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Researchers are trying to find out more about the safety of a new treatment, Allogeneic (coming from a healthy donor) Bone Marrow Derived Mesenchymal Stem Cells (BMD-MSCs) which is still experimental, for Interstitial Lung Disease (ILD) associated with Connective Tissue Disorder (CTD).
Hermansky-Pudlak Syndrome (HPS) is a rare genetic disease that is associated with oculocutaneous albinism, bleeding, granulomatous colitis, and pulmonary fibrosis in some subtypes, including HPS-1, HPS-2, and HPS-4. Pulmonary fibrosis causes shortness of breath and progressive decline in lung function. In HPS patients with at-risk subtypes, almost all adults eventually develop fatal pulmonary fibrosis unless they undergo lung transplantation. The purpose of this study is to identify the earliest measurable pulmonary disease activity in individuals at-risk for HPS pulmonary fibrosis. The study also aims to develop biomarkers that will aid...
This Expanded Access Program is intended to facilitate the availability of OFEV to patients suffering from non Idiopathic Pulmonary Fibrosis-Interstitial Lung Disease (non IPF-ILD) with a progressive clinical course despite Standard of Care treatment and for whom no satisfactory authorised alternative therapy exists or who cannot enter a clinical trial.
This is a Phase 1 study, to assess the safety, tolerability, pharmacokinetics, and food effect of single and multiple ascending doses of ANG-3070 in healthy adult participants. This study is comprised of 10 cohorts. 6 single ascending dose (SAD) cohorts and 4 multiple ascending dose (MAD) cohorts. Each cohort will have a total of 8 subjects: SAD and MAD (2 subjects receiving placebo and 6 subjects receiving active ANG 3070)
The I-FILE study is a prospective multicenter, multinational observational study where the feasibility of a patient-led registry using home monitoring in patients with pulmonary fibrosis will be evaluated. The aim of the study is to gain more insights in disease behavior in patients with pulmonary fibrosis, so in future patients with progressive disease can be better identified.
Idiopathic pulmonary fibrosis (IPF) is a rare, progressive life-threatening disease that is characterized by exertional dyspnea and persistent dry cough. Cough in IPF is both a presenting and a complicating clinical feature, which affects approximately three quarters of IPF cases. It is often a debilitating symptom that adversely affects quality of life (QoL) and is usually refractory to medical therapy. Inhaled RVT-1601 (formerly, PA101B), a new inhalation formulation of cromolyn sodium delivered via the eFlow® Closed System (CS) nebulizer, is being evaluated in this Phase 2b study for the treatment of persistent cough in patients with...
Early initiation of treatment for Rheumatoid arthritis (RA) can prevent several of the long term problems associated with the condition. However, many RA patients develop lung inflammation and scarring, called 'interstitial lung disease' (RA-ILD), contributing to early death in 1 in 5 people. There is no proven treatment for these patients and some medications for RA can in fact worsen their lung disease. There is a need therefore to find safe medications that can not only control RA joint disease, but also prevent progression of RA-ILD. Abatacept is an approved drug for treating RA and is used widely. It is a newer RA medication, with a...
Langerhans cell histiocytosis (LCH) is a rare, heterogeneous histiocytic disorder occurring in patients of all ages from neonates to the elderly. The current standard treatment protocol for children with de novo multisystem LCH is vinblastine plus prednisone. This regimen has never been proven effective for adults in a prospective study, since the only prospective trial evaluating the efficacy of a vinblastine/prednisone regimen in adults was prematurely closed due to unacceptable toxicities. A retrospective study showed an advantage for cytarabine monotherapy compared with vinblastine/prednisone in bone LCH patients. This phase 2,...
The objective of this study is to administer and validate a disease specific health related quality of life (HRQOL) survey for patients with Chronic Hypersensitivity Pneumonitis (CHP).
The purpose of this study is to evaluate whether biomarkers of lung injury and remodeling are responsive to effective continuous positive airway pressure (CPAP) treatment in adults with idiopathic pulmonary fibrosis (IPF) and moderate-to-severe obstructive sleep apnea (OSA).
The Pulmonary Fibrosis Foundation rates among top charities in the U.S. The PFF has a three-star rating from Charity Navigator and is a Better Business Bureau accredited charity.
