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NCT ID: NCT01623167 Recruiting - Clinical trials for Severe Aplastic Anemia

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

Start date: July 2, 2012
Phase: Phase 1/Phase 2
Study type: Interventional

Background: - Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment. - Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts. Objectives: - To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia. Eligibility: - Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated. Design: - Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected. - Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease. - Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months. - Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months. - Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months. - Participants may receive other medications to prevent infections during treatment. - Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.

NCT ID: NCT01589874 Recruiting - Clinical trials for Anemia of Acute Inflammation

Anemia of Inflammation: Investigation on Impaired Iron Regulation in Acutely Ill Patients and Their Clinical Outcome

Start date: January 2008
Phase: N/A
Study type: Observational

Anemia of inflammation (AI), a normochromic, normocytic anemia, associated with abnormal iron utilization, erythropoietin hyporesponsiveness, and decreased red blood cells (RBC) survival is a very common problem in hospitalized patients for acute inflammatory diseases and develops within few days from the onset of illness. Deleterious effects of anemia include increased risk of cardiac related morbidity and mortality in critically ill and older patients. Anemia is mediated by hepcidin, the 25-amino acid polypeptide hormone that is central to iron trafficking. Several studies have been performed on pathophysiology of AI in patients affected by chronic diseases but few explore iron metabolism in humans with acute inflammation. The aim is to develop effective new strategies to detect and manage AI in the setting of acutely ill patients, based on the understanding of iron balance underlying this disorder.

NCT ID: NCT01558076 Recruiting - Sickle Cell Anemia Clinical Trials

Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia

Start date: January 2012
Phase: N/A
Study type: Observational

The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.

NCT ID: NCT01459146 Recruiting - Anemia Clinical Trials

Artemisinin-based Combination Therapy-Intermittent Preventive Treatment (ACT-IPT) Trial Among Schoolchildren in Kassena-Nankana, Ghana

ACTIPT
Start date: December 2010
Phase: Phase 4
Study type: Interventional

The purpose of this study is to determine if Artemisinin-based Combination Therapy, ACT,(artemether-lumefantrine) used as intermittent preventive treatment (IPT) alone or in combination with praziquantel, will have any effects on anemia, malaria, schistosomiasis and school sustained attention and concentration.

NCT ID: NCT01418898 Recruiting - Clinical trials for Iron Deficiency Anemia

Nutrient Fortified Oat Drink

Start date: August 2011
Phase: Phase 4
Study type: Interventional

This study will follow a randomized, double-blind, placebo-controlled, parallel-group design to test if an oat beverage fortified with iron, zinc vitamin A and vitamin C will improve nutrient status and iron deficiency in young children compared to an unfortified control beverage.

NCT ID: NCT01310959 Recruiting - Clinical trials for Aplastic Anemia and Other Marrow Failure Syndrome

Establishing a Data Base for Aplastic Anemia and Other Marrow Failure Syndrome

Start date: January 2003
Phase: N/A
Study type: Observational

Establishing a Data Base for Aplastic Anemia and Other Marrow Failure Syndrome

NCT ID: NCT01305694 Recruiting - Aplastic Anemia Clinical Trials

Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia.

MSC
Start date: February 2011
Phase: Phase 1/Phase 2
Study type: Interventional

The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells from related donor to patients with relapsed/refractory aplastic anemia.

NCT ID: NCT01182662 Recruiting - Aplastic Anemia Clinical Trials

Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia

Start date: August 2010
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for the therapy of severe aplastic anemia (SAA).

NCT ID: NCT01174108 Recruiting - Clinical trials for Severe Aplastic Anemia

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

Start date: December 10, 2010
Phase: Phase 2
Study type: Interventional

Background: - Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications. - By reducing the number of white blood cells included in the blood taken during the stem cell collection process, and replacing them with a smaller amount of white blood cells collected prior to stem cell donation, the stem cell transplant may be less likely to cause severe complications for the recipient. Researchers are investigating whether altering the stem cell transplant donation procedure in this manner will improve the likelihood of a successful stem cell transplant with fewer complications. Objectives: - To evaluate a new method of stem cell transplantation that may reduce the possibly of severe side effects or transplant rejection in the recipient. Eligibility: - Recipient: Individuals between 4 and 80 years of age who have been diagnosed with a blood disease that can be treated with allogenic stem cell transplants. - Donor: Individuals between 4 and 80 years of age who are related to the recipient and are eligible to donate blood. OR unrelated donors found through the National Marrow Donor Program. Design: - All participants will be screened with a physical examination and medical history. - DONORS: - Donors will undergo an initial apheresis procedure to donate white blood cells. - After the initial donation, donors will receive injections of filgrastim to release bone marrow cells into the blood. - After 5 days of filgrastim injections, donors will have apheresis again to donate stem cells that are present in the blood. - RECIPIENTS: - Recipients will provide an initial donation of white blood cells to be used for research purposes only. - From 7 days before the stem cell transplant, participants will be admitted to the inpatient unit of the National Institutes of Health Clinical Center and will receive regular doses of cyclophosphamide, fludarabine, and anti-thymocyte globulin to suppress their immune system and prepare for the transplant. - After the initial chemotherapy, participants will receive the donated white blood cells and stem cells as a single infusion. - After the stem cell and white blood cell transplant, participants will have regular doses of cyclosporine and methotrexate to prevent rejection of the donor cells. Participants will have three doses of methotrexate within the week after the transplant, but will continue to take cyclosporine for up to 4 months after the transplant. - Participants will remain in inpatient care for up to 1 month after the transplant, and will be followed with regular visits for up to 3 years with periodic visits thereafter to evaluate the success of the transplant and any side effects.

NCT ID: NCT01146210 Recruiting - Clinical trials for Chronic Myelomonocytic Leukemia

Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia

Start date: October 2009
Phase: N/A
Study type: Observational

This research study is studying identification of de novo Fanconi anemia in younger patients with newly diagnosed acute myeloid leukemia. Studying samples of tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to Fanconi anemia in patients with acute myeloid leukemia.