MarfanPed: Marfan Syndrome and Quality of Life of Pediatric Patients

• Sponsor: IRCCS Policlinico S. Donato (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT05748314
• Collaborator: (none)
• Enrollment: 80
• Location: 1
• Anticipated Duration (Months): 16.7
• Patients Per Site Per Month: 4.8

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Rare Diseases; Pediatric Disorder	Other: Questionnaire

Detailed 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.

Study Design

• Study Type: Observational
• Anticipated Enrollment: 80 participants
• Observational Model: Cohort
• Time Perspective: Cross-Sectional
• 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
• Actual Study Start Date: Jan 9, 2023
• Anticipated Primary Completion Date: Jan 1, 2024
• Anticipated Study Completion Date: Jun 1, 2024

Outcome Measures

Primary Outcome Measures

1. Psychological counseling [12 months]

   Questionnaire to assess psychological support is KINDL-R (Quality of life);

2. Psychological counseling [12 months]

   Questionnaire to assess psychological support is Multidimensional Self Concept Scale (MSCS)

3. Psychological counseling [12 months]

   Questionnaire to assess psychological support is Morisky Medication Adherence Scale

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Signed informed consent;

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

Exclusion Criteria:

• Known cognitive deficits

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	IRCCS Policlinico San Donato	San Donato Milanese	Lombardia	Italy	20097

Sponsors and Collaborators

• IRCCS Policlinico S. Donato

Investigators

• Principal Investigator: Alessandro Pini, MD, Cardiovascular-Gentic Centre, IRCCS Policlinico San Donato

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Alessandro Pini, Principal Investigator, IRCCS Policlinico S. Donato
• ClinicalTrials.gov Identifier: NCT05748314
• Other Study ID Numbers: MarfanPed
• First Posted: Feb 28, 2023
• Last Update Posted: Feb 28, 2023
• Last Verified: Feb 1, 2023

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Alessandro Pini, Principal Investigator, IRCCS Policlinico S. Donato: Quality of Life; Self-identification; Self-care
• Additional relevant MeSH terms: Marfan Syndrome; Arachnodactyly; Rare Diseases

Study Results

No Results Posted as of Feb 28, 2023