Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07745218

Acronym

HEAL-MD

Enrollment

Registered

2026-08-04

Start date

2026-08-01

Completion date

2029-07-01

Last updated

2026-08-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A)

Brief summary

This is a monocentric, no-profit, retrospective and prospective observational cohort study conducted at IRCCS Ospedale San Raffaele. The study does not entail any additional procedures or interventions for participants. Biological samples (including blood/serum and muscle biopsies) are obtained for a previously approved study (LAMA2\_GUP24002) and collected under the existing BancaINSpe informed consent. For the present study, analyses will be performed on biospecimens already stored or that will be stored at BancaINSpe, using portions of material already collected for the LAMA2\_GUP24002 protocol.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

OTHER

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
HMGB1 concentration	36 MONTHS	To quantify total HMGB1 concentration in serum of patients affected by LAMA2-related muscular dystrophy (LAMA2-RD), using serum samples already collected and stored within the LAMA2\_GUP24002 study (complete LAMA2-RD and in patients with partial LAMA2-RD)

Countries

Italy

Contacts

CONTACTAlberto A Zambon, MD, PHD

zambon.alberto@hsr.it+390226432751

Outcome results