The purpose of this study is to evaluate investigational treatments (study drug) in people with gastroesophageal adenocarcinoma that has progressed (become worse) after at least 2 prior treatments.
The goal is to determine the optimal dose level, safety, and tolerability for the study drug MK2870-015. This is a phase 3 study; MK2870-015 is not FDA approved by the U.S. Food and Drug Administration (FDA). Treatment for this study may be up to 3 years. The procedures include taking study drug intravenously, blood and urine samples, MUGA scans and CT scans. Risks include diarrhea, nausea, vomiting, hair loss, weight loss, high blood sugar, and sore throat. You may or may not receive a direct benefit from participating in this trial, however, information learned from the trial may help other people in the future.
This study is a registry for patients who have sickle cell disease (SCD), including pregnant women. A registry is a database of patient health and demographic information that is used to understand how a disease affects different people based on, for example, their other medical conditions, their age, or their treatment regimen.
This study is being done to collect information about how SCD affects you and to try to understand what characteristics of your disease may predict whether you have complications from your disease in the future.
This is a is a Phase 1, open-label, dose-escalation, and dose-expansion multicenter study to evaluate the safety, tolerability, pharmacokinetic (PK) profile, and clinical activity of SNDX-5613 in combination with intensive chemotherapy in participants with newly diagnosed Acute Myeloid Leukemias harboring alterations in KMT2A, NPM1, or NUP98 genes. The primary purpose of this study is to identify the maximum tolerated dose of SNDX-5613 (Revumenib) to be used in both a combination with intensive chemotherapy and alone. SNDX-5613 (Revumenib) is an "investigational" (not yet FDA approved) treatment. The study will enroll approximately 76 patients in 3 phases with each receiving cycles of chemotherapy followed by SNDX-5613 in the Induction and Consolidation phases followed by SNDX-5613 alone in the Maintenance Monotherapy phase if eligible. The study includes screening, treatment, safety follow-up, and survival follow-up periods over the course of 18 months. The main risk is that medical treatments often cause side effects. Patients may have none, some, or all of the effects listed or not listed in the protocol, and they may be mild, moderate, or severe. There is no direct benefit in participating in this study.
The primary objective of this study is to evaluate the safety and the Clostridioides difficile infection (CDI) recurrence rate in participants who receive a 14-day course of VE303 or matching placebo.
Other objective is to measure health-related quality of life and daily CDI symptoms in participants treated with VE303 versus placebo.
The purpose of this study is to evaluate the safety and effectiveness of acoramidis (AG-10) and determine if it can help people with the genetic TTR variant that can cause ATTR-CM. The sponsor (Eidos) is conducting a research study to see if the study medicine will help in slowing the development and/or progression of a rare disease called Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM). AG-10 is an investigational drug. "Investigational" means that AG-10 is not yet approved for use in any settings outside of clinical research studies like this one and is considered experimental. Cardiomyopathy is a disease of the heart muscle that makes it harder for the heart to pump blood to the rest of the body. AG-10 has been shown to reduce the level of TTR in the blood of animals and healthy volunteers tested to date. Reducing the amount of TTR in your blood may reduce the amount of amyloid deposits in your body and may keep your cardiomyopathy from getting worse over time.
If you meet the requirements and agree to participate in this study, you will go through a 2 part screening period and if approved you will begin treatment. You will be randomized, like flipping a coin, to drug or placebo and take two pills once every day for 60 months.
Your participation in this study will last up to 60 month and will consist of clinic visits and follow up visits at home via the telephone. Some tests requires of you will include an echo, ECG, cardioradionuclide scans (scans that use radioactive agents to look at images of the heart like a PET scan), MRIs, blood tests, nerve conduction test, urine samples, and skin biopsies.
The purpose of the study is to determine whether FINErenone reduces total (first and future) Heart Failure events and cardiovascular death compared with placebo in patients hospitalized with acute decompensated Heart Failure with Mid-Range Ejection Fraction and Heart Failure with Preserved Ejection Fraction (HFmrEF/HFpEF. 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. Participation in this study will last approximately 36 months. During the study period subjects will be asked to attend regular study visits with the research coordinator. These visits will include such activities such as vitals, blood tests and questionnaires. There will be 8 visits as part of participation in this clinical trial. Participants will be randomized to either the treatment group (and receive the medication) or the control group (and not receive the 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 flipping a coin). 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 study is for subjects that have been diagnosed with advanced high grade serious ovarian cancer. The investigational drug used in this study is Sovilnesib. The main purpose of this study is to is to establish the recommended phase 2 dose, which will be considered the optimal dose, of Sovilnesib. Additionally, this study will examine the safety, tolerability and preliminary efficacy of Sovlinesib. The total time you will be on the study treatment will depend on if you have any unwanted side effects and how your cancer is responding to treatment.
The purpose of this study is to evaluate investigational treatments (study drug) for locally advanced or metastatic solid tumors with oncogene amplifications to determine if any of these study treatments improve overall survival as compared to standard treatments.
The goal is to determine the optimal dose level, safety, and tolerability for the study drug BBI-355. This is the first study to test the study medicine BBI-355 in humans; BBI-355 is not FDA approved by the U.S. Food and Drug Administration (FDA). Treatment for this study may be up to 3 years. The procedures include taking study drug orally, blood and urine samples, diary entries, and CT scans. Risks include diarrhea, nausea, vomiting, fatigue, muscle weakness, dizziness, and headaches. You may or may not receive a direct benefit from participating in this trial, however, information learned from the trial may help other people in the future.
The purpose of this study is to evaluate the safety and efficacy of the study drug ZILRETTA (Triamcinolone Acetonide, extended release) compared to a placebo (saline solution) and the current treatment of TCA-IR (Triamcinolone Acetonide, immediate release). The study drug is administered through an ultrasound-guided injection to the affected shoulder. Forty percent of subjects will receive Zilretta, forty percent will receive TCA-IR and twenty percent will receive placebo.
The study Drug ZILRETTA, is currently FDA-approved for knee osteoarthritis and is being investigated in this study for treatment of shoulder osteoarthritis. The study population consists of adults 50-85 years of age, BMI under 40, who have been diagnosed with Osteoarthritis of the shoulder by x-ray and has had pain for over 15 days out of the past month. The study has 10 study visits over 24-week's and will include, but is not limited to a questionnaire, medical examination, shoulder x-ray and blood draws. Each visit should last less than 1 hour, depending on the procedures performed. The most common side effects of the study drug are joint pain, headaches, upper respiratory infections, back pain, joint swelling, and cold symptoms.
The purpose of this research is to assess a group-based telehealth parenting program for child ages 2-6 years old with a neurodevelopmental diagnosis and behavior problems. Families will complete an online intake assessment, 6 group-based telehealth PCIT sessions, and an online post intervention assessment. Families will be compensated for their time.