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OpenTrials
Active, Not Recruiting

NCT Number: NCT05748314

Marfan Syndrome and Quality of Life of Pediatric Patients

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Rare Diseases Disease Attributes Pathologic Processes Pathological Conditions, Signs and Symptoms Pediatric Disorder

Age range

Up to 18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

IRCCS Policlinico San Donato

San Donato Milanese, Lombardy, 20097, Italy

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • Known cognitive deficits

Treatment and study plan

Questionnaire

Other

KINDL-R; Morisky Medication Adherence Scale;

Primary outcomes

  1. Psychological counseling

    Time frame: 12 months

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

  2. Psychological counseling

    Time frame: 12 months

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

  3. Psychological counseling

    Time frame: 12 months

    Questionnaire to assess psychological support is Morisky Medication Adherence Scale

Sponsors and collaborators

Lead sponsor

IRCCS Policlinico S. Donato

Other

Registry information

Official study 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

Acronym: MarfanPed

Important dates

Study start
2023
Primary completion
2026
Study completion
2027
First posted
Feb 28, 2023
Registry last updated
Jul 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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