Observational Study for the Epidemiology of Cerebral Palsy in Italy

Summary

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.

Conditions

• Cerebral Palsy

Interventions

OTHER

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.

Sponsors & Collaborators

• Fondazione Pierfranco e Luisa Mariani

  collaborator UNKNOWN

• Fondazione FightTheStroke

  collaborator UNKNOWN

• Istituto Superiore di Sanità

  collaborator OTHER

• IRCCS Eugenio Medea

  collaborator OTHER

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

  collaborator OTHER

• IRCCS Fondazione Stella Maris

  lead OTHER

Eligibility

Min Age

4 Years

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-06-01

Primary Completion

2026-06-01

Completion

2026-06-01

Countries

• Italy

Study Locations