1 2 3 4 5}

How does the nerve tissue implantation treatment for Parkinson's disease differ from other treatments?

This treatment involves implanting peripheral nerve tissue containing Schwann cells into the brain during deep brain stimulation surgery, aiming to restore nerve function by delivering neurotrophic factors. Unlike standard treatments, it focuses on potentially slowing disease progression by enhancing the brain's natural repair mechanisms. ^{4 5 6 7 8}

What data supports the effectiveness of the treatment Peripheral Nerve Tissue Implantation for Parkinson's Disease?

Research shows that implanting peripheral nerve grafts in Parkinson's patients during deep brain stimulation surgery may improve motor function, as seen in a study where participants had better movement scores after 12 months. This suggests the treatment could have potential benefits, although more research is needed to confirm its effectiveness. ^{4 5 6 9 10}

Who Is on the Research Team?

Craig van Horne, MD, PhD

Principal Investigator

University of Kentucky

Are You a Good Fit for This Trial?

This trial is for people aged 40-75 with Parkinson's Disease who are able to undergo surgery and assessments, specifically those getting Deep Brain Stimulation (DBS) surgery. They must be willing to donate their own nerve tissue and not have had previous PD or brain surgeries. Pregnant women or those not using birth control are excluded.

Inclusion Criteria

I am fit for surgery.

I am willing and able to undergo a specific brain scan.

I am between 40 and 75 years old.

Exclusion Criteria

I am not pregnant, breastfeeding, and if able to bear children, I agree to use birth control during the study.

Presence of typical, nonparkinsonian syndrome ioflupane/SPECT signal

My brain pathway to the substantia nigra is blocked.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preoperative Assessment

Participants are assessed preoperatively before undergoing DBS surgery and PNT delivery

1-2 weeks

Surgery and Treatment

Participants undergo DBS surgery and bilateral delivery of autologous PNT to the substantia nigra

At the time of surgery

1 visit (in-person)

Postoperative Monitoring

Participants are assessed postoperatively at two weeks after surgery for adverse events and clinical outcomes

2 weeks

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness at 6 months, 12 months, and annually thereafter

12 months

3 visits (in-person)

Long-term Follow-up

Participants are followed long term through annual visits for the rest of their lives

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Peripheral Nerve Tissue Implantation

Trial Overview The study tests the safety of implanting a patient's own Peripheral Nerve Tissue into their brain region called Substantia Nigra during DBS surgery. It's an open-label trial where participants will be monitored for one year post-surgery, then annually for life.

How Is the Trial Designed?

Group I: Implantation of peripheral nerve tissue Experimental Treatment 1 Intervention

Bilateral deployment of peripheral nerve tissue to the substantia nigra.

Who Is Running the Clinical Trial?

Craig van Horne, MD, PhD

Lead Sponsor

Trials

5

Recruited

120+

Citations

1. United States pubmed.ncbi.nlm.nih.gov

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. [2019]

2. England pubmed.ncbi.nlm.nih.gov

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. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Implantation of autologous peripheral nerve grafts into the substantia nigra of subjects with idiopathic Parkinson's disease treated with bilateral STN DBS: a report of safety and feasibility. [2019]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Clinical experience with cotransplantation of peripheral nerve and adrenal medulla in patients with Parkinson's disease. [2019]

5. Austria pubmed.ncbi.nlm.nih.gov

Towards a neural transplantation therapy for Parkinson's disease: experimental principles from animal studies. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Cell therapy in Parkinson's disease. [2018]

7. United States pubmed.ncbi.nlm.nih.gov

Transplantation of Human Neural Progenitor Cells (NPC) into Putamina of Parkinsonian Patients: A Case Series Study, Safety and Efficacy Four Years after Surgery. [2020]

8. United States pubmed.ncbi.nlm.nih.gov

Persistent dyskinesias in patients with fetal tissue transplantation for Parkinson disease. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Enhancement of the response to levodopa therapy after intrastriatal transplantation of autologous sympathetic neurons in patients with Parkinson disease. [2014]

10. United States pubmed.ncbi.nlm.nih.gov

The case for neural tissue transplantation as a treatment for Parkinson's disease. [2007]