https://clinicaltrials.gov/ct2/show/NCT04797780
Feb 3rd, 2023 - A subset of participants have MDS characterized by an overexpression of the RARA gene. A blood test will be used to identify participants with RARA-positive MDS. Assessment of the RARA biomarker for study eligibility will be done by collection of blood samples from potential study participants at the pre-screening visit and testing at a central laboratory. Participants who meet eligibility requ...
https://clinicaltrials.gov/ct2/show/NCT02181257
Feb 3rd, 2023 - Lung transplantation has become the treatment of choice for selected patients with end-stage lung disease. Long-term survival after transplantation remains disappointing. Chronic rejection in the form of bronchiolitis obliterans syndrome (BOS) has emerged as the leading obstacle to better long-term outcomes, and is the leading cause of death beyond the first year after transplantation. This dis...
https://clinicaltrials.gov/ct2/show/NCT01553461
Feb 3rd, 2023 - Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to...
https://clinicaltrials.gov/ct2/show/NCT04191304
Feb 3rd, 2023 - This is a multicentre, randomised, double-blind (DB), parallel-group, placebo-controlled, 24-week Phase 3 study to compare the efficacy and safety of benralizumab versus placebo administered by SC injection Q4W in patients with hypereosinophilic syndrome (HES). This study comprises 2 distinct periods (together defined as the 'main study'): A 24-week, DB treatment period, during which patients w...
https://clinicaltrials.gov/ct2/show/NCT00716066
Feb 3rd, 2023 - OUTLINE: Patients receive carmustine intravenously (IV) on day -6, etoposide IV and cytarabine IV twice daily (BID) on days -5 to -2, melphalan IV on day -1, and antithymocyte globulin IV on days -2 and -1. Patients then undergo autologous or syngeneic peripheral blood stem cell transplant on day 0. Patients also receive prednisone orally (PO) once daily (QD) on days 7-21, followed by 2 week ta...
https://clinicaltrials.gov/ct2/show/NCT03604198
Feb 3rd, 2023 - This study is designed to allow continued therapy with relacorilant, a potent, selective glucocorticoid receptor (GR) antagonist in patients who have successfully completed participation of a Corcept-sponsored study of relacorilant (referred to as the "parent" study). Patients may qualify to enter this extension study if they complete their last treatment visit in their parent study and in the ...
https://clinicaltrials.gov/ct2/show/NCT01031407
Feb 3rd, 2023 - Objective: The primary objective of the proposed studies is to utilize neuroimaging (functional Magnetic Resonance Imaging [fMRI], structural MRI [sMRI], Magnetoencephalography [MEG]) and neuropsychological tools (eye-tracking, cognitive experiments, clinical neuropsychological measures, questionnaires, etc.) to identify cognitive idiosyncrasies (e.g., social-cognitive deficits, visual perceptu...
https://clinicaltrials.gov/ct2/show/NCT04508530
Feb 3rd, 2023 - A Superiority Study To Compare The Effect of Panzyga Versus Placebo in Patients with Pediatric Acute-onset Neuropsychiatric Syndrome
https://clinicaltrials.gov/ct2/show/NCT04690504
Feb 3rd, 2023 - Adults who meet ICSD-3 criteria for Advanced Sleep Wake Phase Disorder or Delayed Sleep Wake Phase Disorder will be studied at Brigham and Women's Hospital and Stanford University. The outpatient protocol will consist of two segments: clinic visit and in-home saliva sample collection. For the first segment, the patient participant will be scheduled for a daytime visit to the sleep clinic where ...
https://clinicaltrials.gov/ct2/show/NCT03980340
Feb 3rd, 2023 - The goal of the study is to use a small molecule analysis (e.g. metabolomics) device to measure compounds in breath that may change with sleep-wake state and compounds that vary by time of day. Participants will be fitted with a plastic mask connected to the device for a breath analysis test for about 20 minutes. Then, participants may be asked to lie down and take a nap or to remain sitting/ly...
https://clinicaltrials.gov/ct2/show/NCT04880824
Feb 3rd, 2023 - As part of the innovation fund project PRÄP-GO (EA1/225/19), a multimodal intervention is being conducted in patients with a frailty syndrome. However, the health care research project is limited to evaluating clinical issues only. In order to be able to research further pathophysiological, clinical, psychosocial and work-organizational connections, different groups of participants will be offe...
https://clinicaltrials.gov/ct2/show/NCT02226367
Feb 3rd, 2023 - The proposed study is a 19-week, titration to stable dose, randomized, two-group parallel-design, double-blind, placebo-controlled trial to evaluate the efficacy of prazosin for decreasing alcohol use in 200 active duty Service Members who served in the conflicts in Iraq and/or Afghanistan who are receiving standard outpatient treatment for alcohol use disorders at Joint Base Lewis-McChord. Tre...
https://clinicaltrials.gov/ct2/show/NCT04888936
Feb 3rd, 2023 - Study Description:
https://clinicaltrials.gov/ct2/show/NCT04244890
Feb 3rd, 2023 - The study is a "proof-of-concept" feasibility trial that evaluates usage of a simplified respiratory support system for newborn infants. The study aims to evaluate both the feasibility of providing uninterrupted support for the first hours of life as well as a modified respiratory support system. The system is a modified version of the commercially available rPAP system. The revised design has ...
https://clinicaltrials.gov/ct2/show/NCT00006150
Feb 3rd, 2023 - The Hyper IgE Syndromes (HIES) are primary immunodeficiencies resulting in eczema and recurrent skin and lung infections. Autosomal dominant Hyper IgE syndrome (AD-HIES; Job's syndrome) is caused by STAT3 mutations, and is a multi-system disorder with skeletal, vascular, and connective tissue manifestations. Understanding how STAT3 mutations cause these diverse clinical manifestations is critic...
https://clinicaltrials.gov/ct2/show/NCT00001456
Feb 3rd, 2023 - Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disease consisting of oculocutaneous albinism, a platelet storage pool defect and, in some patients, lysosomal accumulation of ceroid lipofuscin. Other manifestations include pulmonary fibrosis (often fatal in the fourth or fifth decade), chronic granulomatous colitis and, rarely, renal involvement or cardiomyopathy. There exist 10 d...
https://clinicaltrials.gov/ct2/show/NCT00001727
Feb 3rd, 2023 - Polyostotic fibrous dysplasia (PFD) is a sporadic disorder which affects multiple sites in the skeleton. The bone at these sites is rapidly resorbed and replaced by abnormal fibrous tissue or mechanically abnormal bone. PFD may occur alone or as part of the McCune-Albright Syndrome (MAS), a syndrome originally defined by the triad of PFD, cafe-au-lait pigmentation of the skin, and precocious pu...
https://clinicaltrials.gov/ct2/show/NCT03396562
Feb 3rd, 2023 - Background: Sex Chromosome Trisomies (SCT) including Klinefelter (XXY), Trisomy X (XXX), and XYY syndromes occur in 1 out of every 500 births and are associated with a broad phenotypic spectrum including increased risk for developmental delays (DD), language/learning disorders, and autism spectrum disorder (ASD). XXY is also associated with testicular failure, XXX increases risk for ovarian fai...
https://clinicaltrials.gov/ct2/show/NCT01351545
Feb 3rd, 2023 - Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to...
https://clinicaltrials.gov/ct2/show/NCT05711966
Feb 3rd, 2023 - BACKGROUND: RDS represents the main cause of respiratory failure in preterm infants and one of the major causes of perinatal morbidity and mortality. Surfactant is a well-established therapy in neonatology, but its optimal administration method remains unresolved, especially with the focus on avoiding MV in preterm infants born before 28 weeks' gestational age (i.e. extremely low gestational ag...
