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Needs of ALS Patients With C9orf72 Mutation and Their Caregivers

Needs of ALS Patients With C9orf72 Mutation and Their Caregivers

Recruiting
18 years and older
All
Phase N/A

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Overview

Individuals with Amyotrophic Lateral Sclerosis (ALS) carrying the C9orf72 HRE mutation (C9Pos) often exhibit different phenotypic traits compared to other patients (C9Neg), presenting a more aggressive form of the disease, with also a higher frequency of comorbidity with frontotemporal dementia (ALS-FTD) and a greater prevalence of family history of ALS and/or other neurodegenerative diseases.

Considering these characteristics (comorbidity with FTD and family history of ALS and/or other neurodegenerative pathologies) as factors that have their importance from an assistance point of view, both C9orf72 patients and their caregivers may have particular needs in several respects, and in this sense this investigation is configured.

This survey aims to describe the various aspects related to the care and quality of life of C9orf72 ALS patients and their respective caregivers, with the goal of identifying actionable steps to improve the quality of life for both.

Eligibility

ALS Patients:

Inclusion Criteria:

  • Diagnosis of ALS with mutation C9orf72 (C9Pos)
  • Age 18 years or older
  • Fluency in Italian language
  • Ability to understand the nature of the study and to reply to the questions in the online survey
  • Informed consent signed

Exclusion Criteria:

  • Clinically relevant cognitive dysfunction
  • Incapacity to reply to at least half of the questions of the online survey
  • Hospital Anxiety and Depression Scale (HADS) ≥ 11
    Caregivers

Inclusion Criteria:

  • Caregiver of patient with diagnosis of ALS with mutation C9orf72 (C9Pos), who participates in this study
  • Age 18 years or older
  • Preserved cognitive functions
  • Informed consent signed

Study details
    Amyotrophic Lateral Sclerosis

NCT07302321

Istituto Auxologico Italiano

1 February 2026

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