Preclinical Assessment of an Engineered HMGB1 Protein as a Novel Companion Therapy for Muscular Dystrophies
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- HMGB1 concentration
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •An individual who meets any of the following criteria will be excluded from participation in this study:
- •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.
结局指标
主要结局
HMGB1 concentration
时间窗: 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)
次要结局
未报告次要终点