A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment With or Without Dialysis PHYOX7
The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.Primary hyperoxaluria is typically diagnosed by measuring oxalate levels in urine. However, as kidney function decreases, the renal excretion of oxalate also decreases and may no longer reflect daily oxalate loads (Perinpam et al., 2017). Decreasing renal excretion of oxalate results in increasing Pox levels (Hoppe et al., 1998, Hoppe et al., 2009). Because a decrease in Pox is reasonably likely to predict clinical benefit due to its causal role in systemic oxalosis in CKD Stages 3b to 5, Pox may be a more relevant endpoint in patients with PH who have severe renal impairment (Milliner et al., 2020).
A Phase 2 Randomized Double-blind Placebo-controlled Dose-finding Study to Evaluate the Efficacy and Safety of ALXN2050 in Adult Participants with Proliferative Lupus Nephritis (LN) or Immunoglobulin A Nephropathy (IgAN)
This is a phase 2, randomized, double-blind, placebo-controlled, dose-finding study to evaluate the efficacy and safety of ALXN2050, an oral complement FD inhibitor, in adult participants with proliferative lupus nephritis (LN) or Immunoglobulin A Nephropathy (IgAN).
A Phase 2 Randomized Double-Blind Placebo-Controlled Study to Assess the Efficacy and Safety of Barzolvolimab (CDX-0159) in Adults with Active Eosinophilic Esophagitis (The EvolvE Study)
To evaluate efficacy of barzolvolimab, compared to placebo, in reducing esophageal intraepithelial infiltration of mast cells as assessed by peak esophageal intraepithelial mast cell (PMC) count in EoE patients.
A Phase 2 Randomized Study of Adjuvant Immunotherapy With the Personalized Cancer Vaccine mRNA-4157 and Pembrolizumab Versus Pembrolizumab Alone After Complete Resection of High-Risk Melanoma (mRNA-4157-P201)
This is an open-label, randomized, Phase 2 study. Approximately 150 eligible patients will be randomly assigned in a 2:1 ratio to the combination arm (approximately 100 patients who receive mRNA-4157 and pembrolizumab) and the control arm (approximately 50 patients who receive pembrolizumab alone). Patients with completely resected cutaneous melanoma at one of the following stages: ? Stage IIIB, only if relapsed within 3 months of initial surgery of curative intent. That is, the patient must have relapsed within 3 months of their first surgery of curative intent and subsequently had a second surgery of curative intent before entering this study (as such, newly diagnosed Stage IIIB patients are not eligible).? Stage IIIC.? Stage IIID.? Stage IV.Patients must have had complete resection (surgery of curative intent) within 13 weeks before study enrollment, and must be disease free at study entry (screening). Once a patient is enrolled, they will be randomized and should commence pembrolizumab treatment as soon as possible (ideally within 13 weeks of their surgery of curative intent). For patients randomly assigned to the mRNA-4157 plus pembrolizumab combination arm:? A pembrolizumab run-in period, typically 2 pembrolizumab cycles, will occur while mRNA-4157 is being manufactured.? Once a patient’s mRNA-4157 is available, the combination treatment period will commence. The first dose of mRNA-4157 will be administered with the next dose of pembrolizumab in order to achieve synchronous combination dosing in 21-day cycles.All patients on both arms of the study may continue on pembrolizumab until disease recurrence, unacceptable toxicity, or they undergo 18 total cycles (approximately 1 year of treatment), whichever is sooner. Crossover to the mRNA-4157 and pembrolizumab combination arm is not permitted for patients who relapse following pembrolizumab monotherapy. Recurrence-free survival (RFS) is the primary endpoint of the trial and is defined as the time between the first dose of pembrolizumab and the date of first recurrence (local, regional, or distant metastasis), new primary melanoma or death (from any cause), whichever occurs first. Patients will be assessed for recurrence by radiological imaging.
A Phase 2 Randomized Study of Osimertinib Versus Osimertinib Plus Chemotherapy for Patients with Metastatic EGFR-Mutant Lung Cancers That Have Detectable EGFR-Mutant cfDNA in Plasma After Initiation of Osimertinib
This study is about testing two different treatments for a type of lung cancer called EGFR-mutant lung cancer. The first treatment is a medicine called osimertinib, which patients will take by mouth every day. They'll take it for three rounds, and then the doctors will check their blood to see if themedicine is working. If the medicine is not working, some patients will getanother treatment called chemotherapy along with osimertinib, while otherswill keep taking osimertinib alone. The doctors will monitor how well thetreatments are working by taking pictures of the inside of the body andchecking the blood again if the cancer starts to grow again.
A Phase 2 Study of EIK1001 in Combination with Pembrolizumab and Chemotherapy in Patients with Stage 4 Non-Small Cell Lung Cancer
This study aims to see how well a new drug, EIK1001, works when given with pembrolizumab and certain chemotherapies in patients with Stage 4 lung cancer who haven't had other treatments. Patients will getEIK1001 through an IV once a week, and pembrolizumab every three weeks for up to 35 cycles. There are two groups: one will get EIK1001 with pemetrexed and carboplatin, and the other with paclitaxel and carboplatin. At first, a small number of patients will get a low dose of EIK1001 to check for serious side effects. If it’s safe, more patients will get a higher dose. The main goal is to see if the treatment has few side effects. The study will also look at how well the treatment works against the cancer, how long patients live without the cancer getting worse, and other important health markers. Patients will have regular scans to check their cancer and will be watched closely for side effects. The treatment will continue until the cancer worsens, side effects are too severe, or other reasons the study teams decides is good enough for patient withdrawal. After treatment ends, patients will be followed for at least 90 days to monitor their health.
A Phase 2 Study of Futibatinib in Combination with PD-1 Antibody-based Standard of Care Therapy in Patients with Solid Tumors
A Phase 2 Study of Futibatinib in Combination with PD-1 Antibody-based Standard of Care Therapy in Patients with Solid Tumors
A Phase 2 Study of XmAb 20717 in Patients With Selected Gynecological Malignancies and High-Risk Metastatic Castration-Resistant Prostate Cancer
This is a Phase 2, multicenter, two-stage, open-label, parallel-group study designed to evaluate the efficacy and safety of XmAb20717 in patients with selected advanced gynecologic and genitourinary malignancies.
A Phase 2/3 Adaptive Double-blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Subjects Adult and Pediatric subjects With APOL1-mediated Proteinuric Kidney Disease
This is an adaptive Phase 2/3 study of VX-147 in subjects Adult and Pediatric with APOL1-mediated proteinuric kidney disease that is designed to select a dose of VX-147 and establish the efficacy and safety of the selected dose
A Phase 2a Double-Blind Randomized Active Controlled Parallel Group Study Evaluating the Efficacy Safety and Tolerability of Bezafibrate Administered in Combination with Obeticholic Acid in Subjects with Primary Biliary Cholangitis
Overview: This Phase 2a, randomized, DB, active-controlled, parallel-group study will evaluate the efficacy, safety, and tolerability of OCA (5 mg/day) administered in combination with two different BZF doses (100 and 400 mg/day) compared to BZF alone (100 and 400 mg/day) in up to 60 subjects with PBC over at least 12 weeks. Subjects will be randomized (1:1:1:1) to the following 4 treatments: Treatment A: BZF 100 mg IR; Treatment B: BZF 400 mg IR; Treatment C: OCA 5 mg + BZF 100 mg IR; and Treatment D: OCA 5 mg + BZF 400 mg IR. This study will use 100-mg and 200-mg immediate release (IR) formulation tablets of BZF.