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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.
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| Measure | Description | Time Frame |
|---|---|---|
| Complete synthesis and characterization of XHALIP for in vitro studies. | Preparation and stabilization of the dry powder formulation of XHALIP. | two years from the enrollment |
| Complete synthesis and characterization of XHALIP for in vitro studies. | Evaluation of XHALIP uptake 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. | two years from the enrollment |
| Complete synthesis and characterization of XHALIP for in vitro studies. | 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. | two years from the enrollment |
| Measure | Description | Time Frame |
|---|---|---|
| Exploration of XHALIP Distribution | 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. | two years from the enrollment |
| Exploration of XHALIP Distribution |
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Inclusion Criteria:
Exclusion Criteria:
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The subjects are enrolled at the Fondazione IRCCS Policlinico San Matteo hospital in Pavia, at the SC of Pulmonology of the Foundation.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Stefano Ghio, MD | Contact | +39 0382503460 | s.ghio@smatteo.pv.it |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fondazione IRCCS Policlinico San Matteo | Recruiting | Pavia | Pavia | 27100 | Italy |
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XHALIP distribution will be explored in a model that replicates the alveolo-capillary microenvironment to analyze its interaction with specific pathological human cells and mucosal/surfactant layers, as well as its ability to cross the alveolo-capillary interface. |
| two years from the enrollment |
| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| D011658 | Pulmonary Fibrosis |
| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D017563 | Lung Diseases, Interstitial |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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