55 Clinical Trials for Various Conditions
This study is being done to learn more about a possible new treatment for pain episodes (called vaso-occlusive crises or VOCs) in children, teens, and young adults with sickle cell disease (SCD). The study will include about 120 participants between the ages of 6 and 21 who come to the emergency department (ED) with a VOC. A VOC is a painful episode that happens with no clear cause and no signs of infection or major problems with organs like the liver or kidneys. Before joining the study, patients and their families may be asked to learn about it and give permission (called consent or assent) while at a regular clinic visit. If that hasn't happened yet, the consent/assent process will happen at the emergency department when the patient comes in for care. If the patient meets all the study requirements, they can join the treatment part of the study. Participants will be randomly assigned (like flipping a coin) to receive either: L-citrulline, the study drug, or A placebo, which looks the same but has no active ingredients. Everyone has an equal chance of getting either one. The study drug is given through an IV. It starts with one larger dose, followed by a steady infusion for up to 12 hours. All patients in the study will still receive the usual pain treatment (called standard of care), which may include opioids. However, some patients may need fewer opioids if the study treatment helps with their pain. If any medicines are not allowed during the study, the doctor will explain this during the consent process. Patients can go home once: Their pain is controlled with oral (by mouth) pain medicine, They're eating and drinking well, and They've been given a personal pain management plan to use at home. After leaving the hospital, the study team will follow up with patients by phone about 2 days later (within a 12-hour window), again around Day 7, and again around Day 30 to check how they're doing.
This study consists of two parts: phase 2 (Part A) and phase 3 (Part B). It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD, and to evaluate the safety and tolerability of CSL889 in study participants.
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises.
Vaso-occlusive crisis (VOC) is the most common complaint in patients with sickle cell disease presenting to the emergency room. VOC is most commonly treated with opioids and NSAIDs. However, new research is demonstrating that opioids in addition to virtual reality (VR) is more effective at reducing the experience of pain and pain nerve signals compared to opioids alone. Numerous research studies have demonstrated that VR reduces the experience of pain during painful medical procedures in children, such as venipuncture and burn wound dressing changes. The study aims to add VR to standard of care medical treatment for pediatric patients with sickle cell disease who present to the pediatric emergency department in VOC. Investigators will conduct a retrospective chart review of patients aged 6 to 21 years with sickle cell disease who present to the pediatric emergency department with VOC for the historical control arm. Investigators will also conduct a prospective convenient sampling of patient who receive VR plus standard medical care in patients aged 6 to 21years with sickle cell disease who present to the emergency department with VOC. Investigators hypothesize that VR, in addition to standard medical care, will reduce the experience of pain and hospital admissions compared to the historical control group (standard medical treatment).
This Phase 3 study will assess the safety and efficacy of inclacumab, a P-selectin inhibitor, in reducing the frequency of vaso-occlusive crises (VOCs) in approximately 240 adult and adolescent participants (≥ 12 years of age) with sickle cell disease (SCD). Participants will be randomized to receive inclacumab or placebo.
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.
The purpose of this study is to find objective biomarkers of vaso-occlusion (blood vessel blockage) in people with SCD. Using information from earlier studies and work being done, researchers have developed a strategy to image VOC, using positron emission tomography (PET). The ability to see and measure VOC in SCD patients can help patient care, by showing when and how a VOC is occurring or going to occur. Studying this method will also help in future drug research, as it will allow researchers to deliver promising new medications that target hyper-adhesion and sickling in people with SCD.
The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opiate usage, and reduction or elimination of hospital admission. The applicant is developing intravenous L-citrulline (Turnobi™) for treatment of sickle cell disease (SCD). The current development program targets treatment of sickle cell-associated vaso-occlusive crisis (VOC) specifically. The aim of Part 1 is to identify the optimum dose regimens for the Part 2 of the trial which is a double-blind, placebo controlled adaptive 'pick-the-winner' design. This study will allow assignment of more subjects to the better treatment arm/s based on emerging data. The study, initially, will evaluate efficacy and tolerability of incremental doses of intravenous (IV) L-citrulline (Turnobi™) in patients with SCD while receiving standard of care therapy for VOC.
The purpose of the study is to Evaluate the Effect of Ticagrelor versus Placebo in Reducing the Rate of Vaso-Occlusive Crises in Paediatric Patients with Sickle Cell Disease
Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated. This is a phase II randomized controlled clinical trial evaluating the efficacy of virtual reality technology when added to standard pain management for patients with sickle cell disease who are experiencing acute pain crisis in the ambulatory care setting. Patients will be randomized to receive either standard management only or standard management in addition to virtual reality therapy. The remainder of care for the painful event will continue per institutional standards according to clinical indication, including reassessment and documentation of pain and additional doses of pain medicines by intravenous (IV) or oral route. Pain scores and opioid requirement will be measured and compared across treatment arms, along with the outcomes of discharge from clinic versus admission to the inpatient unit. PRIMARY OBJECTIVE: To assess the efficacy of virtual reality (VR) technology in reducing pain at 30 minutes after intervention during an acute vaso-occlusive crisis in patients with sickle cell disease. Primary endpoint will be change in pain scores in Standard versus VR arms, between the first pain assessment at the time of presentation and the subsequent pain assessments up to 30 minutes after intervention. Secondary Objectives: * To compare total opioid consumption from the time of presentation to the time of discharge from acute care setting in Standard versus VR arms. * To assess the efficacy of virtual reality (VR) technology in reducing pain at 60 minutes after the first IV medication administered or 60 minutes after completion VR during an acute vaso-occlusive crisis in patients with sickle cell disease.
Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the nociceptive system, and as a receptor antagonist, ketamine mitigates this. Intravenous (IV) ketamine has demonstrated efficacy in reducing post-operative, chronic, and cancer-related pain in pediatrics, as well as in reducing time to pain control in the emergency department (ED) in adults. Limited studies suggest efficacy in adult opioid-refractory SCD patients. This study is investigating the safety and tolerability of adjuvant low-dose IV ketamine bolus for pediatric SCD VOE in the ED, as well as its efficacy in improving pain control and reducing hospitalization.
The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD) patients.
Sickle cell disease (SCD) is the most common inherited blood disorder affecting 80,000 to 90,000 individuals in the United States.\[10\] There are 13,000 hospital admissions for a sickle cell crises, costing $448 million dollars annually.\[10\] In our hospital, the sickle cell population is known to have some of the longest length of stays. Between October 2014 and September 2015, there were 89 admissions for a vaso-occlusive crisis with an average length of stay of 6 days and 12 admissions greater than 10 days and 5 admissions greater than 20 days. We propose to evaluate the feasibility of the new CPP in a pilot randomized control trial to determine if pain and length of stay can be reduced in patients with sickle cell disease. We also propose to evaluate a sleep regimen to determine if this can reduce the hospital stay and help with pain. We hypothesize increased physical activity and proper sleep, as implemented in the CPP, are correlated with decreased hospital length of stay and decreased pain. Additionally, we believe that creating a standardized nighttime environment at the hospital will help the children stay in their circadian rhythm thus promoting improved sleep and a more effective inpatient disease management.
This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis
This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.
Patients who have sickle cell VOC are usually treated with opioids, such as morphine. However, this current way of treating them has not improved the health, medical outcomes, or rates of hospitalizations. In addition, since VOC can happen very frequently over a long period of time, giving opioids over and over again can cause both short-term and long-term problems. Nitrous oxide (N2O) is a way of treating pain that may provide a better alternative to repeatedly giving opioids over long periods of time. N2O has been shown to provide up to 3 hours of pain relief in inpatient patients with VOC whose pain did not improve with morphine infusions, and is used extensively in France, where almost half of 85 pediatric emergency departments use nitrous oxide to treat children with VOC whose pain did not get better with standard treatment with morphine. However, pain relief which N2O provides in the acute setting has not been well described. Therefore, the purpose of our study is to describe how well N2O can relieve the pain in patients with SCD who present to the emergency department and are experiencing a VOC.
The purpose of this study is to evaluate whether MST-188 can reduce the duration of vaso-occlusive crisis (VOC) in subjects with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in subjects who receive MST-188 to those who do not receive MST-188.
The purpose of this study is to determine if intranasal fentanyl can decrease the pain of patients with sickle cell disease who present to the pediatric emergency department with a vaso-occlusive crisis.
This research is being done to find out the best way to give narcotics for pain relief in adults with sickle cell disease and painful crisis. This study is a comparison of two ways of giving narcotics. The first way is what occurs now in the Emergency Acute Care Unit (EACU) where patients are given a single intravenous (iv) dose of a narcotic which is repeated by the nurse as needed to control the pain. The second way is to provide a single iv dose of narcotic and then allow the patient to push a button and receive one or more additional doses of narcotic when he/she thinks it is needed. Our hypothesis is that PCA will be a more effective way of controlling pain.
The purpose of this study is to determine the safety and efficacy of intravenous magnesium in shortening the duration of a pain crisis and to determine the health-related quality of life and short term outcomes of children treated with intravenous magnesium during an acute pain crisis.
The purpose of this study is to learn more about the feasibility of oral ketamine for the treatment of painful sickle-cell crises in children and adolescents as a supplement to intravenous (IV) opioids. There is a need for improved non-opioid analgesia for patients experiencing sickle-cell crises in the hospital and prehospital setting, as children and adolescents with sickle cell disease who experience sickle-cell crises often have severe pain that is not well controlled by high dose opioids, leading to poor pain management and opioid-related side effects. The study will begin when patients are admitted to the Emergency Department of Boston Children's Hospital for treatment of a sickle-cell crisis. Oral ketamine will be administered every 8 hours for the next 48 hours. Patients will have continuous cardiorespiratory monitoring for the duration of the study, as per routine care, as well as monitoring by the hospital's Acute Pain Service at least twice daily for pain management and side effects of pain treatment. At the end of the 48-hour study duration, patients will discuss with the Pain Service and Hematology Service whether to continue oral ketamine, change to intravenous ketamine, or discontinue ketamine based on clinical indications such as level of pain and sedation while on opioids.
Sickle cell anemia is a genetic blood disorder where red blood cells are abnormally shaped, crescent- or sickle-shaped, instead of the normal, round shape. This misshapen cell is rigid and sticky, causing them to clump together and block small blood vessels. This blockage can lead to pain, infections, and organ damage, and the shortened lifespan of sickle cells causes anemia. The purpose of this study is to explore if hyperbaric oxygen (HBO) therapy would decrease pain and hospital length of stay associated with acute sickle cell pain crisis. Adults presenting with an uncomplicated acute pain crisis (i.e., acute chest syndrome, acute myocardial infarction/stroke) would be eligible. The intervention would be 1-3 hyperbaric oxygen sessions depending on response to therapy. Each treatment session will be approximately two hours in length. Evaluation would be through participants' self--assessment via the visual analog scale for pain level before and after treatments as well as tracking length of stay in the hospital.
In patients with SCD, the use of low dose anticoagulation as an outpatient may lead to a significant decrease in morbidity and as a result, decrease healthcare utilization and costs. This study attempts to critically avoid admissions by reducing daily pain scores and pain crisis as an outpatient by use of a novel oral anticoagulant.
This study aims to characterize sex differences in the pathophysiology of vaso-occlusive crises (VOC) occurring among individuals with sickle cell disease (SCD). * The study will compare CRP and other biomarkers between females with SCD in the follicular phase of the menstrual cycle and males with SCD. * The study will explore potential sex differences in biomarker changes between females and males with SCD during and following resolution of VOC. * The study will compare neutrophil and platelet adhesion to the endothelium and real time fibrin deposition in the blood.
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
This will be a descriptive cohort study of intranasal ketamine as the initial analgesic for children with sickle cell disease who present to the pediatric emergency department with vaso-occlusive crisis and are awaiting intravenous line placement.
The purpose of this study is to determine whether giving abciximab (ReoPro) to children with sickle cell disease who are hospitalized for acute pain crisis will improve their pain and shorten the time spent in the hospital, when compared with standard supportive care.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).