Marfan Syndrome and Quality of Life of Pediatric Patients

Rare Diseases Clinical Trial

— MarfanPed  

Official title:

Marfan Syndrome: the Impact on the Quality of Life, Phenotypic Manifestations on Self-image and Self-management Skills, as an Index of Adaptation to the Disease in Pediatric Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05748314
• Other study ID #: MarfanPed
• Status: Recruiting
• Start date: January 9, 2023
• Est. completion date: December 2024

Study information

• Verified date: March 2024
• Source: IRCCS Policlinico S. Donato
• Contact: Alessandro Pini, MD
• Phone: +390252774705
• Email: alessandro.pini[@]grupposandonato.it
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The goal of this observational study is to learn about the impact of Marfan syndrome (MFS) in paediatric affected subjects. the main questions it aims to answer are: - The assessment of quality of life in MFS Italian patients; - The impact of phenotypic manifestations on self-image and self-management skills; - Stratify patients according to the need of psychological support. Participants will take part in the study by completing four self-report questionnaires.

Description:

In literature, adolescents with MFS experience difficulty in connecting with peers, and are convinced that they would have been more physically attractive without the disease. Additionally, due to physical activity restrictions and orthopedic limitations, these patients reported having difficulty adjusting and participating in educational events. Data from present study will be able to support clinicians in identifying patients who need psychological support (through parental counseling interviews) aimed at increasing compliance with treatments and adaptation to the disease.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 80
• Est. completion date: December 2024
• Est. primary completion date: March 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

• Signed informed consent;
• MFS diagnosis according to Revised Ghent criteria (2010);

Exclusion Criteria:

• Known cognitive deficits

Related Conditions & MeSH terms

• Marfan Syndrome
• Pediatric Disorder
• Rare Diseases

Intervention

Other:
• Questionnaire
KINDL-R; Morisky Medication Adherence Scale;

Locations

Country	Name	City	State
Italy	IRCCS Policlinico San Donato	San Donato Milanese	Lombardia

Sponsors (1)

Lead Sponsor	Collaborator
IRCCS Policlinico S. Donato

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Psychological counseling	Questionnaire to assess psychological support is KINDL-R (Quality of life);	12 months
Primary	Psychological counseling	Questionnaire to assess psychological support is Multidimensional Self Concept Scale (MSCS)	12 months
Primary	Psychological counseling	Questionnaire to assess psychological support is Morisky Medication Adherence Scale	12 months