
A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease
Sickle Cell DiseaseAcute Chest SyndromeAcute chest syndrome (ACS), a lung complication in sickle cell disease (SCD), is the second most common cause of hospitalization and leading cause of death in SCD. ACS is associated with airway inflammation, and a major cause is pulmonary infection from atypical organisms. To date, there are no drugs available to reduce inflammation and risk of recurrent ACS. Macrolides are a group of antibiotics that exert immunomodulatory and anti-inflammatory actions both in vitro and in vivo. In addition, macrolides reduce bacterial burden in the airway of atypical organisms, all of which play an important role in the pathophysiology of ACS. Numerous studies have evaluated macrolide prophylaxis in conditions associated with lung inflammation, such as cystic fibrosis, asthma, bronchiectasis etc., and high quality evidence have found macrolides to be beneficial as a disease modifying agent that leads to improvement in airway inflammation, reduced pulmonary exacerbations and improved lung function. The investigators hypothesize that azithromycin prophylaxis is well tolerated and has the potential to reduce inflammation and improve lung outcome in children with SCD with a history of ACS. A prospective, single arm, open label feasibility study of azithromycin prophylaxis will be performed in children with SCD with a history ACS with the specific aim to examine the feasibility, safety and tolerability of azithromycin prophylaxis administration in participants with SCD , and to examine whether azithromycin prophylaxis has the potential to improve lung outcome. In addition, this study will determine whether azithromycin prophylaxis reduces inflammation in participants with SCD with a history of ACS.

Patent Versus Generic Clopidogrel in Acute Coronary Syndrome
Acute Coronary SyndromeIschemic heart disease is the leading cause of death and disability in developed countries and is responsible for a third of deaths in persons over 35 years . The most severe form of ischemic heart disease is sudden death and acute coronary syndrome (ACS). There is evidence that early and optimal treatment of ACS decreases mortality. Within the optimal treatment, these patients must receive a reperfusion therapy as mechanical or pharmacologic treatment. In addition to reperfusion treatment, antiplatelet therapy is a central part of the management. Aspirin plus a P2Y12 inhibitor have been shown to decrease mortality. In our country, clopidogrel is the more accessible and used P2Y12 inhibitor; however, it has been shown to have a wide variability in response and this variability could be influenced by different pharmacological, genetic and environmental factors. Platelet reactivity measured by aggregometry predicts major cardiovascular events in ACS patients treated with clopidogrel. Due to their frequent prescription, generic clopidogrel efficacy must be evaluated. The purpose of this study is to compare the platelet reactivity in patients with ACS receiving clopidogrel generic versus patent.

the Effect of Diane-35 Pretreatment on In-vitro Fertilization Outcome for Patients With Polycystic...
Polycystic Ovary SyndromePolycystic Ovary Syndrome is the most common endocrine disorder in women, is estimated to affect more than 5% of the population and is associated with chronically elevated serum androgen concentrations.Evidence suggests that polycystic ovary syndrome has a negative impact on pregnancy outcomes, with an increased risk of gestational diabetes, hypertensive disease during pregnancy,and preterm birth,which is partially related with the hyperandrogenic environment.Diane-35 has been proved to be the most effective anti-androgenic drug.At present, there is no direct evidence that Diane-35 has a positive effect on the clinical outcome of polycystic ovary syndrome patients undergoing In-vitro fertilization/Intracytoplasmic sperm injection.

Lenalidomide After Failure of Hypomethylating Agents in Myelodysplastic Syndrome
Myelodysplastic SyndromeThis is a Phase II study to evaluate the efficacy of second-line lenalidomide monotherapy for myelodysplastic syndrome (MDS) patients who failed to hypomethylating agents.

Effects of Micronized Trans-resveratrol Treatment on Polycystic Ovary Syndrome (PCOS) Patients
Polycystic Ovary Syndrome (PCOS)The purpose of this study is to determine whether micronized trans-resveratrol improves clinical (excessive hair, menstrual cycle), endocrine (androgens)and metabolic (lipids, markers of systemic inflammation) in women with polycystic ovary syndrome (PCOS).

Safety of 6-month Duration of Dual Antiplatelet Therapy After Acute Coronary Syndromes (SMART-DATE)...
Acute Coronary SyndromeObjective : To test the safety of 6 month-duration of dual antiplatelet therapy (DAPT) compared to conventional 12-month-or-longer duration after second-generation drug-eluting stent (DES) implantation in patients with acute coronary syndrome (ACS). Hypothesis : A 6-month duration of DAPT is non-inferior to a conventional 12-month-or longer duration of DAPT at preventing the occurrence of major adverse cardiac and cerebrovascular events (MACCE) at 18-month after second-generation DES implantation in patients with ACS.

The Oxford Marfan Trial
Marfan SyndromeThe primary objective of the trial is to estimate the effects of allocation to irbesartan, or doxycycline, or a combination of both irbesartan and doxycycline, compared with placebo, on measures of elastic function of the aorta in people with the Marfan syndrome and enlargement of the aorta.

Intensive Education on Lipid Management
Acute Coronary SyndromeAmong the patients with coronary heart disease, those with ACS (acute coronary syndrome) are extremely high-risk patients. Therefore, management outside hospital, especially the regular administration of drugs, is vital to prevent the recurrence of cardiovascular events. However, most patients often fail to stay on a long-term administration regimen, especially the administration of statins. According to the statistics, the average duration adhered with statin in patients with ACS is less than 3 months, use of statin at hospital discharge was only 80% and 65% in 6 month, with a very low LDL-C control rate (about 11% at 6 months), which poses a threat to the recurrence rate of cardiovascular events in patients with ACS. It was found in previous studies that there were many factors influencing patients' compliance, in which patients' refusing to take medicine accounted for a higher proportion. It suggests that patient had not recognized the importance of long-term administration. Therefore, it is extremely important for physicians to strengthen patient education and regular follow-up visits during disease management. Moreover, the effectiveness of patient education during chronic disease management has already been proved in some studies abroad, and the interventional effect of multiple patient education process outweighs that of single approach education. Thus, we intend to conduct a randomized and controlled study to explore the effect of multi-channel intensive patient education on LDL-C target achieving rate and statin adherence in patients with ACS in China.

Study to Evaluate Safety and Efficacy of Dexpramipexole (KNS-760704) in Subjects With Hypereosinophilic...
Hypereosinophilic SyndromeBackground: - Eosinophils are white blood cells that fight infections. In people with hypereosinophilic syndrome (HES), eosinophil levels are too high and can damage their organs. HES is usually treated with steroids, but steroids can cause side effects and stop working over time. Researchers want to see if a drug called dexpramipexole, being developed by Knopp Pharmaceuticals, can help people with HES to reduce their steroid dose. Objective: - To test whether dexpramipexole can reduce the steroid dose needed to control eosinophilia and HES symptoms. Eligibility: - Adults 18 and older with HES who respond to steroids, but need more than 10 mg daily to control eosinophilia and symptoms. Design: The study will last 9 months with 6 visits to NIH. Participants will be screened with medical history, physical exam, and urine and blood samples. Participants steroids will be tapered to the lowest effective dose. During this time, blood will be drawn weekly. Participants will take this dose for 2 weeks before starting the study drug. Participants will take the study drug twice daily by mouth for 12 weeks along with steroids. The steroid dose will not be decreased during this time and participants will be seen monthly for a medical history, physical examination and blood work. Just before and 12 weeks after starting the study drug, the following tests will be performed: medical history and physical exam blood and urine tests lung function tests electrocardiogram (measures heart electrical activity) echocardiogram (takes pictures of the heart using sound waves) bone marrow biopsy (a needle inserted into the hip bone that removes bone marrow cells for study) After 12 weeks, the participants steroid dose will be tapered again to the lowest effective dose while on study drug. Two weeks after the lowest effective dose is reached, participants will return for a medical history, physical examination, blood work, lung and heart tests. Participants who respond to the study drug may be able to continue to receive the drug on a planned separate study. Four weeks after stopping the study drug, participants will have medical history, physical exam, and blood tests.

The Effect of Combination Ultra Q10 and L-carnitine on the Course of Myelodysplastic Syndrome
Myelodysplastic SyndromeThe purpose of this study is determine whether combination Q10 , L-carnitine and multivitamin and mineral complex is effective in treatment of patients with low and intermidiate1-2 risk Myelodysplastic syndrome