
The Effect of Topical Brimonidine Tartrate on Hand-foot Syndrome (HFS) in Cancer Patients
Hand-foot SyndromeComparative analysis of the severity of Hand-Foot-Syndrome (HFS) of palms treated with brimonidine tartrate gel or with standard care Urea 10% containing lotion in cancer patients receiving antineoplastic therapy to show a preventive effect of cutaneous brimonidine treatment on severity of HFS symptoms.

Study of Lademirsen (SAR339375) in Patients With Alport Syndrome
Alport SyndromePrimary Objectives: To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function. To assess the safety and tolerability of lademirsen (SAR339375) in participants with Alport syndrome. Secondary Objectives: To assess plasma pharmacokinetic (PK) parameters of the parent compound and its active major metabolite. To assess the potential formation of anti-drug antibodies (ADAs) following administration of lademirsen (SAR339375). To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes in renal injury and function biomarkers.

HL2351 CAPS Phase II Study
Cryopyrin-Associated Periodic Syndromes (CAPS)This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).

Melatonin Treatment for Night-Eating Syndrome
Night-eating SyndromeThis is an 8-week randomized, double-blind, placebo-controlled, parallel, fixed-dose pilot clinical trial to test the effectiveness of melatonin treatment versus placebo on symptoms of Night Eating Syndrome (NES). The investigators will also look at the effect of melatonin treatment on weight, metabolic parameters and sleep parameters in individuals with NES.

NAFLD in Adolescents and Young Adults With PCOS
Polycystic Ovary SyndromeNon-Alcoholic Fatty Liver Disease1 moreThis project focuses on an at-risk adolescent and young adult population who may gain long-term health benefits from detection of risk factors at a young age. The primary aims of this proposal are: 1) To observe whether adolescents and young adults with Polycystic Ovary Syndrome (PCOS) are more likely to have elevated liver fat (>/=4.8%) than controls by studying liver fat deposition measured by magnetic resonance spectroscopy (MRS); 2) To assess the association of percentage liver fat with biomarkers of Non-alcoholic fatty liver disease (NAFLD), dyslipidemia, insulin resistance and body composition in PCOS and controls. In the proposed study, 40 adolescents and young adults with PCOS and 40 age-comparable control subjects will be evaluated for metabolic disturbances and elevated liver fat using noninvasive and state-of-the-art techniques including MRI, dual-energy x-ray absorptiometry and an oral glucose tolerance test in order to fully assess the metabolic and body composition differences between these groups. This research proposal represents a critical step in understanding the metabolic and cardiovascular comorbidities of PCOS and their relationship to NAFLD. The investigator hopes to use the results generated by this research proposal in order to lay the groundwork for the prevention and treatment of metabolic disorders in adolescents with PCOS. The overarching goal is to decrease and prevent lifelong morbidity associated with this common disorder.

Everolimus Therapy in People With Birt-Hogg-Dube Syndrome (BHD)-Associated Kidney Cancer or Sporadic...
Renal CancerBirt-Hogg-Dube SyndromeBackground: - Research has shown that the drug everolimus can stop cancer cells from growing. It is approved for people with advanced kidney cancer. Researchers want to see if it also helps people with two other types of kidney cancer. Objective: - To see if everolimus is safe and effective in people with Birt-Hogg-Dube Syndrome (BHD)-associated kidney cancer or sporadic (nonfamilial) chromophobe renal cancer. Eligibility: - People ages 18 and over with BHD-associated kidney cancer or advanced sporadic chromophobe renal cancer. Design: Participants will be screened with: Medical history, physical exam, and blood and urine tests. Computed tomography (CT) scan or magnetic resonance imaging (MRI) scan. They will lie in a machine that takes pictures of their chest/abdomen/pelvis. They may also be screened with: Another scan, of the brain or neck. Bone scan. Positron emission tomography scan with fludeoxyglucose (FDG-PET). Heart and lung tests. Tests for hepatitis. Participants will take a tablet once a day by mouth for up to a year. They will keep a diary of when they take the tablet and any symptoms. During the study, participants will have physical exams and urine and blood tests. They will have scans of the chest/abdomen/pelvis. They may have FDG-PET and bone scans. Participants will have tests for hepatitis and may have a tumor sample taken. Participants will have a follow-up visit 4-5 weeks finishing taking the drug. They will have a physical exam and blood tests. They may have scans and/or hepatitis tests. Participants will be called about every 3-6 months after the study ends to see how they are doing

Evaluation of the Efficacy and Safety of Lubiprostone in Adults With Mixed or Unsubtyped Irritable...
Irritable Bowel SyndromeA study of the efficacy and safety of Lubiprostone in subjects diagnosed with IBS-M/IBS-U.

Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure...
Myelodysplastic SyndromeMDS2 moreThe study's primary objective [in a population of patients with MDS after failure of treatment with azacitidine (AZA) or decitabine (DAC)], is to compare the overall survival (OS) of patients in the rigosertib group vs the Physician's Choice group, in all patients and in a subgroup of patients with IPSS-R very high risk.

Lenalidomide After Allo-Hematopoietic Cell Transplant (HCT) in Acute Myelogenous Leukemia (AML)...
LeukemiaMyeloid1 moreThe purpose of this study is to determine the maximum tolerated dose, dose limiting side effects, and the safety of increasing doses of lenalidomide in patients with AML and MDS who have a small amount of detectable disease after allogeneic stem cell transplant.

Allogeneic hMSC Injection in Patients With Hypoplastic Left Heart Syndrome
Hypoplastic Left Heart SyndromeThis study is intended to evaluate the safety and feasibility of intramyocardial injection of allogeneic mesenchymal cells during the Bi-Directional Cavopulmonary Anastomosis (BDCPA) surgery for hypoplastic left heart syndrome (HLHS) pediatric patients.