One Gene, Two Diseases: the Pathologic Role of IGLV1-44 in AL Amyloidosis and POEMS
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome
研究概览
简要总结
By detailed sequence analysis and subsequent biophysical characterization of prototypic light chains, this project aims to identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome. This understanding might help improve the risk stratification and early diagnosis of patients overexpressing pathologic IGLV1-44 LCs. Moreover, the development of nanobodies efficient in recognizing and stabilizing IGLV1-44 light chains which exert direct toxicity in cardiac AL amyloidosis and POEMS syndrome might form the basis for future development of therapeutic agents capable of counteracting IGLV1-44 light chain proteotoxicity.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 99 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Biopsy-proven diagnosis of systemic AL amyloidosis, POEMS syndrome or multiple myeloma
- •Planned peripheral blood sampling +/- bone marrow aspiration
- •Age > 18 years
- •Willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes through signing a written informed consent.
排除标准
- •Undefined monoclonal gammopathy or non-AL amyloidosis
- •Patients fulfilling the criteria for complete hematologic response after anti-clonal therapy
- •Age <18 years
- •Failure to show willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes.
结局指标
主要结局
identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome
时间窗: two years
By detailed sequence analysis and subsequent biophysical characterization of prototypic light chains, this project aims to identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome.
次要结局
未报告次要终点
研究者
Mario Nuvolone
Principal Investigator
Fondazione IRCCS Policlinico San Matteo di Pavia