View clinical trials related to Syndrome.
Filter by:The goal of this clinical research study is to learn if it is safe and feasible to transplant patients with one of two units of cord blood that has been changed in the laboratory before it is given. Only patients with leukemia, lymphoma or myelodysplastic syndrome will be allowed on this study. The secondary goal is to obtain the preliminary efficacy outcome. Researchers also want to learn if using cord blood that has been changed can help to control the disease. One cord blood unit will not be changed before it is administered to you. The cord blood unit that will be altered will be changed to use sugar that is found in small amounts in blood cells. It plays a role in telling transplanted cells where they should go in the body. Adding more sugars to the cord blood cells in the laboratory helps the cord blood cells find their way to the bone marrow faster. This process is called fucosylation. "Conditioning" is the chemo and other medicines and will be given to patients to prepare to receive cord blood transplant cells. This prevents immune system from rejecting the cells. Conditioning will be started before the transplant. ATG is a protein that removes immune cells that cause damage to the body. Clofarabine is designed to interfere with the growth and development of cancer cells. Fludarabine is designed to interfere with the DNA of cancer cells, which may cause the cancer cells to die. This chemotherapy is also designed to block your body's ability to reject the donor's bone marrow cells. Melphalan and busulfan are designed to bind to the DNA of cells, which may cause cancer cells to die. MMF and tacrolimus are designed to block the donor cells from growing and spreading in a way that could cause graft versus host disease (GVHD -- a condition in which transplanted tissue attacks the recipient's body). This may help to prevent GVHD. Rituximab is designed to attach to cancer cells, which may cause them to die. A Phase I study for treatment of patients (N=25) with hematologic malignancies and MDS who are candidates for dual-cord UCBT is ongoing at M.D. Anderson Cancer Center under an Investigator-initiated IND Application, E.J. Shpall, MD, PI. Since August, 2012, Preliminary results indicate that ASC-101 UCBT is well-tolerated and no ASC-101 related untoward adverse events have been observed. To date, the median time to neutrophil engraftment (N=9) is 15 days, and the median time to platelet engraftment (N=9) is 33 days. The trial remains ongoing.
Expanded access to Stiripentol for patients with Dravet Syndrome.
To evaluate the safety, efficacy and pharmacokinetics of gabapentin enacarbil in moderate to severe primary RLS patients with moderate renal impairment and to confirm dosage and administration in such population.
Background: - Li Fraumeni Syndrome (LFS) is a highly penetrant, autosomal dominant cancer predisposition disorder. Four main cancer types including sarcoma, adrenocortical carcinoma, breast cancer, and malignant brain tumors commonly characterize LFS but the syndrome can include other cancers. - Metformin is an oral biguanide drug that is approved by the FDA for the treatment of type II diabetes. Metformin has been associated with reduced cancer risk in several epidemiologic studies and reduced cancer mortality in patients with type 2 diabetes. - Metformin decreases circulating insulin and IGF1, and promotes glucose uptake in skeletal muscle and inhibits gluconeogenesis in the liver. Elevations in circulating insulin and IGF1 levels have been associated with increased cancer risk. - Preclinical research in animal models shows that metformin may be more toxic in cancer cells that have lost p53 function. - Lifetime risk of cancer in LFS patients with germline TP53 mutations is estimated to be up to 70% by age 60, with women having excess lifetime cancer risk (up to 100%) compared to men (up to 80%). There are currently no approved chemopreventive agents for patients with LFS. - Metformin has been shown to be safe and tolerable in diabetic and non-diabetics, and may be an ideal candidate for chemoprevention of cancer in this population. Objectives: - Determine the tolerability of oral daily metformin in patients with LFS caused by germline TP53 mutations. - Determine if 8 weeks of daily metformin administration has any effect on circulating IGF-1, insulin, and IGFBP3 Eligibility: - Must have a germline TP53 mutation and provide documentation of testing. - Must have adequate organ function. - Age greater than or equal to 18 years. Design: - This is a pilot study to assess the tolerability of daily oral metformin administration in patients with LFS caused by germline TP53 mutations and to study the effect of metformin on biomarker levels in these subjects. - In the absence of intolerable toxicity, a minimum of 22 patients will take metformin by mouth for a total of 14 weeks and then discontinue metformin for 6 weeks. The total time on study will be 20 weeks. - Patients will be assessed for biomarker levels (IGF-1, insulin, IGFBP3) by blood sample at baseline, and weeks 0 and 8.
Study WCMC IST/VOS/MDS evaluates the safety and tolerability of escalating doses of vosaroxin in adult patients with pathologically confirmed Myelodysplastic Syndrome, or MDS, (< 20% blasts in bone marrow, peripheral blood, or both) by World Health Organization (WHO) classification with an intermediate 2 (INT-2) or high-risk score (ie, ≥ 1.5) as assessed by the International Scoring System (IPSS) after failure of hypomethylating agent-based therapy. Based on 3 completed studies and xenograft models, Vosaroxin is hypothesized to be safe and will effective in this patient population.
This study is designed to investigate whether the treatment of non-anemic iron deficiency with intervenous iron sucrose will result in decreased symptom reporting and improved cardiovascular indices in adolescents (age 12-21) with Postural Orthostatic Tachycardia Syndrome (POTS).
The primary objective is to demonstrate safety and tolerability of switching patients with Restless Legs Syndrome (RLS) from an oral dopamine agonist to rotigotine. As a secondary objective, the investigators will evaluate control of RLS symptoms on rotigotine compared to the prior oral regimen.
Background: - The human immunodeficiency virus (HIV) causes acquired immune deficiency syndrome (AIDS). Combination antiretroviral therapy (ART) drugs treat HIV infection. They generally decrease the amount of HIV virus in the blood (called viral load) to very low levels. This happens only if the drugs still fight HIV and if taken every day exactly as prescribed. When not taken as directed, or if the ART drugs are not strong enough, the virus can become resistant to them, and the ART will not work to control the virus. Researchers want to know how to control HIV in people who can t lower their viral load with their current ART drugs. Objective: -<TAB>To better control HIV in people who can t get a lower viral load even with ART drugs and to learn more about why the HIV is not under control. Eligibility: - People at least 18 years old and with HIV. - People who have been on at least two combinations of ART drugs (including current ART). - People whose last two viral loads were greater than 1,000 copies/mL. Design: - Participants will be screened with medical history, physical exam, and blood tests. - Participants will then have a baseline visit. They will have another physical exam, blood tests, plus answer questions about what they know about HIV and ART, and how they take their ART. - Participants will arrange to stay in the NIH hospital for 7 8 days. - They will take their medications as usual. At the time to take the ART drugs, they will have to ask a nurse to bring them. If they forget, the nurse will bring them. - Participants will meet with a doctor, pharmacist, social worker and nurse to discuss ways to help participants remember to take their drugs. - Participants will have blood drawn about every other day. - Researchers will study the test results. Some participants will be put on different ART drugs. If that happens, participants will have another NIH hospital stay for 7-8 days. - Participants will have 4 follow-up visits over 12 weeks, then every 3 months for 2 years or more.
This is a historical cohort study with retrospective collection of data comprising all Nordic patient's diagnosed with Eisenmengers syndrome in the period 1977 through 2011. The goal is to determine prognostic factors for mortality and morbidity.
The purpose of this study is to determine if a lower back treatment would change the knee pain intensity and the level of functional activities as well as the muscle activities in people with anterior knee pain. The investigators hypothesized that the lower back treatment may change the hip and knee muscle activities as well as reduce the knee pain intensity and improve the functional activities in people with anterior knee pain.