This is a Phase 1/2, open-label, multi-center clinical study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of TSRA-196, a gene editing compound, in adults with severe alpha-1 antitrypsin deficiency (PiZZ genotype) and associated lung and/or liver disease. Participants will receive a single intravenous dose of TSRA-196 in a dose-escalation phase followed by dose-expansion cohorts.
The study will assess safety outcomes, pharmacokinetics, and changes in serum alpha-1 antitrypsin levels and lung function to determine whether TSRA-196 can safely increase functional AAT levels and inform selection of an appropriate dose for further clinical development.
The purpose of this study is to understand how exposure to harmful substances during military service may affect the health of Veterans with or without lupus. Lupus is an autoimmune disease that can increase the risk of cardiovascular problems.
We believe that Veterans who were exposed to toxic substances during their military service may develop more harmful antibodies that attack the lining of their blood vessels. These antibodies may contribute to poorer blood vessel and heart health, and could contribute to the development of lupus.
This study aims to improve our understanding of how toxic military exposures may increase the risk of blood vessel complications in Veterans with and without lupus. Ultimately, this research may help identify new ways to better prevent, monitor, or treat cardiovascular disease in this population.
Research procedures for this study will include:
1. The study team will check subject medical records to gather information about medical history and medications being taking. The study team may continue to follow updates in the medical record.
2. Subjects will be given a survey to assess military and occupational toxic inhalant exposures.
3. Subjects will have a brief physical examination during which vitals will be recorded (height, weight, heart rate, respiration, temperature). Women of childbearing ages will be asked for the date of their last menstrual cycle within the past 2 months.
4. Subjects will have blood pressure taken three times three minutes apart.
5. Subjects will then provide a urine sample. Urine collection will occur in a private restroom using a sterile container provided by the study team. For women of childbearing ages, a pregnancy dipstick test will be undertaken on urine to confirm subjects are not pregnant.
6. Subjects will undergo a blood draw where approximately 4 teaspoons of blood will be drawn.
This study is for participants with symptoms of mast cell activation (SMAC). The primary purpose of this study is to learn about clonal mast cell diseases. Clonal mast cell diseases are hard to diagnose because symptoms are not specific, and they can overlap with other diseases. The tools currently used by doctors to look for clonal mast cell diseases in the blood may not identify all patients. This study is being done to develop an investigational blood test that looks for a change in a gene called KIT. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). Two types of blood tests will be compared against each other. Participants in this study can expect to be in this study for about 6 months.
This study is for patients that have been diagnosed with squamous cell, adenocarcinoma, or adenosquamous cervical cancer. This study is testing an investigational drug called pembrolizumab. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). A computer will be used to assign you to one of the study groups. This process is called "randomization." Like flipping a coin, everyone has an equal chance of being placed in any group. The primary purpose of this study is to determine whether induction immunotherapy (IO) and chemotherapy prior to concurrent chemoradiotherapy plus immunotherapy (CCRT+IO) improves progression-free survival (PFS) compared to CCRT+IO alone. The study drug is given by infusion. Participants in this study can expect to be on the study for 7 years.
This is an open-label, long-term study assessing the safety and efficacy of abrocitinib in participants aged 2 years and older with moderate-to-severe AD. It includes an extension cohort and a de novo cohort to meet regulatory requirements for a minimum of 300 participants exposed to 52 weeks of abrocitinib. Extension cohort participants must have completed 16 weeks of treatment in parent studies B7451023 or B7451030 and remain eligible. De novo cohort participants must be aged 6 to under 12 years at enrollment and not have participated in previous abrocitinib studies. Enrollment for the de novo cohort will begin after enrollment in Study B7451023 is complete. The study will have two periods lasting up to 2 years or until commercial availability, whichever comes first. All participants will receive abrocitinib oral suspension.
This study is for patients who have been diagnosed with prostate cancer that is prostate-specific membrane antigen (PSMA)-positive and has spread despite treatment with another androgen receptor pathway inhibitor (ARPI). This study is testing an investigational drug called AAA817. "Investigational" means it has not been approved by the United States Food and Drug Administration (FDA). In this study, participants will be randomly assigned (like flipping a coin) to receive AAA817 alone, AAA817 with an androgen receptor pathway inhibitor (ARPI), or standard of care treatment. The primary purpose of this study is to determine whether AAA817, given alone or in combination with an ARPI, is safe and effective compared to standard of care treatments. This drug is given to participants as a radioligand therapy infusion. Participants in this study can expect to be in the study for up to 6.1 years, including two visits before starting treatment, visits every 4 weeks during treatment, and visits every 12 weeks during long-term follow-up for up to 5 years after treatment ends.. There will be a total of 9 patients enrolled locally.
This study compares two common laser treatments used to lower eye pressure in participants with glaucoma or high eye pressure. Both procedures, Selective Laser Trabeculoplasty (SLT) and Direct Selective Laser Trabeculoplasty (DSLT), are established standard-of-care medical treatments. Participants are randomly assigned by a computer to receive one of these two lasers to compare efficacy and safety. To maintain clinical consistency, both eyes of each participant receive the same assigned laser treatment during a single visit. There are no additional research-only appointments required beyond standard clinical care. The study team reviews medical records from the participants' standard 3, 6, and 12-month clinical follow-up visits. This research aims to help clinicians understand which treatment is more effective or efficient for future use. Participation is entirely voluntary and does not impact the quality of medical care provided at the institution.
The purpose of this study is to measure the efficacy and safety of KP-001 compared to placebo in patients with common venous malformations (VM), common lymphatic malformations (LM), or KTS/CLOVES syndrome. This phase 3, double-blind, randomized, placebo-controlled, parallel-group study will take place at multiple sites across North America. Vascular malformations like VM, LM, and KTS/CLOVES syndrome are serious, rare diseases with significant unmet medical needs. The study includes a 24-week double-blind treatment period with either KP-001 or placebo, followed by an open-label phase where all patients receive KP-001 up to Week 52. Patients weighing 40 kg or more will receive 100 mg of KP-001 once daily after breakfast, while those weighing less than 40 kg will receive a reduced dose based on their body weight.
LiveWell mBC is a group-based skills training program for women with metastatic breast cancer (mBC). The program teaches skills from dialectical behavioral therapy, an evidence-based psychotherapy, that have been adapted specifically to help women live as well as possible, with metastatic breast cancer. LiveWell mBC involves meeting with a small group of women with mBC plus two skills trainers 9 times total via telemedicine. Meetings include orientation plus 8 sessions learning and practicing skills including mindfulness, emotion regulation, distress tolerance and interpersonal effectiveness and they last 90 minutes each. We are interested in seeing whether the program can help women to balance their emotions and better manage distress (e.g., anxiety, sadness) and symptoms (e.g., fatigue, breathlessness, pain) that can be common when living with mBC.
Participants will be randomly assigned to one of two groups. They will have a 2 in 3 chance of being assigned to LiveWell mBC (experimental group) and a 1 in 3 chance of being assigned to Usual Care (control group). Women in the usual care group will continue to receive their standard oncology care. Participants in both groups will complete questionnaires four times total. Participants may be invited to participate in an exit interview about their experience. Participants will be compensated for completing questionnaires and the interview. For most people, participation will last approximately 4 months.
A single weight based dose of alcohol will be given to approximate an alcohol binge (aiming to achieve a blood alcohol concentration of approximately 0.08%) to investigate downstream effects on intestinal permeability and changes in hepatic steatosis. Several studies have been performed in humans under similar conditions and looking at similar things. The PI, Dr. Garth Swanson, conducted one of these studies previously. Here, participants will be given a weight-based dose (2mL vodka per kg body weight) of alcohol, with assessment of intestinal permeability via urine collection over the 24 hours directly following alcohol consumption, assessment of changes in hepatic steatosis by evaluation with fibroscan before and after alcohol consumption, and blood markers of endotoxemia and related inflammatory markers after. The weight-based dosing of alcohol was chosen to reflect previous studies looking at the effects of a single episode of alcohol binge use, with the goal of achieving a blood alcohol concentration of approximately 0.08%, the lower limit of what is needed to be considered an acute alcohol binge.