PROOF OF CONCEPT STUDY ON THE EFFICACY OF INHALATORY TARGETED IMATINIB NANOFORMULATIONS IN PULMONARY HYPERTENSION AND POST-INFLAMMATORY FIBROSIS.
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Complete synthesis and characterization of XHALIP for in vitro studies.
研究概览
简要总结
The PROMPTLY study is a proof-of-concept research project evaluating the efficacy of XHALIP, an innovative inhalable formulation of Imatinib, in treating pulmonary arterial hypertension (PAH) and post-inflammatory fibrosing lung diseases (LFD).
Main Objective To demonstrate the ability of XHALIP to improve the prognosis of PAH and LFD by reducing pulmonary fibrosis and vascular remodeling.
Key Research Questions Is XHALIP effectively absorbed by pathological lung cells? Does it have a beneficial biological effect on cell proliferation and extracellular matrix deposition? Study Plan Preclinical phase: In vitro tests on cells obtained from patients with PAH and LFD.
Absorption and distribution analysis: In vitro lung tissue models to assess drug penetration and effectiveness.
Biological activity assessment: Evaluating XHALIP's ability to inhibit epithelial-mesenchymal transition and cell proliferation.
Participants Patients aged ≥ 18 years diagnosed with PAH or LFD, either candidates for or recipients of lung transplantation.
Biological samples (bronchoalveolar lavage and explanted lung tissues). Methodology Confocal microscopy and flow cytometry to analyze absorption. RT-PCR and Western Blot to assess biological efficacy. Alveolo-capillary models to test drug release and distribution. The study aims to translate preclinical findings into a potential future clinical trial for the development of XHALIP as a novel inhalation-based therapeutic strategy for rare lung diseases.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥ 18 years diagnosed with fibrosing lung disease, obliterative bronchiolitis, or PAH, who are candidates for or have undergone lung transplantation.
- •Obtaining informed consent for all patients enrolled prospectively and all those enrolled retrospectively at their first clinical occurrence at our center.
排除标准
- •Suspected or confirmed diagnosis of pulmonary neoplastic disease.
结局指标
主要结局
Complete synthesis and characterization of XHALIP for in vitro studies.
时间窗: two years from the enrollment
Evaluation of CD44 expression by various cell types obtained ex vivo from patients with LFD and PAH, as well as transplant recipients (endothelial, epithelial, MC, PASMC, inflammatory effector cells), cultured in vitro and in co-culture systems.
次要结局
- Exploration of XHALIP Distribution(two years from the enrollment)
研究者
Stefano Ghio
MD
Fondazione IRCCS Policlinico San Matteo di Pavia