A Multi-Centre Study in Patients Undergoing Total Hip Arthroplasty with the Smith+Nephew CATALYSTEM Primary Hip
This study will evaluate the use of the CATALYSTEM Primary Hip System for patients who require a total hip replacement. The CATALYSTEM is approved for use in patients that require a primary (or first-time) total hip replacement. The CATALYSTEM is the device that is placed in the femur, or long leg bone. The objective of this study is to track the performance of the CATALYSTEM in patients that receive it as part of their primary total hip replacement based on feedback from the subjects and the study doctors up to 5-years after surgery.
A Multi-phase Dose-Escalation followed by an Open-label Randomized Crossover Study of Oral ASTX030 (Cedazuridine and Azacitidine Given in Combination) Versus Subcutaneous Azacitidine in Subjects with Myelodysplastic Syndromes (MDS) Chronic Myelomonocytic Leukemia (CMML) or Acute Myeloid Leukemia (AML)
Study ASTX030-01 is being conducted in subjects with myelodysplastic syndromes (MDS), MDS/myeloproliferative neoplasms (MPN) including chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML) who are candidates to receive treatment with single agent azacitidine based on local country approvals and/or local institutional standard practice.Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3.• Phase 1 is an open-label Dose Escalation Stage (Stage A) followed by a Dose Expansion Stage (Stage B) of oral ASTX030 (cedazuridine in combination with azacitidine). Multiple doses/formulations will be evaluated. New formulations will generally be investigated in Phase 1 Stage A. Approximately 54 (Stage A) and 24-64 (Stage B) subjects will be treated in Phase 1. As of Amendment 1, an assessment of food effect (12 subjects) on the PK of ASTX030 was implemented for Phase 1 Stage B and is described below and in Section 5.3.1.As of Amendment 2, each additional dose level/formulation selected by the DSRC forevaluation in Phase 1 Stage B will be evaluated in approximately 12-24 additional subjects or as determined by the DSRC (ie, if emerging data indicates that this dose level/formulation does not achieve parity with SC azacitidine, fewer subjects may be evaluated).• Phase 2 is a randomized open-label crossover study comparing oral ASTX030 to SCazacitidine administered using the once daily × 7 days dosing schedule for each 28-day cycle as detailed in Section 1.3.1. It is expected that up to approximately 60 subjects will be treated at the RP2D depending on the number of doses/formulations tested in Phase 2.• Phase 3 is a randomized open-label crossover study comparing oral ASTX030 FDC tablet(s) to SC azacitidine administered using the once daily × 7 days dosing schedule for each 28-day cycle as detailed in Section 1.3.1. Depending on the intrapatient variability observed during Phase 2, it is expected that between approximately 75 and 115 subjects will be randomized in Phase 3.This study is designed to assess the safety and tolerability, pharmacokinetics, and clinical activity of ASTX030 and identify an oral dose combination of ASTX030 that yields azacitidine systemic exposure that approximates parity to that of SC azacitidine alone (based on total cycle azacitidine AUC0-24).
A Multi-Phase Study Examining Hospital to Home Transitions for Children with Medical Complexity
The overarching objective of this study is to make it easier for parents of children with medical complexity (CMC) to take care of their children after discharge home from the hospital and reduce the chance of post-hospitalization morbidity (meaning bad outcomes such as readmissions) after discharge. CMC, or those with multiple chronic conditions, progressive conditions, or technology dependence, are at high risk for post-hospitalization morbidity. This study will take place in 3 phases at 2 sites: Bellevue Hospital Center (BHC) and Hassenfeld Children’s Hospital (HCH). We will recruit parents of CMC with a prior or current admission at these two sites, as well as pediatricians who care for these children in the inpatient setting for the following 3 aims: - In Aim 1, we will interview parents of CMC and pediatricians to understand their views on what makes it challenging, and what can make it easier, for parents to understand and follow the instructions they get from the hospital about how to take care of their CMC after leaving the hospital. We will also ask pediatricians what may make it difficult to provide relevant education to families. We hypothesize that we will identify several contributing factors.- In Aim 2, we will design a tool to make it easier for parents to understand and follow the discharge instructions for their CMC. We will use structure of existing tool, findings from Aim 1, and extensive interviews and testing of the tool with parents and pediatricians as we design the new tool. We hypothesize that we will successfully design a tool that will be usable by both parents and pediatricians.- In Aim 3, we will use a randomized controlled trial (RCT) to study the impact of the tool on parent comprehension and adherence (or how well they can follow) their child's discharge instructions, as well as its impact on post-discharge morbidity (such as readmissions and emergency department visits). Parents will be randomized to either receive usual hospital care and instructions or the intervention/tool (in addition to the usual care and instructions). We will also ask parents who receive the intervention about its usability. We hypothesize that, compared to subjects who receive usual care, subjects in the intervention group will have higher comprehension and adherence, and their children will have lower post discharge morbidity. Parents will find the intervention to be usable.
A multicenter open label Phase 2 study to evaluate the efficacy and saftey of Sutetnib maleate capsule in locally advanced or metastatic NSCLC
This study is for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR mutations. The main goal is to see if a medicine called sutetinib (which may be able to stop or slow down the cancer from growing) can help these patients. The study team wants to check if the medicine has few side effects and how it works in the body. They'll do this study with two groups of patients, one with the gene changes from the start, and the other group who got them later. The study team is doing this because there are no good treatments for these patients right now, and earlier tests showed this medicine might help some of them. Patients will take the medicine for a few weeks, and doctors will watch to see if it helps with the cancer.
A Multicenter Open-Label Study of RMC-6236 in Patients with Advanced Solid Tumors Harboring Specific Mutations in RAS
A Multicenter Open-Label Study of RMC-6236 inPatients with Advanced Solid Tumors Harboring Specific Mutations in RAS
A Multicenter Randomized Double-blind 2-Part Phase 2 Study to Evaluate the Efficacy and Safety of GS-1427 in Adult Participants With Moderately to Severely Active Ulcerative Colitis (UC)
The purpose of this study is to learn more about the study drug, GS-1427. This will include: Testing if 3 different doses of GS-1427 work to treat moderate to severe Ulcerative Colitis (UC) Testing if GS-1427 is safe for people with UC Testing how the body processes and responds to GS-1427
A Multicenter Randomized Double-Blind Placebo-Controlled Non-inferiority Crossover Study Evaluating the Safety and Immunogenicity of the Herpes Zoster Subunit Vaccine in Patients with Systemic Lupus Erythematosus
This randomized, double-blind, placebo-controlled, non-inferiority crossover study will evaluate the HZ/su vaccine in SLE patients in order to evaluate safety and immunogenicity in patients with variable baseline clinical activities, ages and immunosuppressant exposures. We hypothesize that HZ/su administration will be non-inferior to placebo with respect to the risk of moderate or severe SLE flare(s) occurring within 24 weeks of receiving the first dose of the assigned treatment. In addition, we hypothesize that immunogenicity of the vaccine in SLE patients will be at least 50% of levels observed in healthy subjects from prior large clinical trials.
A Multicenter Randomized Double-blind Placebo-controlled Phase 3 Study Evaluating the Efficacy and Safety of Subcutaneous Anifrolumab in Adult Subjects with Active Systemic Lupus Erythematosus
This is a Phase 3, multicenter, multinational, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of an SC treatment regimen of anifrolumab versus placebo in adult patients with moderately-to-severely active, autoantibody-positive SLE despite receiving SOC. Subjects must be receiving stable SOC prior to Screening consisting of any of the following: OCS monotherapy, OCS plus antimalarials and/or immunosuppressants (azathioprine, antimalarials, mycophenolate mofetil / mycophenolic acid, methotrexate, or mizoribine); or immunosuppressants with or without OCS; please refer to the Section X- Inclusion Criteria for more detail.Approximately 360 subjects will be randomized in a 1:1 ratio to receive a fixed SC dose of anifrolumab (120 mg) or placebo QW with the primary endpoint evaluated at Week 52. Investigational product (IP) will be administered SC via an aPFS. All patients with a baseline prednisone (or equivalent) dose of = 10.0 mg/day must attempt tapering of their baseline dose to = 7.5 mg/day. Tapering will start at Week 8 and continue through Week 40, unless there are signs of increased disease activity. This study includes:• A screening period of up to 30 days • A 52-week double-blind treatment period with a total of 52 anifrolumab or placebo doses• A 10-week safety follow-up after last IP dose
A Multicenter Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of BIIB059 in Adult Participants With Active Systemic Lupus Erythematosus Receiving Background Non-Biologic Lupus Standard of Care
This is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 study designed to evaluate the efficacy and safety of BIIB059 in participants = 18 years of age with active SLE, including joint and/or skin manifestations, who are receiving background nonbiologic lupus SOC therapy. The study will be conducted at approximately 135 sites globally. Approximately 540 participants will be randomized 1:1:1 to receive BIIB059 450 mg, BIIB059 225 mg, or placebo SC Q4W, with an additional dose at Week 2, as add-on to background nonbiologic lupusSOC therapy.The study includes a screening period of up to 4 weeks; a double-blind, placebo-controlled treatment period of 52 weeks; and an SFU period (off-treatment) of 24 weeks. Details of these different study periods are given below.
A multicenter randomized double-blind risankizumab-controlled parallel-group study to evaluate the efficacy and safety of bimekizumab in adult study participants with active psoriatic arthritis
The purpose of this research study is to evaluate the efficacy (how well something works) and safety of bimekizumab in study participants with active psoriatic arthritis (PsA) in comparison to risankizumab.