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NCT06683365 | NOT YET RECRUITING | Multiple System Atrophy

Autologous SuraL NervE Grafting to the Substantia NigrA in Patients with Synuclienopathies
Sponsor:

Craig van Horne, MD, PhD

Information provided by (Responsible Party):

Craig van Horne, MD, PhD

Brief Summary:

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.

Condition or disease

Multiple System Atrophy

Parkinsons Disease

Intervention/treatment

Sural Nerve Graft to the Substantia Nigra

Sham surgery

Phase

PHASE1

Detailed Description:

This phase I double blind clinical trial will be used to plan future, larger clinical trials that would test how autologous cells from the peripheral nerve may help in the repair of damaged brain cells in Parkinson's Disease (PD) or Multiple System Atrophy (MSA) and slow the progression of the diseases. We will be judging the feasibility of implanting a participant's own cells from a nerve in the leg into the substantia nigra area of the brain. Patients eligible for participation will be at an earlier in stage of the disease with symptoms being less severe and therefore would not yet qualify for DBS. The LEAP trial is a study where the first participant will receive an implantation of the cells from their own sural nerve (a nerve near the ankle), into the substantia nigra on both sides of their brain. The 6 participants who follow, will be randomized to one of two arms. The 3 participants assigned to the experimental arm will receive the graft. The 3 participants assigned to the control arm will receive a sham surgical procedure, where the sural nerve will be biopsied, and bilateral scalp incisions will be made. Those who do not receive the cells initially may be eligible to undergo another surgery at the end of the study, after un-blinding has occurred, to receive the cell implants.

Study Type : INTERVENTIONAL
Estimated Enrollment : 7 participants
Masking : QUADRUPLE
Primary Purpose : TREATMENT
Official Title : A Phase I Study of the Feasibility and Safety of SuraL NervE Tissue Grafting to the Substantia NigrA in Patients with Synucleinopathies (LEAP)
Actual Study Start Date : 2024-12-15
Estimated Primary Completion Date : 2028-12-02
Estimated Study Completion Date : 2030-12-02

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 40 Years to 75 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
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 = 20 points and = 35 points off anti-parkinsonian medication for PD or MDS-Unified Multiple System Atrophy Rating Scale (UMSARS) = 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.
Autologous SuraL NervE Grafting to the Substantia NigrA in Patients with Synuclienopathies

Location Details

NCT06683365

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Locations

Not yet recruiting

United States, Kentucky

University of Kentucky

Lexington, Kentucky, United States, 40536