Health TrialsbyHealthPanel

Autologous suraL nervE Grafting to the Substantia nigrA in Patients With Synuclienopathies

ConditionsMultiple System AtrophyParkinsons Disease
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age40-75
SponsorCraig van Horne, MD, PhD

About this trial

This phase I double-blind study focuses on the safety and feasibility of implanting autologous peripheral nerve tissue (PNT) into the substantia nigra area of the brain in persons who have been diagnosed with either Parkinson's disease (PD) or Multiple System Atrophy (MSA). 7 participants will be enrolled, with 4 participants receiving the graft and 3 receiving a sham surgery. Eligible participants will be early in their diagnosis with a lower burden of symptoms. Participants will be followed initially for one year after surgery.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis of clinically established or clinically probable PD or MSA as defined by MDS criteria

Disease duration greater than 2 years

Age 40-75, inclusive

MDS-Unified Parkinson's Disease Rating Scale (UPDRS) Part III greater than or equal to 20 points but less than or equal to 35 points, off anti-parkinsonian medication for PD or MDS-Unified Multiple System Atrophy Rating Scale (UMSARS) less than or equal to 30 points off anti-parkinsonian medication

Disqualifiers

Previous PD surgery or intracranial surgery

Ongoing major medical or psychiatric disorder incl. depression and psychosis

Other concomitant treatment with neuroleptics

Typical, nonparkinsonian syndrome ioflupane/SPECT signal

Trial design

Design model

Parallel

Treatments tested in this trial

  • Sural Nerve Graft to the Substantia Nigra

    Procedure/Surgery

    Participants assigned to this arm will have the sural nerve biopsied from one of their ankles. This cellular tissue will be deposited bilaterally into the substantia nigra area of their brain by a specialized cannula via bilateral scalp incisions and skull burr holes.

  • Sham surgery

    Procedure/Surgery

    Participants assigned to this arm will have the sural nerve from one of their ankles biopsied in the same fashion as the experimental arm. Bilateral incisions will be made on the participants scalp but no burr holes into the skull and no cannula passes into the brain will occur.

Treatment groups

7 Participants
are divided into 2 treatment groups
Group A: Nerve Graft RecipientsExperimental treatment 1 intervention
Group B: Placebo GroupPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Meet recruitment goal

Ability to recruit, enroll, and assign participants to the trial within 12 months of the trial opening.

Time frame
Trial opening through 12 months

Secondary outcomes

1

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

Total number of serious adverse events associated with bilateral PNT collection and deployment to the substantia nigra. Serious adverse events will be defined as an abscess, tumor, infection of the bed of the graft, altered mental state, seizure, ankle or foot infection, wound dehiscence at the ankle incision, spread of neuropathy of the ankle or foot on the ipsilateral side of the nerve biopsy, or other event determined by the PI/investigators to be important.

Time frame
Enrollment through 12 months
2

Study-related adverse events as assessed by MedDRA v27

Total number of adverse events experienced by participants categorized by System Organ Class and/or High Level Group Term using MedDRA v27 for designation.

Time frame
Enrollment through 6 month study visit
3

Number of deployment attempts required to deliver bilateral PNT

Per protocol, the surgeon has two chances to successfully deploy 60% of the PNT tissue loaded in the guide-tube into the brain per hemisphere. The number of attempted deployments will be documented and if \<60% of the tissue is deployed after two attempts, a failed delivery will be documented and no other attempts will be made.

Time frame
During the procedure
4

Duration of procedure

Number of minutes it takes for the procedure to be completed. This will be defined by the anesthesia start time and anesthesia end time.

Time frame
During the procedure

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Craig van Horne, MD, PhD

Lead sponsor

University of Kentucky

Sponsor institution

University of Kentucky CCTS

Collaborator

National Center for Advancing Translational Sciences (NCATS)

Collaborator

Source ClinicalTrials.gov
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