Active clinical trials for "Syndrome"

OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders. II. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells. Chemoprotective drugs such as amifostine may protect normal cells from the side effects of chemotherapy. PURPOSE: Phase II trial to study the effectiveness of combining topotecan and cytarabine given with amifostine in treating patients who have myelodysplastic syndrome.

This study will evaluate the use of intravenous immunoglobulins (IVIG) at a dose of 1g/Kg/body weight given every three weeks for 6 infusions in pediatric subjects ages 4 - 16 years with moderate to severe PANS. The study will compare biomarkers and behavioral scales before treatment, after the last infusion, 2 months, and at a minimum 6 months post-treatment.

Objective of this study is to assess the efficiency of NOVATEX MEDICAL compression garments in patients with an Ehlers-Danlos syndrome (EDS). To answer this objectif a comparison before/after use of compression garments will be performed for all patients.

Ticagrelor is a potent and fast-acting P2Y12-ADP receptor antagonist recommended as first-line agent in ACS (2). This drug was associated with a 20% relative reduction in the rate of MACE in ACS patients undergoing PCI compared to clopidogrel. This benefit came without any increase in major bleedings compared to clopidogrel (6). In the PLATO trial, a limited number of kidney failure patients were included (21%) and patients with terminal CKD were excluded. A sub-group analysis focused on CKD patients was performed. Only 214 patients with CKD below stage 4 (creatinine clearance <30 ml/min) were included (7). No patient with terminal CKD or undergoing chronic hemodialysis was included. Of importance, kidney function impairment is frequent and affects up-to 40 % of ACS patients. In addition, CKD is a powerful independent predictor of ischemic complications during ACS (8-9).Indeed, CKD patients have a very high risk of MACE following ACS with an odd ratio between 2 and 3 compared to patients with normal kidney function and event rates above 40% at one year follow-up (8-13). Of importance these patients more often have high on-clopidogrel platelet reactivity which was strongly associated with a worse clinical outcome (3,14-16). In CKD patients HTPR was associated with death after PCI (15). Accordingly ticagrelor which overcomes these limitations of clopidogrel could be associated with a major clinical benefit in severe or terminal CKD patients. Most of ticagrelor and is active metabolites are excreted through the feces. Preclinical data suggested that renal impairment had little effect on systemic exposure to the drug(EMEA/H/C/1241 (28)). Recent pharmacodynamic and kinetic studies confirmed these preclinical data on the safety of ticagrelor in severe and end-stage CKD (17-19). Therefore based on the rational above and to the lack of relevant clinical data, the optimal P2Y12-ADP receptor antagonist for patients with stage 4 and 5 and patients undergoing chronic dialysis remains undetermined in ACS treated with PCI. We aimed to compare the clinical efficacy ticagrelor and clopidogrel in patients with stage 4 and 5 or on chronic hemodialysis undergoing PCI for ACS.

This retrospective, multi-center, chart review study will collect patient data from medical charts of pediatric patients who have been treated with Korlym for Cushing's syndrome.

The study team will evaluate the medication Hydroxocobalamin (B12a) for treatment of low blood pressure after cardiac surgery.

The purpose of this study is to test the safety and efficacy (benefits) of an investigational drug LB-100, for treatment of myelodysplastic syndromes. LB-100 has previously been administered to patients with various solid tumors. In this study, LB-100 will be administered as an intravenous infusion over 120 minutes. This study will be conducted in 2 phases. In phase Ib, escalating doses of LB-100 will be administered to patients to study the safety and to determine a safe dose of LB-100. In phase 2, patients will be administered LB-100 at the dose that was found to be safe in phase Ib. The efficacy (benefits) and safety of LB-100 will be determined in this phase of the study.

A prospective, nonrandomized, open-label, single-arm clinical trial to study efficacy of rapamycin (sirolimus) in the treatment of Blue Rubber Bleb Nevus Syndrome, hereditary or sporadic venous malformation

This study is intended to determine if compression garments effectively reduce pain in the treatment of tibial stress syndrome (TSS) in the military population. The study will include diagnoses of shin splints, which is medically known as tibial stress syndrome and medial tibial stress syndrome, and compare a treatment group using compression garments to a control group using passive recovery. Both groups will engage in a progressive walk-to-run program. The outcomes will be measured by comparison in pain scales pre and post treatments. The ability to perform a two mile run without pain symptoms will be assessed at the conclusion of the study by participant's self-reported evaluation. The TSS screening questionnaire will be obtained at each medical encounter to include the initial visit and follow-up visits at the four and eight week (+/- 3 days) marks. The self-reported 2-mile run evaluation is included on the TSSS questionnaire.