Active clinical trials for "Syndrome"

This research is being performed to try to understand if the use of deep brain stimulation or DBS can treat the symptoms of Tourette syndrome that do not respond well to current medications. In order to do this the investigators will place small stimulation leads on both sides of the brain in a region (a portion of the thalamus) that may alter the abnormal activity in the brain contributing to the symptoms of Tourette syndrome. This requires two surgical procedures, and several preoperative and postoperative visits for tuning the stimulation parameters and recording stimulation effects. The FDA has not approved DBS for use in people with Tourette syndrome, and Medtronic (the manufacturer of the device) has not conducted testing for the system in Tourette syndrome. Therefore its use in this study is experimental.

Objective: To determine the metabolic effects of the adrenalectomy in subclinical Cushing's syndrome of adrenal incidentaloma. Methods: 234 subclinical cushing syndrome and adrenal incidentaloma will be randomly assigned into two groups(e.g adrenalectomy group and conservative treatment group).

Background: - Researchers want to develop better ways to treat cancer. In this study, they will give people with cancer two drugs. These drugs have been used on their own to treat some blood cell cancers. Objectives: - To test the safety and efficacy of the drug combination of bortezomib and clofarabine. Eligibility: - Adults age 18 and over with advanced cancer that has progressed after receiving standard treatment or that has no effective therapy. Design: Participants will be screened with medical history, physical exam, and scans to measure their tumors. They will also have heart, blood, and urine tests. All of these may be done by their regular doctors. Participants will get the study drugs in 21-day cyles. They will stay at the clinic for week 1 of every cycle, then have 2 weeks off. <TAB>- Bortezomib will be injected under the skin on days 1 and 4. <TAB>- Clofarabine will be injected in a vein for days 1-5. During cycle 1 only, participants will go to the clinic or their doctor to have a physical exam and blood tests at the start of the second and third week. Participants will have clinical evaluations throughout the study, including before receiving treatment and then before the start of each cycle. Participants may stay in the study as long as they are tolerating the drugs and their tumor is not getting worse. Participants will have follow-up for 30 days after the last dose of study drugs. The first part of this study tests the safety of different doses of clofarabine and bortezomib. The second part of this study involves a separate group of participants who will undergo mandatory research biopsies to learn more about the effects of clofarabine and bortezomib on cancer cells.

The purpose of this study is to evaluate the safety and efficacy of custom made devices, Zenith t-Branch devices and physician modification of FDA approved off-the-shelf endovascular grafts in the treatment of patients with complex abdominal aneurysms, aortoiliac aneurysms, thoracoabdominal aneurysms and aortic arch aneurysms who (1) have anatomy not suitable for endovascular repair using grafts currently marketed in the United States,(2) are deemed unsafe to wait the required time necessary for commercial endograft manufacturing, and (3) are at high risk for open surgical repair. Amendment to the study has created a cohort open to people with connective tissue diseases such as Marfan, Ehlers-Danlos or Loey-Dietz syndromes to enroll in the trial. An additional amendment to the study allows the use of a custom made device to treat an aneurysm in the aortic arch.

This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.

Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications. By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications. Objectives: - To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient. Eligibility: Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants. Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program. Design: All participants will be screened with a physical examination and medical history. DONORS: Donors will undergo an initial apheresis procedure to donate white blood cells. After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood. After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood. RECIPIENTS: Recipients will provide an initial donation of white blood cells to be used for research purposes only. From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant. After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion. After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant. Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.

RATIONALE: Giving chemotherapy, such as busulfan, fludarabine, and melphalan, before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal or cancer cells and prepares the patient's bone marrow for the 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 cyclosporine and mycophenolate mofetil may stop this from happening. PURPOSE: This phase II trial is studying how well combination chemotherapy followed by a donor umbilical cord blood transplant works in treating infants with high-risk acute leukemia or myelodysplastic syndromes.

The goal of this clinical trial is to compare outcomes of two reduced intensity conditioning (RIC) regimens (fludarabine plus busulfan and fludarabine plus melphalan) in allogeneic hematopoietic stem cell transplantation (HSCT) for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) patients. The main questions it aims to answer are: The safety of two reduced intensity conditioning (RIC) regimens (fludarabine plus busulfan and fludarabine plus melphalan) in allogeneic hematopoietic stem cell transplantation for adult AML/MDS patients with HCT-CI≥3 or aged ≥55 years. The efficacy of two reduced intensity conditioning (RIC) regimens (fludarabine plus busulfan and fludarabine plus melphalan) in allogeneic hematopoietic stem cell transplantation for adult AML/MDS patients with HCT-CI≥3 or aged ≥55 years. Participants will be randomized to one of two reduced intensity conditioning (RIC) regimens (fludarabine plus busulfan and fludarabine plus melphalan)

This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.

This clinical trial aims to learn about the electrophysiological and clinical effects of radiofrequency catheter ablation in patients with Brugada syndrome. The main questions it seeks to answer are: What are the immediate effects of catheter radiofrequency ablation in cardiac electrophysiology? What is the relation between invasive and clinical features in patients with Brugada syndrome undergoing ablation? Researchers will compare ablation and control groups to see if there is a difference in clinical and invasive markers of the disease in one year of follow-up.