This is a Phase 3 clinical research study for adults with pulmonary hypertension associated with interstitial lung disease, also called PH-ILD. The study will compare an investigational inhaled drug called treprostinil palmitil inhalation powder, or TPIP, with placebo. Participants will be assigned by chance to receive TPIP or placebo once daily for 24 weeks. The main purpose is to determine whether TPIP improves how far participants can walk in six minutes and whether it delays worsening of PH-ILD. Participants will complete study visits, physical examinations, blood tests, heart and lung testing, questionnaires, and six-minute walk tests. Participation may last up to approximately 32 weeks, including screening, treatment, and follow-up. An optional hemodynamic sub-study will include an additional right heart catheterization near the end of study treatment for eligible participants who separately consent.
Patients who are scheduled to have a robotic-assisted procedure to examine a lung nodule and a procedure to check the lymph nodes in their lungs, will be eligible to participate in this randomized trial. The study will compare two different orders of these procedures to see if the order affects the results. The purpose of this research study is to learn which order works best when performing two standard medical procedures: a robotic-assisted bronchoscopy to examine a lung nodule and an endobronchial ultrasound (EBUS) to examine the lymph nodes in the lungs.
This study is for patients who have undergone surgery for non-small cell lung cancer (NSCLC), and your doctors have found that some cancer may still be present in nearby lymph nodes. The study is being done to figure out if adding radiation therapy after surgery can help lower the chance of lung cancer coming back while keeping side effects at a safe level.
The study is being done to find out if this approach is better or worse than the usual approach for your non-small cell lung cancer (NSCLC) after surgery. The usual approach is defined as care most people get for non-small cell lung cancer and is approved by the Food and Drug Administration (FDA).
If you decide to take part in this study, you will either get chemotherapy or immunotherapy or you will get chemotherapy or immunotherapy plus an additional type of radiation therapy called I²-PORT for up to 24 months
After you finish your treatment, your doctor and study team will continue to follow your condition for side effects and cancer recurrence. Follow-up visits will be every 3 months for 2 years and every 6 months for 3 more years, totaling 5 years of follow-up.
There is evidence that I²-PORT radiation therapy is effective in reducing the risk of cancer returning in certain patients with lung cancer. It is not possible to know now if this approach will extend your time without disease compared to the usual approach. This study will help the study doctors learn things that will help people in the future.
If you choose to take part in this study, there is a risk that the I²-PORT radiation therapy may not be as good as the usual approach for your cancer at preventing your cancer from coming back. Some of the most common side effects that the study doctors know about are reddening, tanning, mild pain, hair loss in treated area, and or tiredness. There may be some risks that the study doctors do not yet know about.
The purpose of the Re-Spire Study is to help researchers learn more about the efficacy and long-term safety and tolerability of the research drug L606 in participants with PH-ILD.
There are two phases of the Re-Spire Study participants can potentially join with the second phase starting directly after the first phase ends. The first phase is called the "Blinded Treatment Phase" (BTP). This is the "double-blind," assigned by chance portion of the study and will last approximately 28 weeks (a 30-day Screening period followed by a 24-week study period). The second phase of the Re-Spire Study is called the "Open Label Extension Phase" (OLEP). This phase is optional. It is called "open-label" because there is no placebo and everyone who joins will take the active study drug, L606.
About 350 participants will take part in this research study from around 120 study centers or research sites in various countries or regions.
This study is designed to evaluate a new therapy formulation for Alpha-1 Antitrypsin Deficiency (AATD). AATD is an inherited condition in which a person has low blood levels of a protein known as alpha-1 protease inhibitor (called Alpha1-PI). AATD causes an increased risk of chronic obstructive pulmonary disease (COPD) in the form of emphysema (long term lung disease) and, less frequently, other diseases.
This study is being conducted to evaluate the safety and tolerability of 2 different doses of Alpha-1 drugs (Alpha-1 15% and Liquid Alpha1-PI) in participants with AATD. Participants will be placed into one of two groups. Each group will receive both drugs, but different doses of each, at different points in the treatment period (8 Weekly IV infusions of Liquid Alpha1-PI 60 mg/kg followed by 8 Weekly SC (subcutaneous) infusions Alpha1-PI 90 mg/kg OR 8 Weekly IV infusions of Liquid Alpha1-PI 120 mg/kg followed by 8 Weekly SC (subcutaneous) infusions Alpha1-PI 180 mg/kg).
This is an "open label" study, meaning participants and the study staff know which dose of study drug participants receive. The study will last up to 161 days (23 weeks). Many visits are able to be conducted through home health care, lessening the need to come into the clinic.
Alpha-1 15% is an investigational product, meaning it is not approved by the U.S. Food and Drug Administration (FDA). The other drug in this study is Liquid Alpha1-PI (licensed as Prolastin®-C Liquid) and is an FDA approved treatment for adults with emphysema due to AATD. However, it is only approved for the recommended dose of 60 mg/kg. This study includes both the FDA approved 60mg/kg of Liquid Alpha1-PI and an experimental dose of 120 mg/kg that is not FDA approved. Alpha-1 15% is given as an injection under the skin and Liquid Alpha1-PI is given as an infusion into the veins.
This study is being conducted to evaluate the drug AZD0292, including how safe it is, how long it stays in the blood, and if it may help in reducing the number of exacerbations in people with Pseudomonas aeruginosa in their lungs. These patients have more frequent lung exacerbations and reduced quality of life. Pseudomonas aeruginosa is a bacteria that can make the symptoms of bronchiectasis worse. The study drug (AZD0292) works by attaching to Pseudomonas aeruginosa, thereby reducing its effects on the lungs and improving symptoms.
This study aims not only to test AZD0292 but is also being done to better understand bronchiectasis disease and its associated health problems.
Study drug (AZD0292) or placebo, will be given to participants as an intravenous (IV) infusion. The study is double-blinded.
About 435 participants with bronchiectasis 12 years of age and older, weighing at least 35kg will take part in this study. This study will be conducted globally in approximately 25 countries.
This is a Phase 1/2, open-label, multi-center clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of TSRA-196, a gene editing compound, in adults with severe alpha-1 antitrypsin deficiency (PiZZ genotype) and associated lung and/or liver disease. Participants will receive a single intravenous dose of TSRA-196 in a dose-escalation phase followed by dose-expansion cohorts.
The study will assess safety outcomes, pharmacokinetics, and changes in serum alpha-1 antitrypsin levels and lung function to determine whether TSRA-196 can safely increase functional AAT levels and inform selection of an appropriate dose for further clinical development.
The purpose of the study is to find out if mosliciguat, the investigational drug that is being studied, with treprostinil, is safe and effective in treating adults who live with PH-ILD. Approximately 20 participants diagnosed with PH-ILD who are currently on a stable dose of inhaled treprostinil who meet all inclusion and no exclusion criteria will be enrolled. Part 1 of the study will take about 5 ½ months. Part 2 of the study is optional if you choose to participate in the extension period. Part 2 does not have a set end date and your participation in Part 2 will end if you or your study doctor decide to stop your participation or the Sponsor ends the study.
This an observational study collecting data for up to 8 years on patients who have been diagnosed with PiZZ or PiSZ Alpha-1 Antitrypsin Deficiency with or without liver disease. Patients' clinical, medical, and laboratory data will be collected prospectively per routine care and questionnaires will be collected during the clinic visits with the hopes of getting a better understanding of the natural progress of lung and liver disease associated with Alpha-1 Antitrypsin Deficiency.
The purpose of the study is to learn more about an experimental drug called ORC-13661 that may be helpful in treating hearing loss due to antibiotic use. The study is researching whether the drug is safe and tolerable, and trying to find out whether the drug may be able to mitigate or prevent hearing loss in patients being treated with intravenous (IV) amikacin. Participation in the study will consist of up to 10 visits over up to 129 days. The Day 1 visit (following the screening visit) will last up to 8 hours, while other visits will last up to 5 hours Participants will be randomly assigned to receive either ORC-13661at a higher dose or a lower dose or a placebo (a pill that looks the same as the study drug but has no real medicine in it), for up to 90 days to be taken along with IV amikacin. Participants will be asked to have a number of tests and procedures, which include questionnaires about family, medical, and hearing history, physical and hearing examination, and assessments of hearing.