
Study to Assess the Efficacy and Safety of Different Doses of BIM 23A760 in Patients With Carcinoid...
Carcinoid SyndromeThe purpose of the protocol is to assess the efficacy and safety of BIM 23A760 on patient's overall satisfaction in terms of symptom relief (diarrhoea and/or flushes) in patients with carcinoid syndrome after 24 weeks of treatment.

Efficacy and Tolerability of the Combination of Valproic Acid and Lenalidomide in the Treatment...
Myelodysplastic Syndrome MDSAs part of a palliative therapy concept, feasibility, toxicity, and effectiveness of treatment with the combination of Valproic acid and lenalidomide in Myelodysplastic Syndrome patients with a favorable risk profile will be investigated.

Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
Wiskott-Aldrich SyndromeThrombocytopenia1 moreThe purpose of this project is to describe the pathophysiology of thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome (WAS) and determine the response to thrombopoietic agents in vitro and in vivo.

An Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
Fragile X SyndromeStudy 22001, "A Double-Blind, Placebo-Controlled, Flexible-Dose Evaluation of the Efficacy, Safety, and Tolerability of STX209 in the Treatment of Irritability in Subjects with Fragile X Syndrome" currently is evaluating the efficacy of STX209 (R-baclofen) for management of typical problem behaviors, such as irritability and aggression, in subjects with FXS. This study (22002) will enter subjects who complete Study 22001 into a long-term, open-label study.The open-label extension protocol will provide necessary data on the long-term safety and tolerability of STX209 among subjects with FXS who receive treatment under conditions more closely reflective of their general medical care.

Alemtuzumab, Busulfan, and Cyclophosphamide Followed By a Donor Stem Cell Transplant in Treating...
Graft Versus Host DiseaseLeukemia2 moreRATIONALE: Monoclonal antibodies, such as alemtuzumab, can find cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Giving chemotherapy drugs, such as busulfan and cyclophosphamide, before a donor stem cell transplant helps stop the growth of cancer cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving tacrolimus and methotrexate after the transplant may stop this from happening. PURPOSE: This phase I/II trial is studying the best dose of alemtuzumab when given together with busulfan and cyclophosphamide followed by a donor stem cell transplant and to see how well it works in treating patients with hematologic cancer.

Effect of Ranitidine on Hyper-IgE Recurrent Infection (Job's) Syndrome
JOB's SyndromeHyper-IgE Recurrent Infection Syndrome1 moreThis study will examine the safety and effectiveness of ranitidine (Zantac) in patients with Hyper-IgE recurrent infection syndrome, a disease characterized by recurrent infections of the ears, sinuses, lungs and skin, and abnormal levels of the antibody immunoglobulin E (IgE). Patients age 2 and older who have Hyper-IgE recurrent infection syndrome and who have had chronic or frequent infections in the last 12 months may be eligible for this study. Participants are randomly assigned to take ranitidine or placebo in pill or liquid form twice a day for 12 months. In addition to treatment, patients undergo the following procedures during visits scheduled on day 0 of the study (baseline) and at 3, 12, 15 and 24 months. Evaluations at 6, 9, 18 and 21 months are by telephone. Medical history and physical examination - baseline and 3 and 24 months. Clinical severity score - baseline and 3, 6, 9, 12, 15, 18, 21 and 24 months. Dermatology exam - baseline and 3, 12, 15 and 24 months. Pulmonary function test - baseline and 12 and 24 months. Chest CT - baseline and 12 and 24 months. Quality of life assessment - baseline and 3, 12, 15 and 24 months. Pregnancy testing - baseline and 3, 12, 15 and 24 months. HIV test - baseline and 12 and 24 months. Contraception evaluation - baseline and 3, 6, 9, 12, 15, 18, 21 and 24 months. Missed school/work days assessment - baseline and 3, 12, 15 and 24 months. Medication adherence - baseline and 3, 6, 9, 12, 15, 18, 21 and 24 months. In addition to the above procedures, participants who are not enrolled in study 00-I-0159 have a baseline scoliosis series and genetic consult.

Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis...
Graft Versus Host DiseaseLeukemia3 moreRATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving tacrolimus and mycophenolate mofetil before and after transplant may stop this from happening. PURPOSE: To look at the ability of umbilical cord blood cells from one or two unrelated donors to serve as a source of stem cells for people needing a bone marrow transplant.

Treatment of Refractory Excessive Daytime Sleepiness in Patients With Obstructive Sleep Apnea/Hypopnea...
Sleep ApneaObstructive2 moreThe purpose of this study is to determine the safety and efficacy of MK0249 in treating refractory excessive daytime sleepiness (EDS) in patients with Obstructive Sleep Apnea/Hypopnea Syndrome (OSA/HS) using nasal continuous positive airway pressure (nCPAP) therapy.

Polycystic Ovarian Syndrome: Impact of Flaxseed Supplementation
Polycystic Ovarian SyndromeThe overall aim of this study is to explore the effects of flaxseed supplementation and determine whether it is a feasible and potentially effective dietary intervention among women with polycystic ovarian syndrome (PCOS). Women, ages 18-45, with clinically confirmed PCOS (N=20) will be scheduled for baseline measures and then instructed and given supplies necessary to follow a flaxseed supplemented (30 g/day) diet for a period of three months, whereupon follow-up measures will be taken. Subjects will be asked to resume their typical (unsupplemented) diet for another three months and a second set of follow-up measures will be taken. Baseline levels of bioavailable and total testosterone, fasting insulin, glucose, and triglycerides (TG), total/LDL/HDL cholesterol, body weight, degree of hirsutism and acne, and menstrual cyclicity will be compared to levels at 3 and 6 month follow-up. The overall hypothesis (based upon our work in men at risk for prostate cancer and data from one case-study conducted in a woman with confirmed PCOS) is that during the time women receive flaxseed supplementation they will experience reduced serum levels of testosterone, and total and LDL cholesterol, as well as clinical evidence of hirsutism. Given the pilot nature of this study, statistical analyses will be limited to simple descriptive statistics. We have observed no negative side effects, other than minor gastro-intestinal occurrences (i.e., temporary increased flatulence, borborygmi, increased number of stools, etc.) associated with flaxseed supplementation in our previous studies either with short or long-term use. Flaxseed supplementation will be discontinued if indicated and the events reported to the institutional review board.

Study of Association of Arsenic Trioxide (ATO) and Ascorbic Acid in Myelodysplastic Syndromes
Myelodysplastic SyndromesThis is a prospective, multicenter phase II trial designed to evaluate the safety and activity of the combination of association of arsenic trioxide (ATO) and ascorbic acid in patients with myelodysplastic syndromes