Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorIRCCS San Raffaele
About this trial
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
This trial does not accept healthy volunteersQualifiers
Patients affected by LAMA2-related muscular dystrophy (LAMA2-RD) actively enrolled in the LAMA2_GUP24002 study.
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.
Disqualifiers
Withdrawal of informed consent (to the LAMA2_GUP24002 study or to biobanking) prior to laboratory analysis.
Insufficient or degraded biological material (e.g., depleted aliquots; volumes below assay requirements; biopsy not representative of LAMA2-RD muscle).
Re-classification of diagnosis as a non-LAMA2-RD condition after the original enrolment in the LAMA2_GUP24002 study.
Trial population
LAMA2-RD patients (children and adults, both sexes)
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
HMGB1 concentration
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)
Time frame
36 MONTHSSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov