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NCT01767909
An urgent need exists to find effective treatments for Alzheimer's disease (AD) that can arrest or reverse the disease at its earliest stages. The emotional and financial burden of AD to patients, family members, and society is enormous, and is predicted to grow exponentially as the median population age increases. Current FDA-approved therapies are modestly effective at best. This study will examine a novel therapeutic approach using intranasal insulin (INI) that has shown promise in short-term clinical trials. If successful, information gained from the study has the potential to move INI forward rapidly as a therapy for AD. The study will also provide evidence for the mechanisms through which INI may produce benefits by examining key cerebral spinal fluid (CSF) biomarkers and hippocampal/entorhinal atrophy. These results will have considerable clinical and scientific significance, and provide therapeutically-relevant knowledge about insulin's effects on AD pathophysiology. Growing evidence has shown that insulin carries out multiple functions in the brain, and that insulin dysregulation may contribute to AD pathogenesis. This study will examine the effects of intranasally-administered insulin on cognition, entorhinal cortex and hippocampal atrophy, and cerebrospinal fluid (CSF) biomarkers in amnestic mild cognitive impairment (aMCI) or mild AD. It is hypothesized that after 12 months of treatment with INI compared to placebo, subjects will improve performance on a global measure of cognition, on a memory composite and on daily function. In addition to the examination of CSF biomarkers and hippocampal and entorhinal atrophy, the study aims to examine whether baseline AD biomarker profile, gender, or Apolipoprotein epsilon 4 (APOE-ε4) allele carriage predict treatment response. In this study, 240 people with aMCI or AD will be given either INI or placebo for 12 months, following an open-label period of 6 months where all participants will be given active drug. The study uses insulin as a therapeutic agent and intranasal administration focusing on nose to brain transport as a mode of delivery.
NCT02380274
The purpose of this study is to describe patterns of care in CRPC patients, as well as health-related quality of life (HRQoL) outcomes associated with CRPC and its management. This study will also describe factors influencing treatment decisions including reason(s) for treatment choices and triggers for treatment changes for CRPC as well as describe clinical outcomes based on patient characteristics.
NCT02561455
The purpose of the study was to provide access to continued treatment for those who participated in other Astellas sponsored ASP2215 trials that completed the primary analysis and, had the potential to continue to derive clinical benefit from the treatment with ASP2215, and who did not meet any of the study discontinuation criteria in the present study.
NCT05276349
The purpose of this study is to study use of advance Digital Health Technologies (DHT) and its validity as measures for assessing progression in Amyotrophic Lateral Sclerosis (ALS) patients. A total of 80 ALS patients will be recruited across US, and will involve two sites - St. Joseph Hospital and Medical Center in Phoenix, AZ, and Emory University ALS Clinic in Atlanta. This will be a fully remote observational study and will employ remote data collection platforms such as (a) A digital spirometry device powered by a mobile app will be used to measure vital capacity; (b) A clinical-grade voice recording app will be used to evaluate speech function; (c) A medical-grade wearable sensor will be used to monitor activity levels and sleep patterns; and (d) Standardized Electronic Clinical Outcome Assessments (eCOA) and Patient Reported Outcomes (ePRO) will be used to evaluate quality of life and cognitive abilities. The main goals of this study is to answer some of these questions: 1. Can ALS patients measure important aspects of disease progression at home, either by themselves with appropriate training or with assistance of a coordinators via virtual visit? 2. Which clinical outcome measures collected through DHT are sensitive indicators of ALS progression? 3. Are the measures reproducible and whether they can correlate with gold standard assessments? The results of this study have the potential to provide valuable information for designing future ALS trials that are more decentralized, more patient-centric, and require less visits to the clinic which typically become a major burden with disease progression
NCT03682536
The purpose of this study is to determine the effectiveness of luspatercept (ACE-536) compared to epoetin alfa on red blood cell (RBC) transfusion independence (for at least 12 weeks) with a concurrent hemoglobin increase of at least 1.5 g/dL in participants with anemia due to revised international prognostic scoring system (IPSS-R) very low, low, or intermediate risk myelodysplastic syndromes (MDS) who require RBC transfusions and have never been exposed to erythropoiesis stimulating agent (ESA).
NCT05112237
The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.
NCT02614066
The primary objectives of this study are to determine the safety and efficacy of brexucabtagene autoleucel (KTE-X19) in adult participants with relapsed/refractory (r/r) B-precursor acute lymphoblastic leukemia (ALL).
NCT06690177
The main objective of this study is: To assess the safety of EscharEx (EX-03 5% formulation) compared to placebo control and compared to Collagenase (NSSOC) in patients with VLU.
NCT03921541
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.
NCT05715346
Phase 1/2a, multicenter, randomized, vehicle-controlled, double-masked, multiple-dose, parallel-group study conducted in adult subjects with acquired blepharoptosis.
NCT05509855
This study will provide long-term follow-up for patients who have received treatment with WU-CART-007 in a previous clinical trial. In this study, patients will be followed for up to 15 years after their last dose of WU-CART-007 for evaluation of delayed adverse events, presence of persisting WU-CART-007 vector sequences, and overall survival and progression-free survival.
NCT01064622
This randomized phase I/II trial studies gemcitabine hydrochloride and vismodegib to see how well they work compared with gemcitabine hydrochloride alone in treating patients with recurrent or metastatic pancreatic cancer. Drugs used in chemotherapy, such as gemcitabine hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Vismodegib may slow the growth of tumor cells. It is not yet known whether giving gemcitabine hydrochloride together with vismodegib is more effective than gemcitabine hydrochloride alone in treating patients with pancreatic cancer.
NCT03761914
To evaluate the safety, tolerability, and anti-tumor activity of galinpepimut-S in combination with pembrolizumab in patients with selected advanced cancers.
NCT01101906
This is a randomized, placebo-controlled, double-blind phase 2 study of OSI-906 or placebo at a continuous 150 mg twice daily (BID) dose.
NCT05262400
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called PF-07220060 and PF-07104091) in people with breast cancer. This clinical study consists of 2 parts (part 1 and part 2). In part 1, we are seeking participants who: * Have been diagnosed with Breast Cancer (BC) of either types: * Have HR+, HER2- BC * Refractory HR-positive/HER2-positive BC * Have other solid tumors other than BC In part 2, we are seeking participants who: -Have HR-positive/HER2-negative BC Part 1 will include increasing doses of PF-07220060 with PF-07104091. In part 2, participants will take 1 of 2 study medicine combinations. This will help us decide the highest amount of study medicines that can be safety given to people. All participants in this study will receive PF-07220060 with PF-07104091 by mouth. We will compare participant experiences to help us determine if PF-07220060 with PF-07104091 is safe and effective. Participants will take part in this study for about 2 years. During this time, they will receive the study medicine, an x-ray imaging, and will be observed for safety and effects of the study medicines.
NCT03769116
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2. In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).
NCT05512962
The purpose of this clinical trial is to evaluate the safety and tolerability of suprachoroidal microcatheterization with the Oxulumis® device for a randomized treatment with two dose levels of Triesence® in subjects with Diabetic Macular Edema.
NCT05199272
This is a first-in-human open-label Phase 1/2a study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary clinical activity of 23ME-00610 given by intravenous infusion in patients with advanced solid malignancies who have progressed on all available standard therapies
NCT02635672
Determine the safety, tolerability, pharmacokinetics, maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of VIP152 (BAY 1251152) as monotherapy or in combination in patients with solid tumors and aggressive non-hodgkin's lymphoma (NHL).
NCT06034275
Dose Escalation - Determine the maximum tolerated dose (MTD), if possible, or minimum optimal biologic dose (OBD), and evaluate the safety and tolerability of VIP943 in subjects with advanced CD123+ hematologic malignancies