A Randomised Open-Label Phase III Study of Saruparib (AZD5305) Plus Camizestrant compared with Physician's Choice CDK4/6 Inhibitor Plus Endocrine Therapy or Plus Camizestrant for the First-Line Treatment of Patients With BRCA1 BRCA2 or PALB2 Mutations and Hormone Receptor Positive HER2-Negative (IHC 0 1+ 2+/ ISH non-amplified) Advanced Breast Cancer (EvoPAR-Breast01)
This study is testing how well a new medicine, saruparib (AZD5305), works when combined with camizestrant compared to other common treatments for advanced breast cancer. The patients in the study have a specific type of cancer that is hormone receptor (HR) positive, HER2 negative, and caused by mutations in BRCA1, BRCA2, or PALB2 genes. Patients will be randomly assigned to one of three groups: saruparib with camizestrant, a doctor’s choice of CDK4/6 inhibitor plus endocrine therapy (ET), or a CDK4/6 inhibitor with camizestrant. The study has four parts: testing for specific genetic markers, screening to see who qualifies, treatment, and follow-up to check progress. Treatment will continue as long as the cancer is under control, side effects are manageable, or the patient chooses to stop. After treatment, doctors will monitor safety, survival, and cancer growth.
A Randomized Comparative Effectiveness Study of Staged Complete Revascularization with Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone in Patients with Symptomatic Aortic Valve Stenosis undergoing Elective Transfemoral Transcatheter Aortic Valve Replacement: The COMPLETE TAVR Study
This study is being conducted in Aortic Stenosis patients who have undergone successful TAVR with a balloon expandable heart valve who also have coronary artery disease (CAD) or narrowing of the heart arteries.The study will compare:1. opening all suitable stenosis or blockages with an additional procedure (either during the same hospitalization as the TAVR or as a separate procedure) called percutaneous coronary intervention (PCI). This procedure opens or widens the stenosis with a stent thereby allowing blood to flow to the heart muscle. This procedure will happen within 45 days of your TAVR procedure. You will also receive optimal medical therapy.OR2. treating these additional stenosis or blockages with medication only and not opening them with an additional PCI procedure.
A Randomized controlled study of a health literacy-informed technology-based approach to support safe medication use by parents after discharge of infants from the neonatal intensive care unit
This is a randomized controlled study of parents of children to be discharged from the neonatal intensive care unit at Bellevue and Elmhurst hospitals. A total of 425 subjects will be recruited across two sites over preparatory phases and two primary study phases. Subjects will be assigned to 1 of 3 intervention groups: usual care, HELPix (health literacy-informed written materials and verbal counseling in addition to usual care), or HELPix + TECH (a health literacy-informed web application in addition to HELPix and usual care). Phase A is a pilot study, in which 120 parents will be enrolled at Bellevue Hospital and randomly assigned to usual care or HELPix + TECH. Phase B will take place at both Bellevue and Elmhurst Hospitals, and 225 subjects will be randomly assigned to usual care, HELPix, or HELPix + TECH. Parents will be screened and recruited, written informed consent will be obtained, and a brief survey will be administered on the day of the child’s expected discharge from the neonatal intensive care unit Subjects will then receive usual care followed by the intervention if randomized to one of those groups (Visit 1, Day 0). Medication knowledge, dosing, and adherence will be assessed in-person at a subsequent follow-up visit (Visit 2, Day ~1-7). Adverse events will be assessed via phone call (Visit 3, Day ~30-45). Additional data will be assessed using a chart review. There will be a preparatory phase before each of the 2 phases. For the preparatory phase before Phase A: 10 cognitive interviews will be conducted to assess parent comprehension/ acceptability of HELPix followed by 20 parent interviews focused on HELPix app usability.For the preparatory phase before Phase B: 12 cognitive interviews will be conducted to assess parent comprehension/ acceptability of HELPix. In addition to the cognitive interviews, 3 rounds of feedback with 6 parents per round will be conducted to iteratively refine the HELPix digital app. Once adapted, 20 parents will be asked about HELPix app usability.
A Randomized Double-blind Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Lumateperone for the Treatment of Major Depressive Episodes (MDEs) Associated with Bipolar I or Bipolar II disorder (Bipolar Depression) in Pediatric Patients Aged 10 to 17 Years
The purpose of this study is to investigate how “CAPLYTA (lumateperone)” works on symptoms of Major Depressive Episodes (MDEs) associated with Bipolar Disorder in young people (10-17 years of age). This study will last about 9 weeks and will involve 9 visits to our center over this period.
A Randomized Double-Blind Placebo-Controlled Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in Subjects with Progressive Pulmonary Fibrosis (PPF)
The purpose of this study is to evaluate the safety and efficacy of multiple doses of AP01 compared with placebo (a study treatment containing no active ingredients), over 52 weeks in participants with Progressive Pulmonary Fibrosis (PPF). During the study, you will continue your current PPF treatment regimen; the study drug will be added to your current treatment regimen.
A Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiranin the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease with METAVIR Stage F2 to F4 Fibrosis
This is a phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of fazirsiran (TAK-999, previously called ARO-AAT) in the treatment of subjects aged 12 to 75 years (inclusive) with AATD-LD moderate to advanced fibrosis and compensated cirrhosis.The study will enroll subjects with PiZZ AATD-LD with METAVIR stage F2, F3, or F4 liver fibrosis. Approximately 126 up to 140 subjects are planned to be randomized 1:1 to receive either placebo or fazirsiran administered subcutaneously (SC). Subject randomization will be stratified according to METAVIR stage (F2 or F3 vs F4). The F4cc population will be capped at 25% of the total subjects with the remaining 75% being comprised of F2 and F3 subjects.
A RANDOMIZED DOUBLE BLIND PLACEBO-CONTROLLED STUDY OF THE EFFECTS OF STELLATE GANGLION BLOCK ON NEURAL ACTIVITY AND SYMPTOMS IN PARTICIPANTS WITH POST-TRAUMATIC STRESS DISORDER
We are doing this research study to learn more about how a Stellate Ganglion Block (SGB) procedure changes activity in the brain in subjects with Post-traumatic Stress Disorder (PTSD). PTSD is a mental health disorder that some people develop after witnessing or experiencing a traumatic event. People who have PTSD re-experience the traumatic event and feel the same fear and discomfort that they associated with the trauma long after the danger has gone.
A Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients with Primary Sclerosing Cholangitis (VISTAS)
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, single, inferentially seamless, adaptive design clinical study that will be conducted in 3 parts.The primary and secondary objectives and endpoints will be evaluated in participants with PSC who have a baseline score of =4 on the Adult Itch-Reported Outcome (ItchRO), as assessed during the single-blind placebo run-in period during the core study.
A Randomized Open-label Multicenter Phase 2 Study Evaluating the Efficacy and Safety of Zilovertamab Vedotin (MK-2140) Plus R-CHP Versus Polatuzumab Vedotin Plus R-CHP in Treatment-na ve Participants With GCB Subtype of Diffuse Large B-cell Lymphoma (DLBCL)
This is a Phase 2 study testing two different medicines to treat a blood cancer called GCB DLBCL(diffuse large B-cell lymhoma). Zilovertamab Vedotin (an antibody-drug conjugate) is a new drug that targets a protein called ROR1, which shows up in many blood cancers. The goal of this study is to see if more patient's cancer gets better when they get atreatment of Zilovertamab Vedotin (MK-2140) along with other medicines called R-CHP compared to patients who get a different treatment called Polatuzumab Vedotin with R-CHP. R-CHP is a combination of rituximab, cyclophosphamide, doxorubicin, and prednisone medicines. Patients with the GCB subtype of DLBCL will be split into two groups randomly.The first group will get Zilovertamab Vedotin + R-CHP and second group will get Polatuzumab Vedotin + R-CHP. The study team will then decide which treatment can give patients better results with fewer side effects. Allpatients will have frequent medical check ups to see how they are doing. Blood samples will be taken from all patients to see how their bodies are handling the study medicine. The study team will have special scans done for all patients to see how the cancer is responding to the study medicines. The study team will be monitoring the patients for potential side effects and safety concerns.
A Randomized Open-label Phase 2/3 Study of Izalontamab Brengitecan (BMS-986507) versus Platinum-based Chemotherapy in Patients with EGFR-mutated Non-small Cell Lung Cancer and Disease Progression on EGFR Tyrosine Kinase Inhibitor Therapy
This is Phase 2/3 study testing a new medicine called iza-bren (also known as izalontamab brengitecan or BL-B01D1) for treating advanced lung cancer that has a specific mutation called EGFRmt. Iza-bren works in a special way: it targets two proteins, EGFR and HER3, which are often involved in cancer growth. It delivers a medicine that can kill cancer cells directly to the tumor. The goal of this study is to find the best dose of iza-bren and see how well it works compared to standard chemotherapy (a combination of platinum and pemetrexed). This study has two parts: phase 2 and phase 3. In Phase 2, the patients are randomly put into one of three groups. Once enough patients have been treated in Phase 2, the study will choose the best dose of iza-bren and move to Phase 3. In Phase 3, patients will randomly get either the chosen dose of iza-bren or standard chemotherapy. This phase will compare how well iza-bren works, how safe it is, and how well people can tolerate it, compared to the usual chemotherapy treatment (platinum-pemetrexed) for EGFRmt lung cancer. This part of the study is for patients whose cancer keeps growing even after using another medicine called EGFR-TKI. All patients will have frequent medical check ups and blood samples taken to see how their bodies are handling the study medicine. The study team will have special scans done for all patients to see how the cancer is responding to the study medicines. The study team will be monitoring the patients for potential side effects and safety concerns.