Actively Recruiting

Age: 18Years +
All Genders
NCT07302321

Needs of ALS Patients With C9orf72 Mutation and Their Caregivers

Led by Istituto Auxologico Italiano · Updated on 2026-02-04

208

Participants Needed

3

Research Sites

38 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Needs of ALS Patients With C9orf72 Mutation and Their Caregivers

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • 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
  • Caregiver of patient with diagnosis of ALS with mutation C9orf72 (C9Pos), who participates in this study
  • Preserved cognitive functions (for caregivers)
Not Eligible

You will not qualify if you...

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

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 3 locations

1

Istituto Auxologico Italiano IRCCS

Milan, Lombardy, Italy, 20145

Actively Recruiting

2

IRCCS Ospedale San Raffaele

Milan, Italy

Not Yet Recruiting

3

IRCCS Fondazione Mondino, Istituto Neurologico Nazionale a Carattere Scientifico

Pavia, Italy

Not Yet Recruiting

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Research Team

V

Vincenzo Silani, Prof.

CONTACT

L

Luca Grappiolo

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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