Active clinical trials for "Anemia, Sickle Cell"

This study will assess the safety and efficacy of bosentan therapy (in a study known as ASSET) for patients who have high blood pressure in the lungs associated with sickle cell disease. That form of hypertension places people at risk for complications, including shortness of breath, pain, pneumonia, and death. Previous studies have shown that bosentan can be helpful in reducing pulmonary hypertension. Patients ages 16 and older who have completed the 16-week treatment in the ASSET 1 or ASSET 2 study and who are not pregnant or breastfeeding may be eligible for this study. The research will be conducted in about 25 hospitals in the United States and Europe. Up to 30 participants will be enrolled. The screening visit will involve a physical examination, blood sample of about 3 teaspoons for laboratory tests, and a pregnancy test. Patients' doctors will give them bosentan tablets (62.5 mg each), to take one in the morning and one in the evening. After 1 month, patients will be told whether the dose should be increased to 125 mg tablets to take twice a day. Two weeks after the increase in dose, a blood test will be done to analyze the drug's effects on the liver. After the start of treatment, patients will return for visits every 6 months, when there will be a 6-minute walking test to measure exercise capacity and evaluate shortness of breath. There will be follow-up for patients up to the end of the study and for 28 days after the last dose of bosentan is taken, to collect information about side effects. Some patients on bosentan have had changes in liver function and red blood cell count. Side effects commonly reported are headache, flushed appearance, inflammation of the throat and nasal passages, and gastrointestinal symptoms. If patients have sudden worsening in breathing in the first few weeks after taking bosentan, they should immediately tell their doctors, because it may be necessary to change the treatment.

The purpose of this pilot study is to provide a preliminary assessment of the feasibility and efficacy of intravenous ketamine in controlling pain in patients with sickle cell disease (who are admitted to the hospital with severe, acute pain crisis, and who have been resistant to intravenous narcotics).

The purpose of this program is to evaluate the logistic issues and patient requirements for chronic pulsed INOmax delivery in ambulatory, home-care patients. To understand patient needs, patients with a variety of underlying diseases will be included. Safety of chronic therapy will be monitored by serial measurements of methemoglobin, platelet function assay and reported adverse events.

The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.

This study is designed to address the feasibility of a randomized, double masked, cross-over study of dronabinol as a palliative agent in the treatment of pain, inflammation, and other complications of sickle cell disease (SCD).

Primary Objective Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18 years). Secondary Objective: Compare PK parameters in infants versus older children on this study and those from our previous "Pharmacokinetics and Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle Cell Anemia" (NCT01506544) trial. Exploratory Objectives: Capture information regarding the taste of HU sprinkles using palatability questionnaire. This trial is an open label, single center assessment of the pharmacokinetics of two formulations of hydroxyurea (HU) designed to (1) determine the pharmacokinetic profile of a liquid formulation in infants and to (2) determine the bioavailability of "sprinkles", a novel method of administration for older children. The study aims to generate data to facilitate FDA approval for HU in children and potentially validate a new mode of administration ("sprinkles") that will optimize access and adherence for children in the US and globally.

Benign and malignant hematologic diseases are relatively rare conditions within the spectrum of medical practice in any one site of care. Nonetheless, recent research in hematologic conditions from basic, translational, clinical and population perspectives offer the possibility of improving the way that these diseases are treated, and the outcomes experienced by patients. A repository that aggregates and validates this data across institutions and other practice settings is needed in order to identify variation in care, new findings, and further research.

This study will assess the performances of the Montreal Cognitive Assessment (MoCA) to screen for cognitive impairment in adults with sickle cell anemia. The results of the MoCA and its subscores will be compared to a standardized neuropsychological evaluation using validated tests.

The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.

This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab).