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NCT00999518
In a previous study in patients with interstitial cystitis/ painful bladder syndrome (IC/PBS), tanezumab has shown to be efficacious in relieving the pain associated with IC/PBS, as well as some effect on reducing urinary urgency. Only one dose was studied, and tanezumab was well tolerated. In this study, the hypothesis being tested is that tanezumab will show efficacy at several doses on reducing pain with sufficient tolerability. Tanezumab's safety will also be assessed at different dose levels
NCT03131453
The purpose of this study is to determine the effects of CNP520 on cognition, global clinical status, and underlying AD pathology, as well as the safety of CNP520, in people at risk for the onset of clinical symptoms of AD based on their age, APOE genotype and elevated amyloid.
NCT03426345
This study will evaluate the safety and efficacy of relamorelin compared to placebo in participants with diabetic gastroparesis. Participants will report daily severity scores of their diabetic gastroparesis symptoms.
NCT02981368
This study evaluates the safety and diagnostic performance of 18F-DCFPyL Injection in patients with at least high risk prostate cancer who are planned for radical prostatectomy with lymphadenectomy (Cohort A) or in patients with locally recurrent or metastatic disease willing to undergo biopsy (Cohort B). Cohort B is complete and no longer recruiting subjects.
NCT03898791
The purpose of this study is to determine the recommended phase 2 dose of LY3295668 erbumine in participants with platinum-sensitive, extensive-stage small-cell lung cancer.
NCT02633046
Focal segmental glomerulosclerosis (FSGS) is a condition that harms the kidney "filters" that remove waste from the blood. Proteins are supposed to stay in the blood. Damaged "filters" let protein get into the kidney. FSGS is a serious condition that can lead to kidney failure. The only treatment for kidney failure is dialysis or kidney transplant. Proteinuria means too much protein came through the kidneys into the urine. If the doctor cannot figure out what is causing the problem, it is primary (idiopathic) FSGS. This kind of FSGS is very hard to treat. This study will test Acthar in patients with this condition who have not responded to other treatments. It primarily investigates how well the therapy is tolerated by the patients and how well they respond to this treatment.
NCT02440789
The purpose of this study was to find out about the safety of sirolimus in individuals with HIV infection who were also being treated with ART. The investigators wanted to learn whether sirolimus decreases inflammation and immune activation in the body; whether sirolimus changes the level of HIV in the participants' blood; and how sirolimus interacts with ART in the blood. Sirolimus is approved by the Food and Drug Administration (FDA) to prevent organ rejection in patients aged 13 years and older receiving kidney transplants. Sirolimus had also been used for the prevention of complications after stem cell transplants and as a treatment for certain kinds of cancers in HIV-infected patients.
NCT03093870
This protocol for Varlitinib is developed for the treatment of Biliary Tract Cancer. Varlitinib (also known as ASLAN001) is a small-molecule, adenosine triphosphate competitive inhibitor of the tyrosine kinases - epidermal growth factor receptor (EGFR), human epidermal growth factor receptor (HER)2, and HER4. Varlitinib may be beneficial to subjects with cancer by simultaneous inhibition of these receptors. The purpose of this study is to determine the safety and efficacy of Varlitinib in combination with capecitabine for the treatment of Biliary Tract Cancer. Treatment groups are Varlitinib+capecitabine and Placebo + capecitabine
NCT03358290
Study to evaluate the efficacy, safety, tolerability and pharmacokinetics of JTE-051 administered for 12 weeks in subjects with moderate to severe plaque psoriasis.
NCT03342872
National mandates for increasing accountability in behavioral healthcare have created an urgent need for effective quality assurance (QA) procedures featuring pragmatic measures of treatment implementation quality in usual care (UC). The most practical method for assessing treatment quality in UC is therapist self-report; unfortunately, therapist-report measures of fidelity to evidence-based practices (EBPs) have demonstrated weak validity to date. This study addresses this need for effective QA procedures by first developing treatment quality assurance procedures designed to increase the adoption and quality of empirically supported family-based services (FBS) for adolescent substance use (ASU) in usual care, and then testing two system-level implementation strategies for installing the new quality procedures in ASU treatment sites. FBS have achieved the strongest evidence base for treating ASU and are a prime candidate for upgrading the quality of ASU services in various systems of care. FBS comprise both family participation in services, the systemic parameters wherein family members are included in assessment and treatment activities; and family therapy techniques, the specific interventions that clinicians use to directly target family members and family functioning for change. For FBS to fulfill their potential to enhance ASU treatment systems, FBS implementation must be supported by effective quality assurance procedures designed to ensure that FBS are delivered with fidelity. The proposed study will leverage a strong research-government partnership between the applicant organization and the New York single-state agency for SU services. The study will first develop innovative quality procedures (Aim 1) that use existing FBS quality metrics to promote high-fidelity FBS: Measurement Training and Feedback System for Implementation (MTFS-I), a pragmatic evidence-based method for increasing FBS quality by providing monthly feedback on therapist-reported FBS delivery along with brief online FBS training modules. The study will then experimentally compare two system-level implementation strategies designed to foster MTFS-I utilization in usual care for ASU. Core Training Only will contain two 3-hour training sessions: Mapping existing FBS and identifying site goals for FBS improvement; and Installing and sustaining the MTFS-I. Core + Facilitation is an additive strategy that will begin with the Core Training sessions and then continue with monthly facilitation meetings for one year to promote MTFS-I use and progress toward FBS improvement. The study will feature a three-group cluster randomized trial testing Core Training Only versus Core + Facilitation versus no-intervention Control in 9 ASU clinics across New York State. MTFS-I utilization data will be collected from Core Training and Core + Facilitation sites for one-year follow-up (after initial Core Training); FBS quality data on family participation and family therapy technique use will be collected from all sites over one-year follow-up; observational coding methods will verify therapist reports of FBS quality; and client outcome data for all sites will be retrieved from administrative data warehouses over one-year baseline and one-year follow-up. These data will enable between-condition comparisons of FBS delivery (Aim 2: MTFS-I utilization, family participation, family therapy technique use) and client outcomes (Aim 3: therapeutic goal achievement, substance use change). If study aims are achieved, investigators would be positioned to mount a fully powered RCT to test the effectiveness of these FBS quality improvement procedures at scale.
NCT00599638
Pregabalin is an alpha-2 delta ligand approved for the treatment of neuropathic pain, however, not all patients will respond to this drug. This study will compare the efficacy of pregabalin when administered with an experimental drug PF-00489791, in patients with post-herpetic neuralgia. The efficacy of this combination will be compared to pregabalin alone.
NCT01928927
The main goal of this study was to see if a drug called telmisartan would decrease fibrosis (scarring) and inflammation (irritation) in people who are infected with HIV and doing well on their HIV medications. The study was also done to see what effects telmisartan has on other signs of disease and inflammation in the body, and to see whether people who have HIV can take telmisartan safely and without side effects that make them want to stop the drug. Telmisartan is FDA-approved for treating high blood pressure and decreasing the chance of heart attacks and strokes in people over the age of 55 years of age who are at high risk for these events.
NCT02041299
This research is being done so that we can look at the safety and efficacy of deferiprone in people with sickle cell disease or other anemias. Deferiprone is a drug that removes iron from the body. We will be comparing deferiprone with deferoxamine, another drug that removes iron from the body.
NCT02738281
The purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT including the range of clinical involvement and to correlate genotype-phenotype over a broad spectrum of phenotypes. While much has been learned about RTT, improvements are required in understanding the role of factors such as X chromosome inactivation, genetic background, and others including the environment, on the great variability observed even between individuals with the same MECP2 mutation. These data will be essential to the development and conduct of clinical trials that are anticipated from ongoing studies in animal models for RTT. This study will not include clinical trials, but should set the stage for such trials and other translational research projects (e.g., development of biomarkers).
NCT02705677
The overarching purpose of this study is to advance understanding of the natural history of Rett syndrome (RTT), MECP2-duplication disorder (MECP2 Dup), RTT-related disorders including CDKL5, FOXG1, and individuals with MECP2 mutations who do not have RTT. Although all these disorders are the result of specific genetic changes, there remains broad clinical variation that is not entirely accounted for by known biological factors. Additionally, clinical investigators currently do not have any biomarkers of disease status, clinical severity, or responsiveness to therapeutic intervention. To address these issues, biological materials (DNA, RNA, plasma, cell lines) will be collected from affected individuals and in some cases from unaffected family members, initial evaluation performed to identify additional biological factors contributing to disease severity, and these materials will be stored for future characterization.
NCT03077308
The overall purpose of this project is to advance understanding of the neurophysiological features of Rett syndrome (RTT), MECP2 Duplication (MECP2 Dup) and RTT-related disorders (CDKL5, FOXG1) to gain insight into disease pathogenesis, with an emphasis on identifying biomarkers of disease evolution and severity. This specific study is intertwined to the core study Natural History of Rett Syndrome and Related Disorders (RTT5211), which characterizes range of clinical involvement and genotype-phenotype correlations and will provide phenotypical data for determining the clinical relevance of the neurophysiologic parameters; study subjects here are co- and primarily enrolled in RTT5211. The proposed studies will serve as basis of future translational investigations, including further refinement of biomarkers, development of outcome measures, and clinical trials per se.
NCT00703820
The purpose of this study is to assess the feasibility and efficacy of a novel form of therapy-haploidentical NK cell transplantation-in patients with standard-risk AML. In addition, we will investigate the efficacy of clofarabine + cytarabine (Clo/AraC) in newly diagnosed patients with AML and attempt to optimize outcome through the use of MRD-adapted therapy and further improvements in supportive care.
NCT03052751
The purpose of the study is to evaluate the clinical efficacy of UCB7665 as a chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe.
NCT02973087
The purpose of this phase 3 study is to investigate the efficacy and safety, including immunogenicity, thrombogenicity and hypersensitivity reactions, as well as pharmacokinetics (PK), health related quality of life (HRQoL) and pharmacoeconomics of prophylactic treatment with recombinant von Willebrand factor (rVWF) (vonicog alfa) in adult participants with severe von Willebrand disease (VWD).
NCT02365480
This randomized, pilot phase I trial studies the side effects of berberine chloride in treating patients with ulcerative colitis and who are in remission (a decrease in or disappearance of signs and symptoms of cancer) to reduce the risk of colorectal cancer. Patients with ulcerative colitis are at increased risk for colorectal cancer. Chemoprevention is the use of drugs, such as berberine chloride, to keep a disease/condition from forming or coming back. The use of berberine chloride may keep colorectal cancer from forming in patients with ulcerative colitis.