Engineered HMGB1 protein as a companion therapy for LAMA2-related muscular dystrophy

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 is a monocentric, non-profit observational study at IRCCS Ospedale San Raffaele that uses stored and prospectively collected biospecimens. It analyzes samples already collected under a prior consent framework (LAMA2_GUP24002) to explore the potential role of an engineered HMGB1 protein as a companion therapy. No new procedures or interventions are performed on participants; analyses rely on existing material in the biobank. Eligible participants are those enrolled in LAMA2_GUP24002 who have signed appropriate consent, including biobank consent. Exclusion criteria focus on withdrawal of consent, degraded samples, or reclassification of diagnosis away from LAMA2-RD.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patients affected by LAMA2-related muscular dystrophy (LAMA2-RD) actively enrolled in the LAMA2\_GUP24002 study.
2. Patients who have ALREADY SIGNED the informed consent for participation in the LAMA2\_GUP24002 study, including the separate biobank-specific consent (INSPE BIOBANK CONSENT), with consent provided directly by adult patients or, in the case of minors, by their parents or legal guardians.

Exclusion Criteria:

An individual who meets any of the following criteria will be excluded from participation in this study:

1. Withdrawal of informed consent (to the LAMA2\_GUP24002 study or to biobanking) prior to laboratory analysis.
2. Insufficient or degraded biological material (e.g., depleted aliquots; volumes below assay requirements; biopsy not representative of LAMA2-RD muscle).
3. Re-classification of diagnosis as a non-LAMA2-RD condition after the original enrolment in the LAMA2\_GUP24002 study.

Where it's running

• Irccs Ospedale San Raffaele
  Milan, Italy Recruiting

Study contacts

• Study coordinator: Alberto A Zambon, MD, PHD
• Email: zambon.alberto@hsr.it
• Phone: +390226432751

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Natural history and biomarker mapping for LAMA2-related dystrophy

  It will track how LAMA2-related dystrophy changes over time in children and adults and look for biological markers that match disease features.

  Recruiting Università Vita-Salute San Raffaele · Milan · NCT07125040

• Natural history study of LAMA2 muscular dystrophy in children

  This study is trying to understand how LAMA2 muscular dystrophy affects children over time to help improve future treatments.

  Recruiting Hospital Universitari Vall d'Hebron Research Institute · Barcelona, Barcelona · NCT06924125

• Tracking movement and health in children 0–5 with LAMA2-related congenital muscular dystrophy

  This project will see how children aged 0–5 with LAMA2-related dystrophy move, breathe, and develop over two years to help find reliable outcome measures for future treatments.

  Recruiting at 13 sites Nationwide Children's Hospital · Los Angeles, California and 13 other locations · NCT06503367

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.