Einstein Montefiore

Background: - People with sickle cell disease and other blood disorders sometimes get chronic leg ulcers. These are wounds that develop on the skin and don t go away. Current treatments do not work very well, so researchers want to learn more about why the ulcers happen. They want to find out which bacteria may cause it, and if external factors play a role. Objective: - To study social and environmental factors of sickle cell disease and the causes of sickle cell disease leg ulcers. Eligibility: - People age 18 and older who have sickle cell disease or another red cell disorder, with or without an active leg ulcer. Design: - Participants will have a medical history and clinical evaluation. They will also have blood drawn. - Participants will complete questionnaires about their life, health, environment, stress, and other topics. - Participants may provide a small sample of hair. - Participants will be asked to collect a small amount of saliva. - Participants with leg ulcers will have their skin microbiome sampled. The microbiome is all of the microbes (bacteria and and/or fungi) and their genes in and on the body. Researchers will use swabs to collect skin samples. Photographs will be taken of the skin sample area. - Some participants without leg ulcers also will have their skin microbiome sampled. - Some participants who have their skin microbiome sampled will return for a second visit. At this visit, their microbiome will be resampled. It will take place more than 30 days after the first visit.

Type: Observational

Start Date: Jun 2014

open study

This three-cohort, multi-stage, randomized, Phase II, multicenter trial will evaluate the safety and tolerability and estimate the efficacy of cobimetinib plus paclitaxel versus placebo plus paclitaxel in Cohort I, of cobimetinib plus atezolizumab plus paclitaxel in Cohort II, and of cobimetinib plus atezolizumab plus nab-paclitaxel in Cohort III in participants with metastatic or locally advanced, triple-negative adenocarcinoma of the breast who have not received prior systemic therapy for metastatic breast cancer (MBC). Participants may continue on study treatment until the development of progressive disease (PD) or the loss of clinical benefit, unacceptable toxicity, and/or consent withdrawal. The Cohort I target sample size is 12 participants for the safety run-in stage and approximately 90 participants in the expansion stage. Each of Cohorts II and III will consist of a safety run-in stage of approximately 15 participants followed by an expansion stage of approximately 15 participants.

Type: Interventional

Start Date: Nov 2014

open study

This randomized phase III trial compares topical or ablative treatment with active monitoring in preventing anal cancer in patients with human immunodeficiency virus (HIV) and high-grade squamous intraepithelial lesions (HSIL). Anal HSIL is tissue in the anal canal that has been damaged by infection with human papillomavirus (HPV) and is at risk for turning into anal cancer. It is not yet known if treating HSIL is more effective than active monitoring in preventing patients from developing anal cancer.

Type: Interventional

Start Date: Sep 2014

open study

Childhood chronic vasculitis describes a group of rare life-threatening diseases that have in common inflammation of blood vessels in vital organs such as kidneys, lungs and brain. Most knowledge about them comes from adult patients. Severe disease requires aggressive life-saving treatments with steroids and some cancer drugs which can themselves cause damage, and increase risks of cancer and severe infections. Conversely, milder disease can be treated with less toxic drugs. Different classification and "scoring tools" are used to define the types and severity of vasculitis and to measure damage caused by disease or drugs. These in turn help direct how aggressively to treat a patient and to measure outcome. None of these tools however have been assessed in children and the best balance of disease and treatment risks against outcome for children is not known. Although causes of these diseases in children and adults are probably the same, the effects of the disease and the response (good and bad) to drugs will differ in growing children. Because specialists may see only one new child with vasculitis each year, obtaining enough information to learn about childhood vasculitis requires cooperation. We will use an international web-based registry to which doctors from 50 or more centers can contribute patient data. We will determine the features which help better classify and diagnose children compared to adults. Through the web we will collect and analyze information on patients similarly classified and "scored" so that most successful treatments can be identified. Children with vasculitis are less likely to have diseases associated with aging, alcohol and smoking etc., and therefore may be a better group in whom to study the underlying biology of vasculitis. We will use this opportunity and collect spit, blood and tissue from registry patients for laboratory study with an aim to find biomarkers to better classify, define and direct optimal treatment and outcomes.

Type: Observational [Patient Registry]

Start Date: Jan 2013

open study

The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)

Type: Interventional

Start Date: Nov 2008

open study

The investigators propose a phase 2 randomized double blind 8-week treatment trial of intranasal OXT vs. placebo in 50 subjects aged 5 to 17 years with PWS in order to assess IN-OXT's affect on measurements of (1) eating behaviors (2) repetitive behaviors (3) weight and body composition (4) quality of life (5) salivary OXT and hormone levels (including ghrelin, pancreatic polypeptide, peptide YY, GLP-1, insulin, glucagon, testosterone, and estrogen). If superior to placebo, this data will add to the current knowledge that OXT is an effective treatment for hyperphagia as well as other symptoms of PWS.

Type: Interventional

Start Date: Apr 2018

open study

This study is a prospective, multicenter, randomized (2:1) controlled study to evaluate the safety and effectiveness of the LARIAT System to percutaneously isolate and ligate the Left Atrial Appendage from the left atrium as an adjunct to planned pulmonary vein isolation (PVI) catheter ablation in the treatment of subjects with symptomatic persistent or longstanding persistent atrial fibrillation. This study will be conducted in two stages: - Limited Early Stage (Stage 1): up to 250 subjects at up to 65 sites. (COMPLETED, transitioned to Stage 2) - Pivotal Stage/ Phase III (Stage 2): up to 600 subjects at up to 65 sites. (ENROLLING) All subjects from both stages will be included in the primary analysis.

Type: Interventional

Start Date: Sep 2015

open study

This phase I trial studies the side effects and best dose of nivolumab when given with ipilimumab in treating patients with human immunodeficiency virus (HIV) associated classical Hodgkin lymphoma that has returned after a period of improvement or does not respond to treatment, or solid tumors that have spread to other places in the body or cannot be removed by surgery. Monoclonal antibodies, such as ipilimumab and nivolumab, may block tumor growth in different ways by targeting certain cells. Ipilimumab is an antibody that acts against a molecule called cytotoxic T-lymphocyte antigen 4 (CTLA-4). CTLA-4 controls a part of your immune system by shutting it down. Nivolumab is a type of antibody that is specific for human programmed cell death 1 (PD-1), a protein that is responsible for destruction of immune cells. Giving ipilimumab with nivolumab may work better in treating patients with HIV associated classical Hodgkin lymphoma or solid tumors.

Type: Interventional

Start Date: Aug 2015

open study

The goal of this study is to understand how activating control centers of the brain with diazoxide can affect how much glucose (sugar) is produced by the liver. This is particularly important for people with diabetes who have very high production of glucose, since this could be at least in part due to impaired regulation of the liver by the brain.

Type: Interventional

Start Date: Jun 2018

open study

This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate AVP-786 for the treatment of Intermittent Explosive Disorder (IED).

Type: Interventional

Start Date: Jan 2018

open study

This randomized phase II clinical trial studies whether the addition of nivolumab to cisplatin (or carboplatin) and etoposide work will improve outcomes when treating patients with extensive stage small cell lung cancer. Drugs used in chemotherapy such as cisplatin, carboplatin, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as nivolumab, may interfere with the ability of tumor cells to grow and spread. Giving cisplatin/carboplatin and etoposide together with nivolumab may work better in treating patients with extensive stage small cell lung cancer.

Type: Interventional

Start Date: May 2018

open study

The purpose of this phase 2 study is to assess the safety and efficacy of oral ifetroban for the treatment of aspirin-exacerbated respiratory disease (AERD). AERD is a disease that involves asthma, recurring nasal polyps, and respiratory reactions to aspirin and other nonsteroidal anti-inflammatory drugs.

Type: Interventional

Start Date: Jul 2017

open study

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Type: Observational

Start Date: Oct 2011

open study

This partially randomized phase II trial studies how well nivolumab, cabozantinib s-malate, and ipilimumab work in treating patients with stage IV non-small cell lung cancer that has come back. Monoclonal antibodies, such as nivolumab and ipilimumab, may interfere with the ability of tumor cells to grow and spread. Cabozantinib s-malate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving nivolumab, cabozantinib s-malate, and ipilimumab may work better in treating patient with stage IV non-small cell lung cancer.

Type: Interventional

Start Date: Mar 2018

open study

This project seeks to develop and pilot test the pediatric ASTHMA-Educator mobile application.

Type: Interventional

Start Date: Jul 2016

open study

The goal of this study is to find the highest tolerated dose of NC-6300 that can be given to patients with advanced solid tumors or soft tissue sarcoma. The safety and tolerability of the drug will also be studied.

Type: Interventional

Start Date: Jun 2017

open study

Phase 2 Study Comparing CB-839 in Combination with Everolimus (CBE) vs. Placebo with Everolimus (PboE) in Patients with Advanced or Metastatic RCC

Type: Interventional

Start Date: Jul 2017

open study

Phase 3, placebo controlled, double-blind, randomized clinical study to determine safety, tolerability, and efficacy of pulsed, inhaled nitric oxide (iNO) versus placebo in symptomatic subjects with pulmonary arterial hypertension (PAH). Part 1 and Part 2

Type: Interventional

Start Date: Apr 2016

open study

This non-interventional, prospective study will characterize the impact of three approved first and second generation BCR-ABL1 tyrosine kinase inhibitors on cardiovascular and metabolic risk factors in chronic phase CML (CP-CML) patients who are TKI naive and initiating first-line TKIs in routine clinical practice in the US. All treatment decisions will be determined at the discretion of the treating physician(s) and data identifying the cardiovascular and metabolic risk factors will be collected. Additional fasting blood samples (collected following 8 hours of fasting) will be collected during standard of care (SOC)/routine office visits. Additional research imaging will be performed and will be reviewed by core imaging laboratory. As the study is collecting data on management of CML, this study will not influence the prescribing or management practices at participating sites.

Type: Observational

Start Date: Mar 2017

open study

The proposed study is a Phase 2 multi-center, randomized, double-blind, placebo-controlled, parallel groups study aimed to evaluate the safety, tolerability and the efficacy of riociguat compared with placebo in patients with sickle cell disease (SCD).

Type: Interventional

Start Date: Apr 2017

open study

The primary objectives of this study are to evaluate the effect of Obeticholic Acid treatment compared to placebo on 1) histological improvement and 2) liver-related clinical outcomes in patients with non-cirrhotic nonalcoholic steatohepatitis (NASH) with liver fibrosis.

Type: Interventional

Start Date: Sep 2015

open study

This will be a single arm, window of opportunity study in men with treatment-naïve prostate cancer who will proceed to prostatectomy. Men will be treated with carvedilol prior to undergoing prostatectomy.

Type: Interventional

Start Date: Apr 2017

open study

This will be an open-label, randomized, multicenter, efficacy and safety study of weekly MOD-4023 compared to daily Genotropin therapy in pre-pubertal children with growth hormone deficiency.

Type: Interventional

Start Date: Dec 2016

open study

This study compares the efficacy of five oral analgesics: 5 mg oxycodone + 325 mg acetaminophen, 5 mg hydrocodone + 300 mg acetaminophen, 30 mg codeine + 300 mg acetaminophen, 400 mg ibuprofen + 1000 mg acetaminophen, and 800 mg ibuprofen + 1000 mg acetaminophen for the treatment of patients with acute musculoskeletal pain who present to the Emergency Department (ED).

Type: Interventional

Start Date: Nov 2017

open study

This is a randomized study, based in emergency departments, in which the investigators determine the utility of adding various skeletal muscle relaxants to standard therapy, which consists of ibuprofen and an educational session. Patients will be enrolled at the time of an emergency visit and followed for three months to determine outcomes.

Type: Interventional

Start Date: May 2017

open study