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About 9,855 results

Surfactant for Neonatal Respiratory Distress Syndrome(NRDS) and Neonatal Acute Respiratory Distress Syndrome(NARDS)
https://clinicaltrials.gov/ct2/show/NCT04777760

Oct 14th, 2022 - To date, PS is not recommended to adult and pediatric ARDS. Meantime, systematic review indicates that PS does not demonstrate statistically significant beneficial effects on reducing the mortality and the rate of BPD in term and late preterm infants with meconium aspiration syndrome(MAS). Therefore, a reasonable speculation is that preterm infants with NARDS do not benefit from one dose of PS....

Selective High Frequency Oscillatory Ventilation (HFOV) for Neonates
https://clinicaltrials.gov/ct2/show/NCT03736707

Oct 14th, 2022 - Similarities to adult and pediatric ARDS, no special pharmacologic therapy for neonatal ARDS has been shown to reduce either short-term or long-term mortality and morbidity, the risk of death therefore remains high for preterm infants. Study reported that 50% chance of death with neonatal intensive care appeared at 24 weeks in most high-income countries and 34 in low-income and middle-income co...

A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
https://clinicaltrials.gov/ct2/show/NCT04940624

Oct 14th, 2022 - The drug being tested in this study is called soticlestat (TAK-935). Soticlestat as an adjunctive therapy will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with DS. The study will enroll approximately 142 pediatric and young adult patients. Participants will be randomized at a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunct...

The Pathogenesis and Natural History of Sjogren s Syndrome
https://clinicaltrials.gov/ct2/show/NCT01425892

Oct 14th, 2022 - BACKGROUND Sj(SqrRoot)(Delta)gren s syndrome (SS) is an autoimmune disease characterized by chronic inflammation involving the exocrine glands. Salivary and lacrimal glands are predominantly affected leading to dry mouth and dry eyes but other exocrine organs are also frequently involved. It is one of the most common rheumatic autoimmune diseases, which effects between 1-4 million Americans, pr...

A Study to Investigate Blinatumomab in Combination With Chemotherapy in Patients With Newly Diagnosed B-Lymphoblastic Leukemia
https://clinicaltrials.gov/ct2/show/NCT03914625

Oct 14th, 2022 - PRIMARY OBJECTIVES: I. To determine in a randomized manner if the addition of 2 cycles of blinatumomab to standard therapy improves disease-free survival (DFS) in patients with standard risk (SR) B-ALL and higher risk features (SR-High), and patients with standard-risk average (SR-Avg) B-ALL who are negative for minimal residual disease (MRD) by flow cytometry but have detectable or indetermina...

A Study of Soticlestat as an Add-on Therapy in Children, Teenagers, and Adults With Lennox-Gastaut Syndrome
https://clinicaltrials.gov/ct2/show/NCT04938427

Oct 14th, 2022 - The drug being tested in this study is called soticlestat (TAK-935). Soticlestat will be assessed for efficacy, safety, and tolerability in pediatric and adult participants with LGS. The study will enroll approximately 234 patients. Participants will be randomly assigned (by chance, like flipping a coin) in a 1:1 ratio to receive standard of care (SOC) plus one of the following adjunctive thera...

Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
https://clinicaltrials.gov/ct2/show/NCT04997811

Oct 14th, 2022 - Over 7,000 people in the UK are living with Myelodysplastic Syndromes (MDS). Approximately 1,600 of these individuals (23%) die each year from their disease. MDS affects the production of blood cells by the bone marrow, causing chronic fatigue, bleeding, and recurrent infections. Many patients die because their disease transforms into acute myeloid leukaemia (AML) an even more aggressive blood ...

Optimal Dose of Surfactant for Preterm Infants With Respiratory Distress Syndrome
https://clinicaltrials.gov/ct2/show/NCT04984057

Oct 14th, 2022 - To date, the optimal dose of PS is inconsistent, although the recommended dose of PS is given in the 2019 update guideline. PS is not recommended to adult and pediatric ARDS. Systematic review indicates that PS does not reduce the incidences of BPD and death in infants with meconium aspiration syndrome(MAS, a subtype of NARDS). A reasonable speculation is that preterm infants with NARDS do not ...

Cancer in Inherited Bone Marrow Failure Syndromes
https://clinicaltrials.gov/ct2/show/NCT00027274

Oct 14th, 2022 - Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders. Pathogenic variant(s) in IBMFS genes are relevant to carcinogenesis in sporadic cancers. Patients with IBMFS who develop cancer differ in their genetic and/or environmental features from patients with IBMFS who do not develop cancer...

Study of Chediak-Higashi Syndrome
https://clinicaltrials.gov/ct2/show/NCT00005917

Oct 14th, 2022 - Chediak-Higashi syndrome (CHS) is a rare autosomal recessive disorder characterized in its classical form by oculocutaneous albinism, a bleeding diathesis, recurrent infection due to abnormal neutrophil and natural killer cell function, and often progression to a lymphohistiocytic infiltration known as the accelerated phase . Death generally occurs within the first decade as a result of infecti...

Study of Proteus Syndrome and Related Congenital Disorders
https://clinicaltrials.gov/ct2/show/NCT00001403

Oct 14th, 2022 - The purpose of this project is to specifically delineate the phenotype and natural history and genetic etiology of Proteus syndrome (PS) and other overgrowth disorders hypothesized to be in the PI3K/AKT pathway. As we have recently determined the molecular cause of PS and the related disorder of fibroadipose overgrowth, our main objectives moving forward include genotype-phenotype correlations,...

Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
https://clinicaltrials.gov/ct2/show/NCT00001727

Oct 14th, 2022 - 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...

Natural History Study of Smith-Magenis Syndrome
https://clinicaltrials.gov/ct2/show/NCT00013559

Oct 14th, 2022 - This project investigates the clinical manifestations and molecular genetic defects of Smith-Magenis Syndrome (SMS), a rare (1/15,000 1/25,000) clinically recognizable yet often under diagnosed multiple congenital anomaly/intellectual disability (MCA/ID) syndrome (OMIM #182290). The syndrome is characterized by a distinct pattern of minor craniofacial and skeletal anomalies, expressive speech/l...

Invasive Ventilation Strategies for Neonates With Acute Respiratory Distress Syndrome Syndrome (ARDS)
https://clinicaltrials.gov/ct2/show/NCT03372525

Oct 14th, 2022 - Severe acute respiratory distress syndrome (ARDS) is one of the serious complications in critically ill neonates. It can result in severe hypoxemia refractory to mechanical ventilation. Usually, invasive ventilation with low parameters is enough for neonates with mild and moderate ARDS. And extracorporeal membrane oxygenation is used to neonates with severe ARDS. However, extracorporeal membran...

Neurobiology of Functional Movement Disorder and Non-Epileptic Seizures
https://clinicaltrials.gov/ct2/show/NCT00500994

Oct 14th, 2022 - Objectives: The study investigates the neurobiological correlates of conversion disorder (CD). The primary objectives are to investigate in CD patients: The role of emotional valence in an implicit emotional processing task (COMPLETE) The frequency of the 5HTTLPR S/S genotype Structural differences in grey matter of the brain as detected by voxel-based morphometry (VBM) The neural correlates of...

Role and Molecular Mechanism of Farnesoid X Receptor(FXR) and RIPK3 in the Formation of Acute Respiratory Distress Syndrome in Neonates
https://clinicaltrials.gov/ct2/show/NCT02598648

Oct 14th, 2022 - Divided into 3 Arms ALI :Diagnostic criteria: 1.Acute onset;2.FiO2/ PaO2< 40.0 kPa (300mmHg, ALI); 3.Chest X-ray showed that the double lung texture increased, the increase of crude, fuzzy, visible diffuse patchy infiltration shadow with compensatory emphysema, for the most early performance; B. double lung field large sheet, asymmetric, edge fuzzy infiltration shadow, the most dense in the lu...

Respiratory COVID-19: A Randomized, Sham-Controlled Study
https://clinicaltrials.gov/ct2/show/NCT05166915

Oct 14th, 2022 - The UV Respiratory Tract Light Therapy Device is intended to emit energy in the UVA region of the spectrum in intubated patients to reduce the viral burden of SARS-CoV-2 (human pathogenic coronavirus (COVID-19)) in intubated patients. The System is to be used in conjunction with the current standard of care measures. The UV Respiratory Tract Light Therapy Device is intended to be used for 20 mi...

Pitolisant in Refractory Restless Legs Syndrome
https://clinicaltrials.gov/ct2/show/NCT05581576

Oct 14th, 2022 - This is an open-label trial in RLS patients who have been inadequately treated with standard therapies, defined by an IRLS > 15. This trial consists of four study center visits (weeks 0, 4, 8, and 16) and four telephone visits (weeks 1, 3, 11, and a post safety phone call at week 17). Subjects will titrate pitolisant for three weeks, starting with a dose of 8.9 mg at week 1, then increase to 17...

Investigation of Prognostic Biomarkers, Host Factors and Viral Factors for COVID-19 in Children
https://clinicaltrials.gov/ct2/show/NCT05576714

Oct 14th, 2022 - Background for COVID-19 associated encephalopathy/encephalitis in children The COVID-19 primarily cause the respiratory diseases, such as croup, bronchiolitis, or pneumonia, but it also can affect the nervous system. According to the data of 1,695 children and adolescents, from March 15, 2020, to December 15, 2020, hospitalized for COVID-19 in 61 hospitals in the United States, 365 (22%) were f...

Functional Remediation for Older Adults With Bipolar Disorder
https://clinicaltrials.gov/ct2/show/NCT05186337

Oct 14th, 2022 - Study design: This is a single-center, single-blind, randomized, controlled test-retest clinical study to evaluate the efficacy of the FROA-BD in a representative sample of patients with BD over 60 years old. This study will be carried out in the Bipolar and Depressive Disorders Unit at the Hospital Clinic of Barcelona, which takes part of the Spanish network Center for Biomedical Research in M...