Respiratory progression in adults with Duchenne muscular dystrophy: a long-term observational study

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This multicenter, longitudinal observational study will collect and analyze clinical, respiratory, motor, and genetic data from adults with DMD over 36 months. Researchers will use spirometry, inspiratory pressures, and sleep studies to track respiratory function, with diaphragm ultrasound in a subset to assess diaphragmatic function. Blood samples will be examined for genetic modifiers and inflammatory biomarkers that may relate to disease progression. The study aims to describe long-term respiratory progression and to validate new assessment methods, while exploring how motor function and genetic factors relate to respiratory decline.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age ≥18 years.
* Genetically confirmed Duchenne muscular dystrophy (DMD).
* Not enrolled in interventional clinical trials.
* Signed informed consent for study participation and personal data processing.

Exclusion Criteria:

* Acute respiratory failure at enrollment.
* Severe cognitive impairment preventing study adherence (IQ \<45).
* Advanced heart failure (left ventricular ejection fraction \<30%).
* Failure to provide informed consent for study participation or personal data processing.

Where it's running

• Istituto delle Scienze Neurologiche di Bologna - IRCCS
  Bologna, Italy Recruiting
• Associazione La Nostra Famiglia Eugenio Medea - IRCCS
  Bosisio Parini, Italy Recruiting
• Fondazione Stella Maris - IRCCS
  Calambrone, Italy Recruiting
• IRCCS Istituto Giannina Gaslini
  Genova, Italy Recruiting
• Azienda Ospedaliera Policlinico Universitario "G. Martino"
  Messina, Italy Recruiting
• Centro Clinico NeMO
  Milan, Italy Recruiting

Study contacts

• Study coordinator: Federica Trucco
• Email: malattieneuromuscolari@gaslini.org
• Phone: +39 010 5636 2912

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.