The purpose of this registry study is to gather more information to answer research questions about upper tract urothelial carcinoma (UTUC) and the use of Jelmyto as a treatment. Jelmyto is not an experimental medication. It has been approved by the Food and Drug Administration (FDA) in the United States for the treatment of low grade UTUC since April 2021. For this study, data will be collected from the medical record including type and location of UTUC, treatments and surgeries received, and health status following treatments and surgeries. Approximately 400 people will participate in this study. Participation in this study will last three (3) years.
Patients with chronic pancreatitis often suffer from severe abdominal pain that reduce their quality of life. The major purpose of this study is evaluate the safety and efficacy of an infusion of donor derived mesenchymal stem cells to relieve chronic pain. After cell infusion into the vein, the participant will be followed for 6 months to evaluate their pain and other outcomes. There are a total of 5 clinic visits with a total study participation of up to 7 months.
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.
This study aims to investigate innovative approaches to managing chronic pain and opioid use. This study consists of two phases, each offering different treatment options. Participation is voluntary.
This study will sequentially evaluate three novel and scalable interventions for at-risk individuals on long term opioid therapy for chronic pain: (1) low-dose transdermal buprenorphine initiation without a period of opioid withdrawal; (2) a brief Cognitive Behavioral Intervention for pain (CBI); and (3) transcranial magnetic stimulation by examining standardized repeated measures of clinical outcomes at baseline, during treatment, and at follow-up.
Phase 1:
In this initial phase, all participants will have a 1-week open-label trial of buprenorphine (worn as a patch on the arm, shoulder or upper-back). This trial aims to assess the safety and effectiveness of buprenorphine in managing chronic pain and opioid use. During this phase, participants will have the opportunity to experience the effects of buprenorphine under close monitoring.
Phase 2:
After completing Phase 1, participants will have the opportunity to choose their next course of treatment. They can decide to continue with buprenorphine, and undergo a 1-week trial of either real buprenorphine or a placebo (an inactive substance). They will be randomly assigned to receive either real or placebo buprenorphine. If participants respond well to buprenorphine treatment, they may continue the medication under the care of their physician.
Alternatively, participants can explore an alternative treatment called repetitive transcranial magnetic stimulation (rTMS) in Phase 2. If they opt for rTMS, they will receive either real rTMS or a sham version interspersed with cognitive-behavioral therapy for pain. Participants will be randomly assigned to receive either real or sham rTMS.
In both phases, participants will receive close monitoring and attend regular study visits to assess safety and progress. Throughout the study, they will be asked to complete questionnaires about pain, functioning and opioid use, undergo physiological monitoring and blood samples will be collected at specific points.
It's important to note that there are potential risks associated with the study medication, such as difficulty sleeping, nausea, and dizziness. Additionally, for the rTMS arm, there is risk of mild headache, pain at the stimulation site, and there may be unknown risks related to the brain stimulation.
Participants' experience in Phase 1 will involve an open-label trial of buprenorphine, and participants' decisions in Phase 2 will determine the treatment path. While the effectiveness of these treatments is uncertain, participants will receive thorough monitoring throughout the study, and have the option to withdraw at any time. Improvement in participant symptoms is possible but not guaranteed.
This research is all about investigating a potential treatment for a specific type of advanced lung cancer. The cancer cells have a protein called c-Met that's acting a bit too excited, driving the cancer to spread and resist treatment. This study focuses on two treatments: one is called Telisotuzumab Vedotin (ABBV-399), which is a smart combination of an antibody and a drug that can stop this protein's activity. The other treatment is called Docetaxel. The researchers have already found in smaller studies that Telisotuzumab Vedotin could be promising for this type of cancer, especially in patients whose previous treatments didn't work well. Now they want to see if it can really make a difference in the long run. They'll be comparing Telisotuzumab Vedotin and Docetaxel to figure out which one is better at helping patients live longer without their cancer getting worse. In this study, participants will be randomly divided into two groups, like flipping a coin. One group will get a new medicine called telisotuzumab vedotin through a vein in their arm every two weeks. The other group will receive a treatment called docetaxel through a vein every three weeks. People in both groups will keep getting their assigned treatment until their doctors say it's time to stop based on certain criteria. Throughout the study, participants will have scans like X-rays or MRIs to see how their tumors are doing. These scans will happen at the beginning and then every few weeks for the first year, and less often as time goes on.Even if someone stops their treatment for a reason other than their cancer getting worse on these scans, the researchers will keep track of them until their cancer gets worse or until they decide they don't want to be in the study anymore.The whole study is expected to last around 38 months, which is a little over 3 years. They'll keep following up with the participants, either by talking to them, checking their medical records, or other appropriate methods, until the participants decide to leave the study or pass away. This study is a big step towards finding better options for people battling this tough form of lung cancer.
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.
Patients with large abdominal aortic aneurysms (AAA) (>5.5cm) are recommended to have surgery, but there is no therapy for small AAA (3-5cm). The purpose of this study is to collect ultrasound images and blood from patients with normal abdominal aorta as well as from patients with small AAA. Study participants include patients scheduled for an aortc ultrasound aorta and may fall into one of these categories. By analyzing the aortic wall ultrasound images and the blood levels of particular proteins, we hope to identify a target for drug therapy to stop AAA growth. Voluntary participation in this study does not change the original intent of the ultrasound and does not change patient treatment. The pictures gathered here are in addition to the standard ultrasound and cause no additional risk. Participation would include the standard aorta ultrasound, specific extra pictures (less than 5 minutes to collect), and collection of a blood sample. If a participant's aorta is within normal limits, no additional ultrasound visits or study visits will be indicated. If the participant has a small AAA, standard of care would be to return once per year for repeat ultrasound. Ongoing participation in the study will be to have the extra ultrasound images captured and repeat blood sampling, for a total of 3 years.
The study is being conducted in order to assess the long-term safety of rocatinlimab in adult and adolescent subjects with moderate-to-severe atopic dermatitis (AD). The study population will include subjects who completed an end of treatment duration visit in a parent study and meet eligibility criteria. Subjects will be randomized to receive a dosage of rocatinlimab based on age and their previous dosage from the parent study. The total duration of participation, including the parent study, will be up to 2.5 to 3 years, with a safety follow-up period after the last dose of investigational product at week 104.
This study involves research, and participation is voluntary. The purpose of the study is to see if a single dose and multiple doses of the study drug, AV 380, are safe and tolerated in cancer participants. This study will also help to look at how AV 380 behaves inside the human body (called pharmacokinetics), how the body responds to AV 380 (called pharmacodynamics), and how the immune system responds to AV 380 (called immunogenicity) when administered along with the standard anticancer treatment that a participant would usually receive if they were not in this study. First, the participant will have some tests to decide if it is safe for to join the study. These tests include blood and urine sampling, electrocardiogram, vital sign measurement, physical examination, cachexia assessment,which checks changes in proteins that affect appetite and computed tomography (CT) scan. If the study doctor thinks a participant is eligible to join, they will be assigned to one of 5 cohorts (a group of people with shared characteristics) (corresponding to 5 dose levels of AV 380) in which then they will receive AV 380 together with the Standard of Care treatment chemotherapy. Participants will need to visit the study site approximately every 1 week for the first 8 weeks and then every 2 weeks. In addition to the above-mentioned tests, then they will be also asked to have exercise tests and complete questionnaires during the study. Participants will also have post study treatment follow up visits 3 times after completion or discontinuation of AV 380. The total duration of the study is up to 7 months (including follow-up visits). Some reasonably foreseeable risks or discomforts with this study include chills, headache, and elevations in an enzyme called creatine phosphokinase which is found mainly in the heart, brain, and skeletal muscle. There is no direct benefit with participating in this study, but the information we get from this study will us improve treatment for people in the future.
This study is seeking participants with arrhythmogenic cardiomyopathy (ACM), also known as arrhythmogenic right ventricular dysplasia/cardiomyopathy (ARVD/C), due to a genetic abnormality known as a PKP2 variant. ARVD/C is an inherited disease where the muscle tissue in the right ventricle, one of the lower pumping chambers of the heart, dies and is replaced with scar tissue. This causes a weakened heart muscle and disrupts the heart's electrical system which can lead to heart failure and/or fatal heart rhythms. This study is looking at the safety and effectiveness of an investigational medication, meaning it is not yet approved for use by the Food and Drug Administration (FDA). The study medication is a gene therapy called LX-2020, and is designed to add new PKP2 genes to replace the faulty ones so your cells can make the correct PKP2 genes. The study medication is given via an intravenous (IV) line meaning in a vein. Participation in this study involve up to 25 visits including a hospitalization over the course of 1 year with an additional 4 years of follow up afterwards. Study related procedures include a variety of heart testing like electrocardiogram (ECG), echocardiogram, a test that records a tracing of the heart's electrical activity, Echocardiogram, (echo) a test that uses ultrasound to capture moving images of the heart, magnetic resonance imaging (MRI), a test that shows an image of the heart and surrounding structures, sample collection including blood, urine, tissue, nasal mucus, saliva and stool, liver ultrasound, questionnaires, physical exams, and at least a two night stay in the hospital. Medications to suppress (meaning weaken) the immune system, before receiving the LX2020 are also required. Risks associated with gene therapy include an immune response that may cause inflammation in the liver, heart or other organs. It may damage red blood cells, cause a low platelet count or cause the formation of small blood clots. There are also risk related to the study procedures including bleeding associated with the heart biopsy, risks related to drawing blood, risks of radiation, and loss of confidentiality. There is potential benefit and in the future, others with ACM may benefit from the knowledge gained from this study.