NCT07745218 Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
| NCT ID | NCT07745218 |
| Status | Recruiting |
| Phase | — |
| Sponsor | IRCCS San Raffaele |
| Condition | LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A) |
| Study Type | OBSERVATIONAL |
| Enrollment | 25 participants |
| Start Date | 2026-08-01 |
| Primary Completion | 2029-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 25 participants in total. It began in 2026-08-01 with a primary completion date of 2029-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
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.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07745218 clinical trial?
This trial is open to participants of all sexes, studying LAMA2-MD (Merosin Deficient Congenital Muscular Dystrophy, MDC1A). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07745218 currently recruiting?
Yes, NCT07745218 is actively recruiting participants. Contact the research team at zambon.alberto@hsr.it for enrollment information.
Where is the NCT07745218 trial being conducted?
This trial is being conducted at Milan, Italy.
Who is sponsoring the NCT07745218 clinical trial?
NCT07745218 is sponsored by IRCCS San Raffaele. The trial plans to enroll 25 participants.