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 project is being conducted in subjects that have been diagnosed with myelodysplastic syndromes (MDS), MDS/myeloproliferative neoplasms (MPN) including chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML) who are candidates to receive treatment with single agent azacitidine based on local country approvals and/or local The study is designed to move efficiently from Phase 1 to Phase 3. This study is testing investigational drug called ASTX030. Investigational means that it is not approved by the Food and Drug Administration (FDA), but it is undergoing testing to find out if it is safe and effective. ASTX030 is a combination of two medicines, azacitidine and cedazuridine, given by mouth. The primary purpose is to test the levels of the investigational drug ASTX030 in your blood, including if food has an effect (Phase 1B only), the safety and tolerability of the drugs, and how subjects respond to the drug. The subject may remain in the study about 3 years. If you benefit from treatment, you may receive study drugs as long as you continue to benefit. If you develop side effects to the study drugs that prevent you from continuing treatment, or if your study doctor believes it is in your best interest to stop the study drug(s), you may be asked to stop the study treatment. After you stop treatment, the Sponsor will continue to collect health information to evaluate long-term effects of the study drugs.

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.

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.

The proposed research study evaluates the potential of an oral FDA-approved medication, suvorexant, as compared with a placebo control to improve symptoms of sleep disturbance and insomnia in breast cancer survivors currently on hormonal based therapies. Eligible participants will randomly be selected to receive either the suvorexant or a placebo control for a duration of 4 Weeks. Participation in the study will include a screening process for eligibility including information on medical history and surveys regarding symptoms at the start of the study and additionally at 2 and 4 weeks. Potential benefits include improved sleep for individuals randomized to the study medication and potential study risks include loss of confidentiality of health information and a risk of sedation and complex sleep behaviors associated with the medication, suvorexant.

This study is for female subjects that have a very specific type of recurrent ovarian, peritoneal, or fallopian tube cancer. These patients must have completed 1 round of chemotherapy for their cancer type. The study is testing an "investigational" (not yet FDA approved) drug called MIRV (Mirvetuximab Soravtansine). The primary purpose of this study is to determine the effectiveness and safety of the study drug to determine what effects, if any, it has on subjects. Subjects may remain in the study for up to 2 years.

This study is enrolling subjects with advanced solid tumors with the TP53 Y220C alteration in cancer cells. This alteration is a mutation of the tumor that can be found through laboratory tests that use a sample of tissue, blood, or other fluids to check for signs of cancer. This study involves research testing the safety, best dose, side effects and timing of the study drug called PC1486. You will take the study drug, PC14586, in the form of a tablet. If assigned to the combination treatment arm (Part 1), you will also be given pembrolizumab as an intravenous (IV) infusion. Pembrolizumab is a type of immunotherapy that is FDA approved to treat your kind of cancer. The study drug PC1486 is not approved by the Food and Drug Administration (FDA). This study drug targets the TP53 Y220C genetic alteration in tumor cells. During the pre-screening portion, participants will provide either archived tissue or a fresh tumor sample to test for the genetic alteration. If the participants show the genetic tumor alteration in the cancer cells, they may be asked to participate in the trial. Treatment arms are groups or subgroups of participants in a clinical trial. This study will have two arms: one studies PC1486 alone and the second arm studies PC1486 in combination with Pembrolizumab against advanced solid tumors. PC14586 is a small molecule (chemical) that is designed to act on the genetic alteration TP53 Y220C in cancer cells to slow the growth of cancer, and it is taken orally. This study involves blood tests, a possible tumor biopsy, CT, and MRI scans. The study will last approximately 6 months of treatment, followed up by a check-up after the first 3 weeks and every 3 months after. Some common risks (observed in greater than 20% of people) include: Nausea, Vomiting, Abnormal liver tests, Abnormal kidney test (possible kidney damage

The purpose of this clinical trial is to evaluate the safety and efficacy of BIA-28-6156 in subjects with Parkinson's Disease (PD). BIA -28-6156 is an investigational drug meaning that its safety, effects and how it works are still being studied. This is a randomized (assigned by chance), placebo-controlled study, which means that some participants will receive a fake treatment (placebo) while others get the real treatment. The placebo treatment looks like the BIA-28-6156 medications but doesn't contain any active ingredient. The medication is in a pill form and will be administered orally. This research is also double blind, meaning that neither the participants nor the researcher will know which treatment they will be receiving. In Part A of this study, participants will be asked to give a blood sample for a genetic screening test. If genetic testing in part A indicates the person can participate, they may be asked to volunteer for part B. Duration of Part B is about 87 weeks, this includes 5 weeks for screening, 78 weeks for the double-blind treatment period, and 4 weeks for follow up. There are at least 11 visits in total in 20 months (9 clinic visits, 2 remote phone/video visits); this does not include unscheduled visits. During visits, participants should anticipate tests including electrocardiograms (ECGs), vitals measurements (including temperature, blood pressure, and heart rate), and a physical/neurological examination. Some of the risks includes fatigue, headache, drowsiness, muscle aches and possible worsening of PD symptoms. As for benefits, participants who receives the BIA drug may see their PD progression slowing.

About 380 males and females aged 55 to 90 years with psychosis associated with Alzheimer's disease will take part in this worldwide study. This study will be conducted at up to 100 sites in the United States, Canada, France, Germany, Spain and others in approximately 10 countries. The study will test KarXT, a combination of 2 study drugs, xanomeline tartrate and trospium chloride. Xanomeline tartrate stimulates the muscarinic receptors (a receptor in the nervous system) that is involved in cognitive and neurobehavioral functions. Stimulation of these receptors has been shown to effect changes in cognitive performance and promote antipsychotic activity. Trospium chloride is a muscarinic inhibitor that is expected to reduce the negative peripheral (outside the brain) muscarinic side effects of xanomeline tartrate without reducing its intended therapeutic effects in the brain.
The goal of this study is to assess whether KarXT helps in relapse prevention in people with psychosis associated with AD as compared to placebo, see how safe and well accepted KarXT is in people with psychosis associated with AD as compared to placebo, and check what the body does to KarXT and how long does it stay in the body (pharmacokinetics) as compared to placebo.

This study will evaluate the associations and interactions between bar-lab/naturalistic drinking variables with participant characteristics in individuals with bipolar disorder and alcohol use disorder. This study includes 4 study visits over approximately 1 week. Questionnaires and clinical interview measures will be completed at study visits.