Lung Fibrosis Interstitial, Pulmonary Arterial Hypertension (PAH)
Conditions
Keywords
PAH, LFD, Pulmonary arterial hypertension, lung fibrosis desease, XHALIP, imatinib
Brief summary
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.
Interventions
None listed
Sponsors
Fondazione IRCCS Policlinico San Matteo di Pavia
Study design
Observational model
OTHER
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* Suspected or confirmed diagnosis of pulmonary neoplastic disease.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Complete synthesis and characterization of XHALIP for in vitro studies. | two years from the enrollment | Preparation and stabilization of the dry powder formulation of XHALIP. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Exploration of XHALIP Distribution | two years from the enrollment | The distribution of XHALIP will be studied in thin lung tissue cultures (Precision Cut Lung Slices Cells, PCLSC) obtained from lung explants of patients with LFD and PAH. |
Countries
Italy
Contacts
Primary ContactStefano Ghio, MD
Outcome results
None listed