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RecruitingPhase 1

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)

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
40 Years to 75 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 1 year

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Participants will be followed initially for one year after surgery."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This early-stage study tests whether placing a person's own nerve tissue into a brain area called the substantia nigra is safe and possible. It is for people with Parkinson's disease (PD) or multiple system atrophy (MSA) who are early in their illness and have fewer symptoms. Of 7 people, 4 will get the nerve tissue and 3 will have surgery without the graft, with follow-up for one year at first.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Are 40 to 75 years old.
  • Have a confirmed or likely diagnosis of PD or MSA under the study's medical guidelines.
  • Have had the disease for more than 2 years.
  • While off Parkinson's medicines, have a PD movement score of 20–35 or an MSA symptom score of 30 or less.
  • While off medicine, score no higher than 3 on each of the seven specified PD movement test items.
  • Can and will have an ioflupane/SPECT scan, a type of brain scan.
  • Can handle surgery and complete all planned tests.
  • Have a sural nerve, a nerve in the leg, that can be reached to collect tissue.

Who can't join

  • Have had surgery for PD or surgery inside the skull.
  • Have an ongoing major medical or mental health condition, including depression or psychosis, or take antipsychotic medicines.
  • Have a brain scan result that does not show a Parkinson-like condition, or cannot have an MRI scan.
  • Have a blocked surgical path to the target brain area.
  • Have major disease of the small blood vessels.
  • Take blood-thinning medicines other than aspirin.
  • Are pregnant or breastfeeding, or could become pregnant but will not use suitable birth control during the study.
  • Lose the ability to understand and agree to the study before surgery. This ability is checked during thinking and memory testing; losing it after surgery does not mean removal from the study.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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 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.

Study sites (1)

KY