NCT07474779

Brief Summary

The project aims to investigate how abnormal accumulation of alpha synuclein and its interaction with tau influence brain function across the Parkinson's disease (PD) spectrum, with particular focus on individuals carrying GBA1 mutations. This interventional, monocentric, cross sectional study includes patients with PD, individuals with idiopathic REM sleep behavior disorder, and participants without PD. All enrolled subjects will undergo clinical and neuropsychological assessments, blood based biomarker analyses related to neurodegeneration, synaptic and mitochondrial function, and multimodal brain MRI to evaluate brain structure, white matter integrity, and functional connectivity. The study aims to:

  • characterize the relationship between alpha synuclein/tau pathology and synaptic mitochondrial dysfunction;
  • identify biomarker and connectivity signatures across disease stages and genetic backgrounds;
  • integrate preclinical, clinical, biological, and imaging data to support the development of mechanistic models of alpha synuclein propagation. In parallel, preclinical studies in GBA PD mouse models and wild type mice will be used to investigate how changes in PD-related pathology (alpha-synuclein and tau) relates to behavior, brain imaging alterations and mitochondrial, axonal and synaptic damage. Animal model will also aid the validation of a new PET tracer that targets alpha synuclein (i.e., \[¹⁸F\]Syntacasyn). Together, human and preclinical studies are designed to provide a translational framework integrating molecular changes with brain network alterations and clinical heterogeneity in PD.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
160

participants targeted

Target at P75+ for not_applicable

Timeline
33mo left

Started May 2026

Typical duration for not_applicable

Geographic Reach
1 country

2 active sites

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 6, 2026

Completed
10 days until next milestone

First Posted

Study publicly available on registry

March 16, 2026

Completed
2 months until next milestone

Study Start

First participant enrolled

May 11, 2026

Expected
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2028

2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2029

Last Updated

May 1, 2026

Status Verified

March 1, 2026

Enrollment Period

2.6 years

First QC Date

March 6, 2026

Last Update Submit

April 27, 2026

Conditions

Keywords

GBA1 carriersBrain ConnectivityVirtual BrainExtracellular vesiclesMitochondrial DysfunctionMice ModelsTranslational Science

Outcome Measures

Primary Outcomes (3)

  • Concentration of alpha-synuclein levels in plasma

    Quantification of levels of Total alpha-synuclein and 129P-alpha-synuclein (pg/ml) in neuronal-derived extracellular vesicles using ultrasensitive immunoassays (NULISA).

    Baseline visit

  • Concentration of tau levels in plasma

    Quantification of total-tau and p-tau18 (pg/ml) in neuronal-derived extracellular vesicles using ultrasensitive immunoassays (NULISA).

    Baseline visit

  • Investigation of glucocerebrosidase activity in Peripheral Blood Mononuclear Cells

    Glucocereborsidase activity will be estimated in Peripheral Blood Mononuclear Cells usign a flourimetry assay

    Baseline visit

Secondary Outcomes (6)

  • Assessment of mitochondrial damage in plasma

    Baseline visit

  • Plasma synaptic protein concentration

    Baseline visit

  • Brain network connectivity in Parkinson's disease

    Baseline visit

  • In vitro neuronal responses in hiPSC-derived dopaminergic neurons

    Baseline visit

  • Estimation of synaptic damage in Neuronal extracellular vesicle

    Baseline visit

  • +1 more secondary outcomes

Other Outcomes (1)

  • Comprehensive clinical and neuropsychological assessement

    Baseline visit

Study Arms (6)

GBA-PD

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw, skin biopsy

Diagnostic Test: brain imagingProcedure: blood drawProcedure: Skin biopsy

nonGBA-PD

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw, skin biopsy

Diagnostic Test: brain imagingProcedure: blood drawProcedure: Skin biopsy

nonGBA-iRBD

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw, skin biopsy

Diagnostic Test: brain imagingProcedure: blood drawProcedure: Skin biopsy

GBA-iRBD

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw, skin biopsy

Diagnostic Test: brain imagingProcedure: blood drawProcedure: Skin biopsy

GBA-nonPD

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw

Diagnostic Test: brain imagingProcedure: blood draw

Healthy controls

EXPERIMENTAL

Diagnostic Test: brain imaging Procedure/Surgery: blood draw

Diagnostic Test: brain imagingProcedure: blood draw

Interventions

brain imagingDIAGNOSTIC_TEST

Participants will undergo a single brain MRI acquisition, including structural, diffusion-weighted, and resting-state functional MRI.

GBA-PDGBA-iRBDGBA-nonPDHealthy controlsnonGBA-PDnonGBA-iRBD
blood drawPROCEDURE

Collection of a venous blood sample for biochemical analyses

GBA-PDGBA-iRBDGBA-nonPDHealthy controlsnonGBA-PDnonGBA-iRBD
Skin biopsyPROCEDURE

A small punch skin biopsy (about 3-4 mm) will be performed under local anesthesia on a small sample of enrolled participants (n=10)

GBA-PDGBA-iRBDnonGBA-PDnonGBA-iRBD

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of PD according to MDS-PD criteria and, for the GBA-PD group, presence of heterozygous GBA mutations (with a balanced distribution of severe, risk, mild, and complex variants);
  • Disease duration between 3 and 7 years;
  • Disease stage according to Hoehn \& Yahr ≤ 3;
  • Absence of mutations in other known genes associated with PD susceptibility;
  • Age \> 18 Years;
  • Ability to understand and voluntarily sign informed consent and to comply with study procedures.

You may not qualify if:

  • Diagnosis of atypical and/or secondary parkinsonism;
  • Diagnosis of dementia according to DSM-5 criteria;
  • Presence of other neurological disorders and/or essential tremor;
  • Presence of systemic inflammatory or infectious diseases, autoimmune diseases, or malignant tumors at the time of enrollment.
  • Age \> 18 Years;
  • Ability to understand and voluntarily sign informed consent and to comply with study procedures;
  • No diagnosis of PD or other neurological disorders;
  • Presence of a heterozygous GBA mutation for the GBA-nonPD group and absence of such mutation for control subjects (nonGBA-nonPD);
  • Absence of mutations in other known genes associated with PD susceptibility.
  • Presence of systemic inflammatory or infectious diseases, autoimmune diseases, or malignant tumors at the time of enrollment;
  • Diagnosis of atypical and/or secondary parkinsonism;
  • Diagnosis of dementia according to DSM-5 criteria.
  • Diagnosis of idiopathic REM Sleep Behavior Disorder according to ICSD-3;
  • Age \> 18 Years;
  • Ability to understand and voluntarily sign informed consent and to comply with study procedures;
  • +6 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Neurological Institute Foundation Casimiro Mondino

Pavia, PV, 27100, Italy

Location

University of Pavia

Pavia, PV, 27100, Italy

Location

Related Publications (6)

  • Baden P, Perez MJ, Raji H, Bertoli F, Kalb S, Illescas M, Spanos F, Giuliano C, Calogero AM, Oldrati M, Hebestreit H, Cappelletti G, Brockmann K, Gasser T, Schapira AHV, Ugalde C, Deleidi M. Glucocerebrosidase is imported into mitochondria and preserves complex I integrity and energy metabolism. Nat Commun. 2023 Apr 6;14(1):1930. doi: 10.1038/s41467-023-37454-4.

    PMID: 37024507BACKGROUND
  • Neumann J, Bras J, Deas E, O'Sullivan SS, Parkkinen L, Lachmann RH, Li A, Holton J, Guerreiro R, Paudel R, Segarane B, Singleton A, Lees A, Hardy J, Houlden H, Revesz T, Wood NW. Glucocerebrosidase mutations in clinical and pathologically proven Parkinson's disease. Brain. 2009 Jul;132(Pt 7):1783-94. doi: 10.1093/brain/awp044. Epub 2009 Mar 13.

    PMID: 19286695BACKGROUND
  • Vogel JW, Corriveau-Lecavalier N, Franzmeier N, Pereira JB, Brown JA, Maass A, Botha H, Seeley WW, Bassett DS, Jones DT, Ewers M. Connectome-based modelling of neurodegenerative diseases: towards precision medicine and mechanistic insight. Nat Rev Neurosci. 2023 Oct;24(10):620-639. doi: 10.1038/s41583-023-00731-8. Epub 2023 Aug 24.

    PMID: 37620599BACKGROUND
  • Pan L, Li C, Meng L, Tian Y, He M, Yuan X, Zhang G, Zhang Z, Xiong J, Chen G, Zhang Z. Tau accelerates alpha-synuclein aggregation and spreading in Parkinson's disease. Brain. 2022 Oct 21;145(10):3454-3471. doi: 10.1093/brain/awac171.

    PMID: 35552614BACKGROUND
  • Guo M, Wang J, Zhao Y, Feng Y, Han S, Dong Q, Cui M, Tieu K. Microglial exosomes facilitate alpha-synuclein transmission in Parkinson's disease. Brain. 2020 May 1;143(5):1476-1497. doi: 10.1093/brain/awaa090.

    PMID: 32355963BACKGROUND
  • Recasens A, Dehay B. Alpha-synuclein spreading in Parkinson's disease. Front Neuroanat. 2014 Dec 18;8:159. doi: 10.3389/fnana.2014.00159. eCollection 2014.

    PMID: 25565982BACKGROUND

MeSH Terms

Conditions

Parkinson DiseaseREM Sleep Behavior DisorderMitochondrial Diseases

Interventions

NeuroimagingBlood Specimen Collection

Condition Hierarchy (Ancestors)

Parkinsonian DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative DiseasesREM Sleep ParasomniasParasomniasSleep Wake DisordersMental DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Diagnostic ImagingDiagnostic Techniques and ProceduresDiagnosisDiagnostic Techniques, NeurologicalInvestigative TechniquesSpecimen HandlingClinical Laboratory TechniquesPuncturesSurgical Procedures, Operative

Central Study Contacts

Silvia P Caminiti, PhD

CONTACT

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Model Details: Participants are enrolled into distinct groups based on clinical status and genetic background. Participants undergo the following procedures: neurological and neuropsychological assessment, collection of biological samples, and brain MRI. Group allocation is determined by predefined inclusion/exclusion criteria.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
PhD

Study Record Dates

First Submitted

March 6, 2026

First Posted

March 16, 2026

Study Start (Estimated)

May 11, 2026

Primary Completion (Estimated)

December 1, 2028

Study Completion (Estimated)

February 1, 2029

Last Updated

May 1, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations