This study is designed to learn about the safety and effectiveness of a new gene therapy called KB408 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.
KB408 delivers copies of the genes that produce AAT to the lungs and is given by inhaling a mist (called nebulization). The genes are carried and delivered by a modified herpes simplex virus type 1 (HSV-1). This virus is not harmful and simply acts as a vehicle to deliver the genes to the lungs. The genes that are delivered by KB408 do not change a person's own DNA. This is an open-label study, meaning that the participants, the study doctor, and the sponsor all know that the participants are receiving KB408. KB408 is an investigational product, meaning it is not approved for commercial use by the FDA.
Eligible participants will receive one of three doses of KB408. Participants will have a screening visit first to make sure that they are able to participate in the study. After the screening visit, participants will need to return to the study center 6 more times over 2 months. At the second visit, participants will receive the study drug. Each visit will take between 2 and 6 hours to complete. Study procedures include medical history collection, vitals, physical exam, ECG, spirometry and DLCO, urine cotinine test, blood work, cheek swab, sputum sample, and bronchoscopy (only for participants in cohorts 3a and 3b).
Possible side effects of KB408 include temporary increases in certain cell types in the lungs and temporary increases in the breathing rate after dosing. Since this is the first time that KB408 has been given to humans, it is possible that participants may have an immune reaction to the study drug. There is also a risk with genetic testing and a risk to confidentiality. Participants may not receive any personal benefit from being in this study. There is no guarantee that the Study Drug will help. The information that is collected from the study may help other people in the future.

This is a seamless, Phase 1b/2 Multiple Ascending Dose (MAD)/Proof of Concept (POC) Study (hereafter referred to as Phase 2 Program) of XTMAB-16 in participants with pulmonary sarcoidosis with or without extrapulmonary involvement. The study is comprised of two parts: Part A is a randomized double-blind placebo-controlled multiple dose-escalating study, and Part B is a randomized double-blind placebo-controlled POC study.
The objective of Part A is to evaluate the safety and tolerability of MADs of XTMAB-16, and to determine the recommended Phase 2 dose and frequency for Part B for XTMAB-16 administration in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations.
The objective of Part B is to confirm preliminary efficacy of XTMAB-16 as measured by the ability to reduce background oral corticosteroid use in participants with pulmonary sarcoidosis with or without extrapulmonary manifestations.

The purpose of this study is to test whether a drug called efzofitimod (the study drug) is a potential treatment for patients with Systemic Sclerosis associated with Interstitial Lung Disease (SSc-ILD).

Efzofitimod is an investigational drug that is given by infusion every 4 weeks for a total of 6 doses. An investigational drug is not approved by The US Food and Drug Administration. It can only be used in a research study like this one. In this study, efzofitimod will be compared with a placebo (dummy drug). The placebo will be a saline solution that does not have any study drug in it. The comparison with the placebo helps to determine whether the effects seen in your body is because of efzofitimod or not. This is a randomized study, meaning that you will be assigned by chance (like flipping a coin) to receive either the study drug or placebo. The study is double-blinded study, meaning you and your study doctor will not know what you are receiving, the study efzofitimod or placebo.

The study is sponsored by aTyr Pharma, Inc. Participation in the study will require 9 visits to the MUSC main campus and will have the following procedures completed over the course of your participation: blood draw, urine collection, physician-led assessments of your disease (for example physical exam and skin thickness testing), tests to assess your pulmonary function and health (Pulmonary Function Test (PFT) and High Resolution Computed Tomography (HRCT)), electrocardiogram, as well as asked to complete surveys.

Compensation is available for participation

This clinical investigation is a prospective, non-randomized, single-arm, multi-center early feasibility study of the Aria CV Pulmonary Hypertension (PH) System implanted in patients with pulmonary arterial hypertension (PAH). The device will be implanted in the pulmonary artery and the gas reservoir of the device will be in the abdominal cavity. The purpose of this study is to validate that the clinical use of the Aria CV PH System is safe for the patient, and to evaluate its performance in treating patients with PAH. Some procedures involved in the study include but not limited too: Questionnaires, physical exams, right heart catherization, echocardiograms, blood work and more. The study will be conducted in a maximum of 10 centers in the United States. Up to 45 patients will be consented, and up to 15 patients will receive implants. There is a total of 11 visits over the course of 2.5 years for the study. Because this is an investigational device under the FDA, there may be risk that include but are not limited to: arrhythmia, device infection, endocarditis, and heart failure. If the Aria CV device performs as intended, you may potentially benefit from reduction in or relief of symptoms caused by PH, and depending on your overall health conditions, prolonged life expectancy and/or improvement in your overall quality of life.

The purpose of this research study is to help us understand patients' experiences with alpha-1 antitrypsin deficiency – lung disease. 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 and other lung diseases.
During screening, participants will be asked questions to assess their eligibility to take part in this study. To be considered for enrollment into the study, participants must: be between 18 to 80 years old, have a diagnosis of severe AATD, meet the additional relevant inclusion criteria, understand the study instructions, and be willing and able to follow the study instructions. Participants will participate in a one-time, 120-minute, telephone or web-based interview. At the end of the interview, participants will complete a brief questionnaire. There is a risk of discomfort answering the questions during the interview and a risk of loss of confidentiality. There is no direct benefit from participation in the study.

This is a randomized, double-blind, dose-ranging, placebo-controlled study to see how effectively and how safely two different doses of bexotegrast (160 and 320 mg) can be taken every day for 52 weeks (about 12 months) by subjects with IPF (Idiopathic Pulmonary Fibrosis) who are taking or not taking background therapy (other drugs for IPF include nintedanib or pirfenidone). The study is designed to test the study drug in subjects who are taking or not taking background therapy and will include about 80 subjects who are not taking background therapy at study entry. Subjects who are not taking background therapy at study entry will be allowed to start it at any time during the study. The study will consist of an up to 28-day Screening Period, a 52-week Treatment Period, and a 14-day Safety Follow-up Period. Participants will undergo an end-of-study visit at the end of the Safety Follow-up Period.

The purpose of the study is to evaluate the safety and how well the medication levosimenden works versus placebo in treating Pulmonary Hypertension and Heart Failure with a Preserved Ejection Fraction measured by a 6 minute walk. This is a condition where the lower left chamber (left ventricle) of the heart is not able to fill properly with blood during the filling phase and the amount of blood pumped out to the body is below normal. The study will also look at information obtained from the tests performed as part of the study to see if subjects have improvement in symptoms of heart failure. Levosimendan is a drug that has been FDA-approved for intravenous (IV) delivery to your body. This study aims to determine if the tablet form of the drug is as effective as the IV route. Tablets are much more attainable for patients to manage their heart failure from home, rather than going to an infusion clinic for treatments. Participation in this study will last approximately 12 weeks with the option to continue to the stage 2 phase of the study. If the stage 2 phase is selected as well, participation will last approximately 26 months or a little over 2 years. These visits will include such activities as blood tests, questionnaires, physical evaluation by a study doctor, echocardiogram, and 6 minute hall walks.

Participants will be randomized to either the treatment group (and receive the medication) or the control group (receive an inactive medication). Subjects will have a 50:50 chance of receiving the study medication during their participation in the trial. The treatment assignment is determined by randomization, where a computer selects at random which treatment group you will be in (like drawing straws). Neither the subject, nor the blinded personnel will know which group subjects are in. Neither the subject nor the study doctor will decide what group subjects are assigned.

This is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and efficacy of the study drug DWN12088 in patients with idiopathic pulmonary fibrosis (IPF), with or without standard-of-care. Patients meeting the eligibility criteria for the study will be randomized with a 2:1 ratio to DWN12088 150 mg BID or the matching placebo. This study will have 8 study visits over a period of a maximum of 36 weeks. This study period includes a maximum 8-week screening period, a 24-week treatment period and a 4-week follow-up period. The duration of each visit will vary depending on the procedures performed.

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled clinical study that will randomize participants with Progressive Pulmonary Fibrosis to study drug BMS-986278 60mg, 120mg, or Placebo, administered orally, twice a day. Participants are allowed to continue background therapy, such as antifibrotic and immunosuppressant therapies. This study will consist of two parts (Cohort 1 and 2). Cohort 1 will enroll approximately 60 participants with Progressive Pulmonary Fibrosis to evaluate the safety and tolerability of BMS-986278 in which participants will be randomized to receive 60 mg, 120mg, or placebo and this will last approximately 52 weeks. Cohort 2 is a registrational, double-blinded study which will investigate the efficacy, safety, and tolerability of BMS-986278 compared with placebo. Based on data from Cohort 1, the study will design 2 or 3 treatment arms for Cohort 2.

This Phase 3 study, comprising a double-blind, placebo-controlled treatment period of up to 48 weeks, will assess the efficacy and safety of seralutinib versus placebo when added to background PAH disease-specific medication in adult PAH subjects who are WHO FC II or III. The primary objective of the study is to assess the efficacy of seralutinib relative to placebo with a primary endpoint of change from baseline to Week 24 in 6MWD.