Observational Study for the Epidemiology of Cerebral Palsy in Italy

NCT ID: NCT06813586

Last Updated: 2025-02-07

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

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-06-01

Study Completion Date

2026-06-01

Brief Summary

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BACKGROUND AND RATIONALE Cerebral Palsy (CP) is the leading cause of childhood disability, with an estimated prevalence of 2 to 2.5 per 1,000 births. This complex clinical condition encompasses motor development disorders as well as sensory, communication, cognitive, and emotional impairments. Despite the significant healthcare impact of CP, standardized guidelines for its study and treatment are lacking both nationally and internationally. Existing literature is limited by small sample sizes, inconsistent assessment tools, and insufficient etiopathogenetic and functional characterizations of different CP types. A national registry, which could offer comprehensive epidemiological data, does not currently exist.

This project aims to create a shared digital platform across clinical centers throughout Italy (three coordinative centers for northern, central, and southern Italy). This platform will allow systematic, standardized, retrospective, and prospective data collection, including clinical, instrumental, and genetic data (where available) for patients with CP. The study intends to complement ongoing national projects, such as the "Italian Network for Early Detection and Intervention in Developmental Disabilities (INEED)" and the "Italian Network for Cerebral Palsy (Ita-Net-CP)."

STUDY OBJECTIVES

This study aims to analyze epidemiological data, specifically:

* Evaluating the prevalence of various clinical types of CP
* Assessing the distribution of functional impairment severity and associated comorbidities.

STUDY DESIGN This is a non-profit, multicenter, observational study involving a cohort of CP patients, promoted within the Mariani Foundation Network. The study involves both retrospective and prospective data collection. It will enroll both patients already under care for follow-up and newly diagnosed patients.

A sample of at least 300 children and/or adolescents with CP of various etiologies, with 100 participants per clinical center, is targeted. Enrollment will be voluntary, either by invitation from the referring physician, who will explain the importance of the registry, or through project dissemination, allowing parents to request their children's inclusion. Basic registry items can be filled out by parents and, with permission, the primary physician for clinical fields. Parents can opt to be included in the registry to receive information on national clinical studies, approved by ethics committees.

Collected essential data includes:

* Identification and contact information for patient pseudonymization
* Socioeconomic status
* CP classification according to the European Surveillance Group
* Anamnesis regarding disorder etiology
* Neuroradiological classification based on SCPE (MRICS)
* Functional impairment level of each child
* Significant comorbidities

STUDY POPULATION At least 300 pediatric CP patients aged 4-18 years are expected to be recruited. Exclusion criteria only include lack of informed consent.

DRUG, MEDICAL DEVICE, NUTRACEUTICAL, BIOLOGICAL SAMPLES, OTHER ELEMENTS This observational study involves systematic, standardized retrospective and prospective data collection of clinical, instrumental, and genetic data (if available). No medical devices, pharmaceuticals, or nutraceuticals will be used. No human biological samples will be collected.

STUDY TIMELINE Study duration per patient: aligned with routine clinical visits. No additional evaluations are foreseen.

Recruitment duration: 12 months Overall study duration: 36 months

SAMPLE SIZE AND DATA ANALYSIS The study aims to recruit at least 300 pediatric CP patients. A control group is not planned.

Detailed Description

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Conditions

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Cerebral Palsy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Children with CP

Children with diagnosed CP age: 4-18 years old

hospital-based registry

Intervention Type OTHER

The clinical data collected for each patient will reflect comprehensive multiprofessional clinical insights necessary for diagnostic definition and typically obtained during diagnostic evaluation and/or monitoring visits. In addition to these, instrumental data commonly acquired in clinical practice, such as neuroradiological and electrophysiological information, will also be collected.

For each enrolled subject, a checklist (CRF) will be completed to gather study-related data. These data will be entered into a specific database constructed on the REDCap platform and managed in an anonymized form. Subject enrollment will be conducted progressively.

This data collection will enable monitoring of study progress, including the number of enrolled subjects, completed assessments, and study adherence. Every three months, monitoring will be performed to assess study progress and the achievement of enrollment targets.

Interventions

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hospital-based registry

The clinical data collected for each patient will reflect comprehensive multiprofessional clinical insights necessary for diagnostic definition and typically obtained during diagnostic evaluation and/or monitoring visits. In addition to these, instrumental data commonly acquired in clinical practice, such as neuroradiological and electrophysiological information, will also be collected.

For each enrolled subject, a checklist (CRF) will be completed to gather study-related data. These data will be entered into a specific database constructed on the REDCap platform and managed in an anonymized form. Subject enrollment will be conducted progressively.

This data collection will enable monitoring of study progress, including the number of enrolled subjects, completed assessments, and study adherence. Every three months, monitoring will be performed to assess study progress and the achievement of enrollment targets.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of cerebral palsy (CP) confirmed clinically in accordance with recent international guidelines (SCPE Guidelines, 2019);
* Age between 4 and 18 years.

Exclusion Criteria

* Progressive neuromotor disorder (defined as a condition involving the loss of previously acquired abilities);
* Patients with a neurological condition defined by isolated hypotonia;
* Patients with neurological deficits secondary to spinal injury;
Minimum Eligible Age

4 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fondazione Pierfranco e Luisa Mariani

UNKNOWN

Sponsor Role collaborator

Fondazione FightTheStroke

UNKNOWN

Sponsor Role collaborator

Istituto Superiore di Sanità

OTHER

Sponsor Role collaborator

IRCCS Eugenio Medea

OTHER

Sponsor Role collaborator

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

OTHER

Sponsor Role collaborator

IRCCS Fondazione Stella Maris

OTHER

Sponsor Role lead

Responsible Party

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Giuseppina Sgandurra

Associate Professor, Head of INNOVATE Lab

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Associazione La Nostra Famiglia - IRCCS Eugenio Medea

Brindisi, Apulia, Italy

Site Status RECRUITING

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Milan, Lombardy, Italy

Site Status RECRUITING

IRCCS Fondazione Stella Maris

Calambrone, Tuscany, Italy

Site Status RECRUITING

Countries

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Italy

Central Contacts

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Giuseppina Sgandurra MD, PhD

Role: CONTACT

050886284

Facility Contacts

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Antonio Trabacca

Role: primary

0831349643

Pagliano Emanuela

Role: primary

02 2394.2218

Giuseppina Sgandurra, MD PhD

Role: primary

050886284

References

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Long HL, Mahr TJ, Natzke P, Rathouz PJ, Hustad KC. Longitudinal change in speech classification between 4 and 10 years in children with cerebral palsy. Dev Med Child Neurol. 2022 Sep;64(9):1096-1105. doi: 10.1111/dmcn.15198. Epub 2022 Mar 9.

Reference Type BACKGROUND
PMID: 35262181 (View on PubMed)

Rosenbaum PL, Palisano RJ, Bartlett DJ, Galuppi BE, Russell DJ. Development of the Gross Motor Function Classification System for cerebral palsy. Dev Med Child Neurol. 2008 Apr;50(4):249-53. doi: 10.1111/j.1469-8749.2008.02045.x. Epub 2008 Mar 1.

Reference Type BACKGROUND
PMID: 18318732 (View on PubMed)

Sellier E, Surman G, Himmelmann K, Andersen G, Colver A, Krageloh-Mann I, De-la-Cruz J, Cans C. Trends in prevalence of cerebral palsy in children born with a birthweight of 2,500 g or over in Europe from 1980 to 1998. Eur J Epidemiol. 2010 Sep;25(9):635-42. doi: 10.1007/s10654-010-9474-0. Epub 2010 Jun 8.

Reference Type BACKGROUND
PMID: 20532622 (View on PubMed)

Scheck SM, Pannek K, Fiori S, Boyd RN, Rose SE. Quantitative comparison of cortical and deep grey matter in pathological subtypes of unilateral cerebral palsy. Dev Med Child Neurol. 2014 Oct;56(10):968-75. doi: 10.1111/dmcn.12461. Epub 2014 Apr 19.

Reference Type BACKGROUND
PMID: 24749496 (View on PubMed)

Surveillance of Cerebral Palsy in Europe. Surveillance of cerebral palsy in Europe: a collaboration of cerebral palsy surveys and registers. Surveillance of Cerebral Palsy in Europe (SCPE). Dev Med Child Neurol. 2000 Dec;42(12):816-24. doi: 10.1017/s0012162200001511.

Reference Type BACKGROUND
PMID: 11132255 (View on PubMed)

Pennington L, Virella D, Mjoen T, da Graca Andrada M, Murray J, Colver A, Himmelmann K, Rackauskaite G, Greitane A, Prasauskiene A, Andersen G, de la Cruz J. Development of The Viking Speech Scale to classify the speech of children with cerebral palsy. Res Dev Disabil. 2013 Oct;34(10):3202-10. doi: 10.1016/j.ridd.2013.06.035. Epub 2013 Jul 24.

Reference Type BACKGROUND
PMID: 23891732 (View on PubMed)

Bax M, Goldstein M, Rosenbaum P, Leviton A, Paneth N, Dan B, Jacobsson B, Damiano D; Executive Committee for the Definition of Cerebral Palsy. Proposed definition and classification of cerebral palsy, April 2005. Dev Med Child Neurol. 2005 Aug;47(8):571-6. doi: 10.1017/s001216220500112x.

Reference Type BACKGROUND
PMID: 16108461 (View on PubMed)

Baranello G, Signorini S, Tinelli F, Guzzetta A, Pagliano E, Rossi A, Foscan M, Tramacere I, Romeo DMM, Ricci D; VFCS Study Group. Visual Function Classification System for children with cerebral palsy: development and validation. Dev Med Child Neurol. 2020 Jan;62(1):104-110. doi: 10.1111/dmcn.14270. Epub 2019 Jun 10.

Reference Type BACKGROUND
PMID: 31180136 (View on PubMed)

Himmelmann K, Horber V, De La Cruz J, Horridge K, Mejaski-Bosnjak V, Hollody K, Krageloh-Mann I; SCPE Working Group. MRI classification system (MRICS) for children with cerebral palsy: development, reliability, and recommendations. Dev Med Child Neurol. 2017 Jan;59(1):57-64. doi: 10.1111/dmcn.13166. Epub 2016 Jun 21.

Reference Type BACKGROUND
PMID: 27325153 (View on PubMed)

Sellers D, Mandy A, Pennington L, Hankins M, Morris C. Development and reliability of a system to classify the eating and drinking ability of people with cerebral palsy. Dev Med Child Neurol. 2014 Mar;56(3):245-51. doi: 10.1111/dmcn.12352. Epub 2013 Dec 18.

Reference Type BACKGROUND
PMID: 24344767 (View on PubMed)

Fiori S, Cioni G, Klingels K, Ortibus E, Van Gestel L, Rose S, Boyd RN, Feys H, Guzzetta A. Reliability of a novel, semi-quantitative scale for classification of structural brain magnetic resonance imaging in children with cerebral palsy. Dev Med Child Neurol. 2014 Sep;56(9):839-45. doi: 10.1111/dmcn.12457. Epub 2014 Apr 19.

Reference Type BACKGROUND
PMID: 24750109 (View on PubMed)

Elvrum AK, Andersen GL, Himmelmann K, Beckung E, Ohrvall AM, Lydersen S, Vik T. Bimanual Fine Motor Function (BFMF) Classification in Children with Cerebral Palsy: Aspects of Construct and Content Validity. Phys Occup Ther Pediatr. 2016;36(1):1-16. doi: 10.3109/01942638.2014.975314. Epub 2014 Nov 6.

Reference Type BACKGROUND
PMID: 25374154 (View on PubMed)

Eliasson AC, Krumlinde-Sundholm L, Rosblad B, Beckung E, Arner M, Ohrvall AM, Rosenbaum P. The Manual Ability Classification System (MACS) for children with cerebral palsy: scale development and evidence of validity and reliability. Dev Med Child Neurol. 2006 Jul;48(7):549-54. doi: 10.1017/S0012162206001162.

Reference Type BACKGROUND
PMID: 16780622 (View on PubMed)

Other Identifiers

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DataNetCP

Identifier Type: -

Identifier Source: org_study_id

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