RECRUITINGPhase 1INTERVENTIONAL

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

A Phase I Study of the Feasibility and Safety of SuraL nervE Tissue Grafting to the Substantia nigrA in Patients With Synucleinopathies (LEAP)

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 - No MDS-UPDRS Part III score \>3 on items 3.3, 3.4, 3.5, 3.6, 3.7, 3.8, 3.14 while off medication - 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 Who Should NOT Join This Trial: - 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 - Unable to undergo an MRI - An obstructed trajectory path to the substantia nigra - Significant microvascular disease - Use of anticoagulants other than aspirin - Female who is pregnant, lactating, or of child-bearing potential unwilling to use an adequate birth control method during the period of the study - Consent capacity will be assessed and determined during and throughout a participant's neuropsychological exam. A participant who experiences a decline in consent capacity prior to surgery, will be removed from the study by the PI. A decline in consent capacity after surgery will not result in the removal of the participant in the study. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * 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 * No MDS-UPDRS Part III score \>3 on items 3.3, 3.4, 3.5, 3.6, 3.7, 3.8, 3.14 while off medication * 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: * 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 * Unable to undergo an MRI * An obstructed trajectory path to the substantia nigra * Significant microvascular disease * Use of anticoagulants other than aspirin * Female who is pregnant, lactating, or of child-bearing potential unwilling to use an adequate birth control method during the period of the study * Consent capacity will be assessed and determined during and throughout a participant's neuropsychological exam. A participant who experiences a decline in consent capacity prior to surgery, will be removed from the study by the PI. A decline in consent capacity after surgery will not result in the removal of the participant in the study.

Treatments Being Tested

PROCEDURE

Sural Nerve Graft to the Substantia Nigra

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.

PROCEDURE

Sham 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.

Locations (1)

University of Kentucky

Lexington, Kentucky, United States