This study is being conducted to determine how well participants with stage IV or advanced stage IIIB/C nonsquamous non-small cell lung cancer (NSCLC), that has certain biomarkers (negative for PD-L1, and positive for KRAS p.G12C) respond to treatment with chemotherapy (carboplatin and pemetrexed) combined with either sotorasib or pembrolizumab. The study will determine whether sotorasib and chemotherapy reduces the risk of your cancer getting worse and improves your survival (i.e. increases the chance of you living longer) compared to the current standard treatment with pembrolizumab and chemotherapy. In this study, participants will be screened and if they meet criteria will be enrolled into the main study and assigned, by chance, into one of 2 groups. You will have an equal chance of taking sotorasib platinum doublet combination chemotherapy or pembrolizumab platinum doublet combination chemotherapy. It is hoped that the information gained from the study will help refine future chemotherapy programs.
This study is for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) whose disease has returned after treatment or has not responded to treatment, and who have previously received a Bruton tyrosine kinase (BTK) inhibitor.
This study is testing an investigational drug called BGB-16673. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA).
This is a randomized study, which means participants are assigned by chance (like flipping a coin) to receive either BGB-16673 or pirtobrutinib.The primary purpose of this study is to compare the safety and effectiveness of BGB-16673 with pirtobrutinib in patients with relapsed or refractory CLL or SLL.The study drugs are taken by mouth. Participants will attend regular study visits for health assessments, laboratory tests, and disease evaluations and will remain in the study until their disease worsens, they experience unacceptable side effects, choose to leave the study, or the study ends.
This study is for patients that have been diagnosed with platinum-resistant, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer. The study is testing and investigational drug called INCB123667. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to evaluate the efficacy and safety of INCB123667 in terms of objective response rate (ORR) as assessed by an independent review committee (IRC). The drug is given to participants orally. Participants can expect to be on this study for approximately 24 months.
This study will compare the efficacy and safety of SD-101-6.0 cream with SD-101-0.0 (placebo) in approximately 80 patients with Epidermolysis Bullosa (EB), including Simplex, Recessive Dystrophic, and JEB-nH subtypes. Patients will apply SD-101-6.0 or placebo topically once daily to the entire body for 60 days. Each patient will have one target wound that is 21 days or older and 10–50 cm² in size selected at baseline. The primary goal is to determine how many patients in each treatment group achieve complete closure of the target wound (skin re-epithelialization without drainage) by Month 1.
Patients will return for follow-up visits around Days 14, 30, and 60 to assess wound healing using the ARANZ SilhouetteStar™ device, with the target wound measured at each visit until it is documented as closed. Secondary assessments include changes in total wound burden based on body surface area (BSA), itching, and incidence of skin infections across the entire body. Itching will be evaluated at multiple time points but will not be recorded as an adverse event. Patients who complete this trial may be eligible to enroll in an open-label extension study (SD-008) to receive active treatment. Safety will be monitored through adverse event reporting, tolerability assessments, and physical examinations, with urine pregnancy testing performed at the investigator's discretion.
We will gather feedback from healthcare providers and pregnant or up to two years postpartum adult women to better understand their needs and experiences related to traumatic stress and alcohol use during pregnancy. We will conduct focus groups with maternal health and mental health professionals, as well as conduct 60-minute interviews along with a survey/questionnaire with pregnant women who are experiencing both alcohol-related challenges and trauma-related symptoms. What we learn from these conversations will help us tailor services to address the unique needs of women during pregnancy and the postpartum period.
This study is enrolling patients with severe tricuspid regurgitation. Tricuspid regurgitation happens when your heart's tricuspid valve does not close tightly. The study will look at the safety and function of the investigational VDyne Transcatheter Tricuspid Replacement System (VDyne System). The VDyne System (Study Device) is experimental, which means it is not approved by the Food and Drug Administration (FDA), or any other relevant regulatory authority for commercial use. The VDyne System consists of an artificial heart valve and tools to place the valve into your heart. The VDyne Valve is designed to replace your tricuspid heart valve without open-heart surgery.
This study is randomized, meaning you will be assigned by chance to receive either the Study Device or the EVOQUE system, which is commercially approved by the FDA. The EVOQUE System is approved for use in the European Union (EU) (CE Mark) and the US (FDA).
Participation in this study will last about 5 years and include about 11 visits. Study related procedures included in these visits are physical exams, questionnaires, walking and moving from sitting to standing tests, blood tests, electrical measure of your heart's performance, ultrasound imaging of your heart, insertion of a thin tube to measure pressures through a blood vessel and monitor blood flow through your heart, and X-rays.
There are risks associated with the study such as: bleeding, injury to the system that controls your heartbeat, movement of the VDyne Valve from its implanted location, irritation of your esophagus, pain, inflammation, and skin/tissue injury. There are also risks related to study procedures such as other risks related with putting you to sleep for the procedure (general anesthesia) or other study related testing risks and the loss of confidentiality. Not all risks are known at known at this time. There may be benefit to you, although this cannot be guaranteed, but the information gained from you being in this study may benefit others with this condition in the future
Iron deficiency is the most common nutritional deficiency worldwide and is a leading cause of anemia, which can result in symptoms such as fatigue, weakness, shortness of breath, and reduced quality of life. This study aims to better understand why some people develop iron deficiency by examining how the body absorbs iron and how this may relate to diet and proteins involved in iron absorption. Participants will complete questionnaires, provide blood, urine, and stool samples, undergo an oral iron absorption test, and complete a short food diary. If an upper endoscopy is already being performed as part of routine medical care, one additional biopsy sample will be collected during the procedure for research purposes. The information gathered may help improve the diagnosis and evaluation of iron deficiency anemia in the future. Participation involves two study visits over approximately one month. There is no direct medical benefit to participants, but the knowledge gained may benefit future patients with iron deficiency anemia.
This is a study to test the safety of ALN-6400 which is considered to be "an investigational drug", meaning it is not currently approved by regulatory authorities, including the United States Food and Drug Administration (FDA), for the treatment of any disease and find out what effects, if any, it has on people with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB). ALN-6400 works by lowering protein in the blood breakdown blood clots and reduce bleeding in women ages 16-45. This study is a 24 week study with an optional extension period of 84 weeks.
This study is being offered for patients with metastatic pancreatic adenocarcinoma who have tried available approved treatments and do not have satisfactory treatment options or are unable to participate in a clinical trial. The purpose of this Expanded Access Program (EAP) is to provide access to daraxonrasib, an investigational drug that has not yet been approved by the FDA.
Daraxonrasib is an oral tablet. Clinic visit are every 28 days to receive a new supply of daraxonrasib. Participation is expected to last until whichever of the following occurs first - 1: you are no longer benefiting from daraxonrasib; 2: daraxonrasib becomes commercially available (FDA approved); 3: Revolution Medicines, the Sponsor of the EAP, the IRB, or the FDA decides to stop the EAP.
Risks include skin rash, diarrhea, nausea and vomiting, mouth sores, and swelling. This EAP is not a research study. Its main goal is to provide access to daraxonrasib to patients who may benefit based on what information is known about the drug so far. There is no guarantee that your cancer will get better as a result of receiving daraxonrasib. It is possible that you will show no improvement or that your cancer could get worse.
This study is for patients that have been diagnosed with advanced solid tumors. This study is testing an investigational drug called At0365-P1-01. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). The primary purpose of this study is to evaluate how safe the drug is, how well participants can handle the drug, how it moves through the body, how it affects the body, and if the drug helps treat the cancer of AT03-65. AT03-65 is administered via intravenous (IV) infusion. Participants can continue to receive this study drug until it no longer gives them benefit. The estimated study duration is approximately 30-35 months for subjects screening, treatment and follow-up.