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About 22,155 results

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 ...

An Efficacy and Safety Study of Somatuline Depot (Lanreotide) Injection to Treat Carcinoid Syndrome
https://clinicaltrials.gov/ct2/show/NCT00774930

Oct 14th, 2022 - This study consisted of a Screening period, conducted up to 4 months before randomisation, followed by three phases: a 16-week, double blind (DB), randomised, placebo-controlled phase; a 32-week initial open label (IOL) phase; and a long term open label extension (LTOLE) phase. The DB phase evaluated lanreotide Autogel versus placebo in subjects with a history of carcinoid syndrome (flushing an...

Trial of the Combination of Bortezomib and Clofarabine in Adults With Relapsed Solid Tumors
https://clinicaltrials.gov/ct2/show/NCT02211755

Oct 14th, 2022 - BACKGROUND: The proteasome inhibitor bortezomib and purine nucleoside metabolic inhibitor clofarabine demonstrated greater than additive activity in combination in preclinical xenograft models, justifying the clinical evaluation of this combination for its antitumor activity OBJECTIVES: To establish the safety, tolerability, and maximum tolerated dose (MTD) of bortezomib and clofarabine in pati...

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...

Invasive Ventilation for Neonates With Acute Respiratory Distress Syndrome(ARDS)
https://clinicaltrials.gov/ct2/show/NCT03591796

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...

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 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...

Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
https://clinicaltrials.gov/ct2/show/NCT04868968

Oct 14th, 2022 - This is an open-label, single-arm, multiple dose, phase II study to assess safety, tolerability and clinical efficacy of DFV890 in participants with FCAS. The study includes a screening period, a treatment period and a follow-up period. At most, the study will last up to 7 months (and up to 13 months for participants with a historical screening cold challenge prior to protocol amendment 04).

Linaclotide Safety and Efficacy in Pediatric Participants, 6 to 17 Years of Age, With Irritable Bowel Syndrome With Constipation (IBS-C) or Functional Constipation (FC)
https://clinicaltrials.gov/ct2/show/NCT04026113

Oct 14th, 2022 - The objective of LIN-MD-64 is to evaluate the safety and efficacy of 12 weeks of linaclotide therapy (72 μg daily) in comparison with placebo in pediatric participants, 6 to 17 years of age, who fulfill modified Rome III Criteria for Child/Adolescent Functional Constipation (FC). The objective of LIN-MD-64 is to evaluate the safety and efficacy of 12 weeks of linaclotide therapy (145 μg or 290 ...

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 ...

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...

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...

Tyrosine Kinase Inhibition to Treat Myeloid Hypereosinophilic Syndrome
https://clinicaltrials.gov/ct2/show/NCT00044304

Oct 14th, 2022 - This study will evaluate the safety and effectiveness of imatinib mesylate in reducing the number of eosinophils (a type of white blood cell) in patients with hypereosinophilic syndrome (HES). Patients with HES have elevated counts of eosinophils in the blood and body tissues, which can cause damage to these tissues. Although HES can involve any tissues, the heart, nerves, and skin are most oft...

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,...

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...

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...

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...

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...

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...

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
https://clinicaltrials.gov/ct2/show/NCT05127226

Oct 14th, 2022 - This is a Phase 1-2a, open-label dose-escalation study of ION582 enrolling up to approximately 44 participants. Following a screening period of up to 4 weeks, eligible participants will receive intrathecal (IT) injections of ION582. Participants will be followed for up to 32 weeks after dosing.