Treatment of Complex Regional Pain Syndrome With Once Daily Gastric-Retentive Gabapentin (Gralise)...
Complex Regional Pain Syndrome I (CRPS I)The purpose of this study is to see if an FDA-approved drug (Gralise) can help people with certain types of neuropathic pain without causing too many side effects.
Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic...
HemochromatosisMyelodysplastic SyndromesHypothesis: Deferasirox can be used as a therapeutic agent to deplete the liver, heart and bone marrow of excess iron in patients with iron overload caused by myelodysplastic syndrome (MDS) and hemochromatosis (HC. Assess the effect of new serum biomarkers (NTBI and hepcidin) and MRI as indicators of iron overload and their usefulness to monitor iron depletion treatment. Study the effect of iron overload and iron depletion on intracellular signal transduction, trace metals concentrations in serum and urine and markers of oxidative stress in blood cells and urine.
A Study of Vismodegib in Patients With Relapsed/Refractory Acute Myelogenous Leukemia and Relapsed...
Myelodysplastic SyndromesMyelogenous Leukemia1 moreThis study will assess the safety and efficacy of vismodegib in patients with relapsed/refractory acute myelogenous leukemia (AML) and relapsed/refractory high-risk myelodysplastic syndrome (MDS). Patients in Cohort 1 will receive single-agent vismodegib 150 mg orally daily. In Cohort 2, patients will receive vismodegib 150 mg orally daily in combination with cytarabine 20 mg subcutaneously for 10 days. Anticipated time on study treatment is until disease progression, intolerable toxicity, or patient withdrawal of consent.
Engage 24: Evaluation of the Safety and Effectiveness of URG101 in Subjects With Interstitial Cystitis/Bladder...
Interstitial CystitisBladder Pain SyndromeThe purpose of this study is to determine if the combination product (URG101) is safe and effective versus its individual components (Heparin Sodium and Lidocaine Hydrochloride) for the treatment of Interstitial Cystitis/Bladder Pain Syndrome. The study is randomized and double-blinded such that neither the subject nor doctor will know which drug or placebo is received.
Study of Vadastuximab Talirine (SGN-CD33A; 33A) in Combination With Azacitidine in Patients With...
Myelodysplastic SyndromeThis is a phase 1/2 study to evaluate the combination of vadastuximab talirine (SGN-CD33A; 33A) and azacitidine in subjects with previously untreated International Prognostic Scoring System (IPSS) Intermediate-2 or high risk myelodysplastic syndrome (MDS).
The Use of DPP-4 Inhibitors in Short Bowel Syndrome
Short Bowel SyndromeThe inhibition of Dipeptidyl peptidase-4 should increase the concentration of glucagone-like peptide 1 and 2, and the increase of the latter should increase the absorptive capacity of the intestine.
Comparison of the Efficacy and Safety of T2762 Versus Optive® in the Treatment of Moderate to Severe...
Dry Eye SyndromeThe purpose of this study is to compare the efficacy and safety of T2762 versus Optive® in the treatment of moderate to severe Dry Eye Syndrome.
Rasburicase and Allopurinol in Treating Patients With Hematologic Malignancies
Adult Acute Myeloid Leukemia With 11q23 (MLL) AbnormalitiesAdult Acute Myeloid Leukemia With Del(5q)17 moreThis randomized phase II trial studies how well giving rasburicase together with allopurinol works in treating patients with hematologic malignancies. Rasburicase may reduce the level of uric acid in the blood. Allopurinol may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. It is not yet known which dose of rasburicase is more effective in treating hematologic malignancies when given together with or without allopurinol.
An Open Label Extension Study in Subjects With Fragile X Syndrome
Fragile X SyndromeThis study will enroll subjects who have completed Protocols 209FX301, 209FX302, or are currently participating in Protocol 2202 into a long-term study in which all subjects will receive active drug (arbaclofen).
Liraglutide Use in Prader-Willi Syndrome
Diabetes Mellitus Type 2Prader Willi SyndromeLiraglutide is a glucagon like peptide -1 (GLP-1) agonist, which is approved for use in patients with type 2 diabetes. Studies of liraglutide have shown an appetite suppressive effect and has been associated with weight loss in patients with type 2 diabetes. Liraglutide use in the treatment of PWS is limited to one case report by Cyganek et el (See attached Citation). In this case report, the subject showed improvements in hemoglobin A1c and body weight over 14 weeks. The investigators plan to examine the use of liraglutide in a single subject with Prader Willi Syndrome and type 2 diabetes for one year. The investigators will examine clinical parameters, clinical assessment of hunger, and biochemical markers of diabetes and lipid control.