Learn about Research & Clinical Trials
All-Case Surveillance of Ofev in Patients With IPF in Japan
This is a non-interventional study based on new data collection to gather real-world information (i.e., data under routine medical practice) on safety and effectiveness of the Ofev® Capsules treatment. The study will consist of a baseline visit and follow-up visits at Week 4, 13, 26, 39, 52, 65, 78, 91 and 104 for patients who have newly initiated Ofev® Capsules. The patients will be followed up until discontinuation of Ofev® Capsules treatment. As this is an observational study, no specific treatment is mandated or withheld from the patients. The choice of maintenance treatment for IPF must be according to regular medical ...
A Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects With Idiopathic Pulmonary Fibrosis
This is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial to evaluate the efficacy, safety and tolerability of HZN-825 in subjects with IPF. Subjects will be screened within 8 weeks prior to the Baseline (Day 1) Visit. Approximately 360 subjects who meet the trial eligibility criteria will be randomly assigned in a 1:1:1 ratio on Day 1 to receive HZN-825 300 mg QD, HZN-825 300 mg BID or placebo for 52 weeks using the following 2 stratification factors: 1. Prior use of approved IPF therapy (i.e., nintedanib or pirfenidone): yes or no 2. FVC % predicted at Baseline: ≥70% or <70%
Analysis of Specimens From Individuals With Pulmonary Fibrosis
The etiology of pulmonary fibrosis is unknown. Analyses of blood, genomic DNA, and specimens procured by bronchoscopy, lung biopsy, lung transplantation, clinically-indicated extra-pulmonary biopsies, or post-mortem examination from individuals with this disorder may contribute to our understanding of the pathogenic mechanisms of pulmonary fibrosis. The purpose of this protocol is to procure and analyze blood, genomic DNA, and specimens by bronchoscopy, lung biopsy, lung transplantation, extra-pulmonary biopsies, or post-mortem examination from subjects with pulmonary fibrosis. In addition, blood, genomic DNA, clinically-indicated...
An International Patient-led Registry in Fibrotic Interstitial Lung Diseases Using eHealth Technology
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.
A Non-pharmacological Cough Control Therapy
Coughing affects almost all individuals with ILD leading to physical, psychological and social distress and prevents individuals from performing their activities of daily living, working or socialising in public places. Unfortunately, there are no licensed medications available to treat chronic cough and the few drugs that have been tried resulted in little efficacy and significant side effects. Drug-free cough control interventions have shown promise in reducing the severity and impact of coughing on patients' lives but have not been tested in individuals with ILD. This study aims to explore the feasibility and effectiveness of a...
A Phase 2 Study of APX-115 in Hospitalized Patients With Confirmed Mild to Moderate COVID-19.
This phase 2 study is to assess the safety and tolerability of APX-115 active doses compared to placebo following multiple oral dosing in hospitalized patients with confirmed, mild to moderate, symptomatic COVID-19. It is anticipated that approximately 80 patients will be randomized into the study in a 1:1 ratio to 100 mg APX-115 or placebo arm.
A Prospective Study for the Treatment of Children With Newly Diagnosed LCH Using a Cytarabine Contained Protocol
From January 2010 to December 2014, 150 children with MS-LCH were treated in our hospital following a LCH II (Arm B) based protocol. Treatment was based on a modification of the LCH-II (Arm B) based protocol. However, the continuation treatment was extended to 56 weeks and etoposide was omitted from the continuation treatment. For the 59 patients with RO involvement (RO+) (the lungs are not considered a RO in the current study), the rapid response rate (week 6) was 61.0% and the 3-year overall survival (OS) 73.4±5.9%. Rapid responders had a better 3-year survival rate than poor responders (90.9±5.0% vs. 45.7±11.0%, P<0.001). The...
AraC for Newly Diagnosed Adult Langerhans Cell Histiocytosis
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,...
A Safety, Tolerability, and Pharmacokinetic Study of NIP292 in Healthy Normal Subjects
This study is being conducted to evaluate the safety and tolerability of single ascending and multiple ascending oral doses of NIP292 tablets administered following an overnight fast in healthy adult subjects.
A Single-cell Approach to Identify Biomarkers of Pulmonary Toxicity for Immune Checkpoint Blockade
The main goal of this prospective non-interventional exploratory monocentric study is to characterize the immune cell composition of bronchoalveolar lavage (BAL) fluid from cancer patients experiencing cancer therapy-induced pneumonitis on a single-cell scale. These mechanistic insights can directly lead to putative diagnostic biomarkers and therapeutic targets. A second highly clinically relevant hypothesis is that single-cell profiling of blood samples will reveal circulating biomarkers of ICB toxicity, making non-invasive diagnosis feasible.