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NCT04068103
This phase II/III trial studies how well circulating tumor deoxyribonucleic acid (ctDNA) testing in the blood works in predicting treatment for patients with stage IIA colon cancer after surgery. ctDNA are circulating tumor cells that are shed by tumors into the blood. Finding ctDNA in the blood means that there is very likely some small amounts of cancer that remain after surgery. However, this cancer, if detected, cannot be found on other tests usually used to find cancer, as it is too small. Testing for ctDNA levels may help identify patients with colon cancer after surgery who do benefit, and those who do not benefit, from receiving chemotherapy.
NCT05761301
To evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-KHK and to evaluate the safety, tolerability, efficacy, PK and PD of multiple doses of KHK.
NCT02624947
The purpose of this study is to determine the efficacy of maternal immunization during the third trimester of pregnancy with the RSV F vaccine against medically-significant RSV lower respiratory tract infection (LRTI), as defined by hypoxemia or tachypnea at rest, through the first 90, 120, 150, and 180 days of life in infants.
NCT03569891
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
NCT04203641
This study will evaluate the safety and tolerability of escalating doses of L-DOS47 in combination with doxorubicin, as well as preliminary anti-tumor activity in patients with previously treated advanced pancreatic cancer.
NCT04974528
INHALE-1 is a Phase 3, open-label, randomized clinical study evaluating the efficacy and safety of Afrezza in combination with a basal insulin (i.e., the Afrezza group) versus insulin aspart, insulin lispro or insulin glulisine in combination with a basal insulin (i.e., the Rapid-acting Insulin Analog \[RAA\] injection group) in pediatric subjects with type 1 or type 2 diabetes mellitus. Following 26 weeks of randomized treatment (i.e., Afrezza or RAA injection combined with a basal insulin), all subjects will enter a treatment extension where subjects will receive Afrezza until Week 52. The purpose of the treatment extension is to assess safety and efficacy with continued use of Afrezza. Pediatric subjects ≥4 and \<18 years of age will be enrolled in this study. Subjects will be randomly assigned in a 1:1 ratio to either the Afrezza group or the RAA injection group. The study is composed of: * Up to 5-week screening/run-in period * 26 week randomized treatment period * 26-week treatment extension * 4-week follow-up period
NCT05141006
Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic and debilitating urological complex of disorders characterized by symptoms of bladder pain or discomfort, mostly upon bladder filling, and often accompanied by lower urinary tract symptoms (LUTS). This study will assess how safe and effective BOTOX (onabotulinumtoxinA) is in treating IC/BPS. Adverse events and change in disease symptoms will be evaluated. BOTOX (onabotulinumtoxinA) is an investigational drug being developed for the treatment of Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS). Study doctors randomly assign the participants to 1 of 2 groups, called treatment arms, to receive BOTOX or placebo. There is a 1 in 2 chance that participants will be assigned to placebo. Approximately 80 female participants, aged 18 to 75 years, with a diagnosis of IC/BPS will be enrolled in approximately 40 sites in the United States and Canada. Participants will receive BOTOX or placebo injected into the bladder on Day 1 and will be followed for at least 12 weeks in treatment 1. Eligible participants may request additional dose of BOTOX between Weeks 12 and 24, and will be followed for 12 weeks in treatment period 2. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.
NCT05892614
This is a 2-Part study with Part A, a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20. Part B is an optional open-label extension to Part A in which participants can receive 450 mg efzofitimod every 4 weeks for 6 doses.
NCT02040714
Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0-14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective study comparing the results of current treatments. The present study will establish a database of prospectively identified patients with Legg-Calvé-Perthes (LCP) Disease and collect information regarding their presentation, treatment, and outcomes in the course of receiving currently available treatments. This study seeks to compare the outcomes of current treatments in the management of different age groups (ages 1-6, 6-8, 8-11, \>11) of patients with Perthes disease at two- and five-year followup and at skeletal maturity. For each age group, two to three common treatment regimens currently used by practicing pediatric orthopaedic surgeons will be compared. The intervention a patient receives is determined through physician treatment expertise, and is not pre-determined by the study.
NCT02764801
The primary objective of this trial is to evaluate the sensitivity and specificity of 2D and 4D contrast enhanced ultrasound for monitoring transarterial chemoembolization (TACE) response 1-2 weeks and 1 month post treatment as an alternative to contrast-enhanced magnetic resonance (MRI) or computed tomography (CT) imaging
NCT04344028
The present study addresses whether placebo effects can possibly account for previous findings in the field, as well as ascertains whether placebo-based mechanisms can be deliberately harnessed for impact. The design is a 2 (positive expectations/negative expectations) x 2 (true cognitive training intervention x control cognitive training intervention) Randomized Controlled Trial (RCT). Participants will be recruited from site-affiliated participant pools or email lists. Participants will first undergo a pre-test battery where various cognitive abilities are assessed via computerized programs. They will also fill out various surveys about personality traits/internal dispositions that may predict the extent to which they are susceptible to placebo effects. After pre-testing, participants will be randomized to an expectations group (positive/negative) and a training protocol (active/control). Participants are then given their group appropriate expectation script. In the positive expectations group, participants receive training described as having previous research supporting its use as a method to enhance cognitive abilities. In the negative expectations group, participants receive training described as having previous research suggesting that it is unlikely to change cognitive abilities or may even decrease cognitive abilities. The participants will then be given instructions regarding how to do their training task. Participants will be asked to complete 10 sessions of training within 15 days. The active training will be a standard working memory (N-back) training task. The control training will be a trivia/quiz training task. After they have completed 10 sessions, participants will complete a "mid-test" session to undergo a battery of perceptual/cognitive tasks. Participants will be told that the perceptual/cognitive tests are identical to those that they took at pre-test. However, in reality, for participants in the "positive expectations" groups, these will be altered to actually be easier than they were at pre-test. For participants in the "negative expectations" groups the tasks will be made more difficult. Participants will then be asked to complete another set of 10 training sessions on their devices within 15 days before completing the post-test. The post-test will be identical to the pre-test. All participants will then be extensively debriefed (e.g., all deceptions will be made clear). On a separate day, participants will then complete a second post-test that is identical to the pre-test and first-post test.
NCT02616484
The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. The investigators predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. The conclusions of numerous laboratory and clinical investigations are consistent with this postulate and have led to the designation of DCA as an Orphan Product for congenital lactic acidosis by the Food and Drug Administration. A novel Observer reported outcome (ObsRO) survey that is completed by study participant's parent/caregiver, is the efficacy outcome measure. Funding Source - FDA OOPD
NCT04467515
This is a Phase 1/2 multi-center, open label, dose escalation and dose expansion study to evaluate safety, tolerability, dosimetry, pharmacodynamics (PD), and efficacy of the targeted radionuclide therapeutic CAM-H2 in patients with progressive, advanced/metastatic HER2-positive breast, gastric, and GEJ cancer with disease progression following anti-HER2 standard of care treatment.
NCT05067972
A study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and antitumor activity of PF-07260437, a B7-H4 x CD3 bispecific mAb, in participants aged ≥18 years of age with advanced or metastatic breast cancer, ovarian cancer or endometrial cancer. Adult participants with other advanced or metastatic high B7-H4 expressing tumors may be considered after discussion with and approval from sponsor.
NCT04591392
Evaluation of the safety and efficacy of the reSept ASD Occluder to treat patients with clinically significant secundum atrial septal defect
NCT05564598
Patients with moderate or severe CMV disease less than 21 days old who have a maternal donor who has a CMV response to the peptivators will be screened. All patients will receive treatment with valganciclovir or ganciclovir. There is a safety run in with treatment with CMV CTLs in cohort 1 and if found to be safe, will proceed to cohort 2 for randomization to receive antiviral therapy with or without CMV CTLs. Funding source: FDA OOPD
NCT04500847
This is a randomized, double-blind clinical trial of a daily oral dose of 200 mg emtricitabine vs. placebo in 35 participants with biomarker-confirmed MCI or mild to moderate dementia due to Alzheimer's disease. Study duration for each subject participating in the placebo-controlled research study will be approximately 12 months (up to a 3 months Screening Period, Baseline visit (1 month), 6 months of placebo or emtricitabine dosing, and 1 month follow-up). Participants will have up to 2 months to complete all procedures for the month 6 study visit.
NCT05093504
Prospective, Multicenter, Double-blind, Randomized, Study to Evaluate DrugSorb-ATR Removal of Apixaban and Rivaroxaban to Reduce Likelihood of Serious Bleeding in Patients Undergoing Urgent Cardiothoracic Surgery
NCT06959316
T1D Pregnancy \& Me will partner with pregnant participants living with type 1 diabetes (T1D) in the United States to collect real-world data on management of T1D in pregnancy. This is a remote study where participants can complete online surveys and share device data (continuous glucose monitor (CGM) data and insulin data). Through the collection of CGM, insulin, and pregnancy outcome data, the study will provide important information to understand how diabetes is being managed during pregnancy. These data will provide much needed evidence to guide modern management of diabetes during pregnancy with a goal of improving care and outcomes.
NCT05424081
This study will test whether EMBER, a self-help tool to increase weight loss treatment engagement, helps veterans engage in Veterans Health Administration (VHA) weight management programs. Participants will be randomly assigned to receive EMBER or a list of weight management programs (the control group). They will answer questions about health beliefs and behaviors and share information from their medical record at the start of the study, 2-months after the start of the study, and 6-months after the start of the study. Participants will receive compensation for each of these contacts. Participants will also receive a reminder call 10 days after they enroll in the study. All study contacts will be over the phone. The main hypothesis is that people who receive EMBER will be more likely to use VHA weight management programs than people in the control group.