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About 35,553 results

Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome
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...

Extracorporeal Photopheresis for Medicare Recipients of Lung Allografts
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...

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
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...

A Phase 3 Study to Evaluate the Efficacy and Safety of Benralizumab in Patients With Hypereosinophilic Syndrome (HES)
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...

Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases
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...

Extension Study to Evaluate the Safety of Long-Term Use of Relacorilant in Patients With Cushing Syndrome
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 ...

Cognitive Neuroscience of Autism Spectrum Disorders
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...

Phase III Study To Compare The Effect of Panzyga Versus Placebo in Patients With Pediatric Acute-onset Neuropsychiatric Syndrome
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

Validation of Circadian Biomarkers in Patients With Sleep Disorders
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 ...

Breath Biomarkers for Sleep Loss and Circadian Timing
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...

Analysis of Frailty Syndrome Within the Framework of the Innovation Fund Project PRÄP-GO (ANA-PRÄP-GO)
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...

Prazosin Augmentation of Outpatient Treatment of Alcohol Use Disorders in Active Duty Soldiers With and Without PTSD
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...

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies
https://clinicaltrials.gov/ct2/show/NCT04888936

Feb 3rd, 2023 - Study Description: The RASopathies are a clinically defined group of disorders caused by pathogenic germline variants in genes encoding components of the Ras/mitogen-activated-protein kinase (Ras/MAPK) pathway. These disorders have overlapping clinical features due to Ras/MAPK dysfunction, including a predisposition to the development of certain malignancies. The aims of this prospective l...

Feasibility of Uninterrupted Infant Respiratory Support Treatment
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 ...

Natural History, Management, and Genetics of the Hyperimmunoglobulin E Recurrent Infection Syndrome (HIES)
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...

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
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...

Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
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...

The eXtroardinarY Babies Study: Natural History of Health and Neurodevelopment in Infants and Young Children With Sex Chromosome Trisomy
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...

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
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...

COMPARISON OF INRECSURE AND LISA IN PRETERM NEONATES WITH RDS: A RANDOMIZED CONTROLLED TRIAL
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...