Study on the Incidence of Malignant Neoplasms in Patients with Parkinson's Disease and Heterozygous Mutation of the GBA Gene

NCT ID: NCT06814431

Last Updated: 2025-02-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

3000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-11-23

Study Completion Date

2026-09-30

Brief Summary

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This multicenter retrospective observational study investigates the incidence of malignant neoplasms in Parkinson's disease (PD) patients carrying heterozygous pathogenic variants in the GBA1 gene. The study compares these patients to individuals with idiopathic PD and to the general population within a large national cohort.

The rationale for the study lies in the growing evidence indicating an increased risk of malignant neoplasms in patients affected by Gaucher disease.

The primary objective is to assess whether PD patients with GBA1 mutations have a higher incidence of malignant neoplasms compared to the general population. Secondary objectives include comparing the incidence of malignant neoplasms between GBA1-PD and idiopathic PD patients, as well as between idiopathic PD patients and the general population. Additionally, the study aims to characterize oncological conditions by sex, age, center, and tumor site, with specific attention to distinguishing hematologic tumors from solid tumors.

By identifying potential associations between GBA1 mutations and malignancies in PD, the findings could guide more comprehensive patient management, including screening for malignant neoplasms alongside Parkinson's disease care.

Detailed Description

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The study will involve a network composed by different Italian Movement Disorders Centres and the corresponding local cancer registries, responsible for the active collection of cancer cases. Data will be collected from approximately 3000 consecutive Parkinson's disease (PD) patients, including about 500 patients carrying GBA1 mutations and 2500 idiopathic PD. Data about the presence of malignancies will be extracted from the Local Cancer Registries for each patient.

Conditions

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Idiopathic Parkinson's Disease (PD)

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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GBA-PD Cohort

Parkinson's disease (PD) patients diagnosed based on the "United Kingdom Parkinson's Disease Society Brain Bank Diagnostic Criteria". Patients in this group must have a documented heterozygous pathogenic variant in the GBA1 gene.

No interventions assigned to this group

Idiopathic PD Cohort

This cohort includes patients diagnosed with Parkinson's disease (PD) based on the "United Kingdom Parkinson's Disease Society Brain Bank Diagnostic Criteria". Patients in this group do not have any documented heterozygous pathogenic variants in the GBA1 or LRRK2 genes.

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* \>18 years
* Diagnosis of Parkinson's Disease through the United Kingdom Parkinson's Disease Society Brain Bank Diagnostic Criteria for Parkinson's Disease.

Exclusion Criteria

* Uncertain diagnosis
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Azienda USL Reggio Emilia - IRCCS

OTHER_GOV

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ospedale A. Perrino

Brindisi, , Italy

Site Status RECRUITING

IRCCS Istituto Neurologico Carlo Besta

Milan, , Italy

Site Status RECRUITING

Azienda USL IRCCS di Reggio Emilia

Reggio Emilia, , Italy

Site Status RECRUITING

Ospedale Santa Chiara di Trento

Trento, , Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Franco Valzania, MD

Role: CONTACT

+39 0522 296494

Facility Contacts

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Francesca Spagnolo, MD

Role: primary

Francesca Spagnolo, MD

Role: backup

Roberto Cilia, MD

Role: primary

Roberto Cilia, MD

Role: backup

Giulia Toschi, Clinical Research Coordinator

Role: primary

+390522295565

Franco Valzania, MD

Role: backup

Maria Chiara Malaguti, MD

Role: primary

Maria Chiara Malaguti, MD

Role: backup

References

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Stirnemann J, Belmatoug N, Camou F, Serratrice C, Froissart R, Caillaud C, Levade T, Astudillo L, Serratrice J, Brassier A, Rose C, Billette de Villemeur T, Berger MG. A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments. Int J Mol Sci. 2017 Feb 17;18(2):441. doi: 10.3390/ijms18020441.

Reference Type BACKGROUND
PMID: 28218669 (View on PubMed)

Mistry PK, Taddei T, vom Dahl S, Rosenbloom BE. Gaucher disease and malignancy: a model for cancer pathogenesis in an inborn error of metabolism. Crit Rev Oncog. 2013;18(3):235-46. doi: 10.1615/critrevoncog.2013006145.

Reference Type BACKGROUND
PMID: 23510066 (View on PubMed)

Other Identifiers

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133/2023/OSS/AUSLRE

Identifier Type: -

Identifier Source: org_study_id

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