
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Fanconi AnemiaDyskeratosis CongenitaFanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects, indicating a need for a different medication. Danazol is a less potent androgen,and may therefore have fewer masculinizing side effects. Danazol is currently approved by the Food and Drug Administration (FDA) for the treatment of other diseases, but it has never been studied in patients with FA and DC. The main purpose of this study is to see if danazol is a safe treatment for FA and DC. Specifically,we would like to determine: the best dose of danazol; how fast hemoglobin (a protein that carries oxygen in the blood) levels rise in FA and DC patients receiving danazol therapy; and the genetic pattern (known as expression profile) of certain cells in response to danazol, which can predict how well people respond to the medication. Subjects who enroll in the study will be treated with danazol for up to 24 weeks (about 6 months), and will have up to 11 study visits, including followup visits at 38 weeks (9 months) and 52 weeks (one year).

A Study of the Efficacy and Safety of MK2578 for the Treatment of Anemia in Patients With Kidney...
AnemiaChronic Kidney DiseaseThis study will evaluate the efficacy and safety of intravenous MK2578, given as maintenance treatment for renal anemia in chronic kidney disease patients on dialysis who were previously receiving erythropoietin stimulating agents.

Dynepo Infrequent Dosing Study
AnemiaKidney FailureThe purpose of this study is to demonstrate non-inferiority of efficacy between twice weekly and once weekly dose schedule of Dynepo in previously erythropoietin (EPO)-naive patients, as measured by haemoglobin at week 24 and secondly to demonstrate the non-inferiority of efficacy between once weekly and once every two weeks dose schedules of Dynepo in patients previously stable on EPO, as measured by Hb over Weeks 16 to 24.

Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia
Aplastic AnemiaPatients have been diagnosed with severe Aplastic Anemia that have not responded to treatment with immunosuppressive therapy (drugs that suppress the immune system, for example Steroids). The immune system is the system in the body that helps protect the body and fights bacterial, viral and fungal infections. Research studies have shown that patients with Aplastic Anemia have improved survival (may live longer) after receiving a HLA (Human Leukocyte Antigen) identical sibling (brother and sister) stem cell transplants. Patients who do not have matched siblings can undergo immunosuppressive therapy, which has also shown to improve outcome. Unfortunately patients who do not respond to immunosuppressive therapy usually die. The best chance of survival for these patients is an HLA matched unrelated or mismatched related stem cell transplant as described below. Stem cells are created in the bone marrow. They mature into different types of blood cells that people need including red blood cells which carry oxygen around the body, white blood cells which help fight infections, and platelets which help the blood to clot and prevent bleeding. For a matched unrelated stem cell transplant, stem cells are collected from a person (donor) who is not related to the patient but who has the same type of stem cells. For a mismatched related stem cell transplant, stem cells are collected from a donor who is related to the patient and whose stem cells are almost the same as those of the patient but not exactly. The patient then receives high dose chemotherapy. This chemotherapy kills the stem cells in the patient's bone marrow. Stem cells that have been collected from the donor are then given to the patient to replace the stem cells that have been killed. The major problems associated with these types of stem cell transplants are graft rejection (where the patient's immune system rejects the donor stem cells) and severe graft versus host disease (GVHD), where the donors stem cell reacts against the patient's tissues in the body.

Idiopathic Anemia of Aging (IAA)
AnemiaAgingThe purpose of this trial is to examine whether Epoetin Alfa, a hormone stimulating production of red blood cells, can reverse idiopathic anemia.

Post-marketing Surveillance of EVRENZO® Tablets (Roxadustat) in Patients With Renal Anemia
Renal AnemiaThe purpose of this study is to assess the safety and efficacy, including the incidence of thromboembolism, in renal anemia patients treated with roxadustat (EVRENZO® Tablets) in actual clinical settings.

Hemoglobin Optimization to Prevent Transfusion and Adverse Events in Perioperative Patients With...
AnemiaIron Deficiency3 moreThe HOPE-Hb trial is a phase II study to determine the feasibility and impact of a combination treatment (intravenous iron plus erythropoietin) versus intravenous iron treatment alone on preoperative hemoglobin concentration before hip or knee arthroplasty.

FGF23 and Cardiovascular Damage in Anemia With an Without Chronic Kidney Disease.
Fibroblast Growth Factor 23Anemia1 moreAnemia is associated with cardiovascular disease. Iron deficiency is usually induced in chronic kidney disease (CKD). In clinical studies, an inverse association between serum levels of iron and fibroblast growth factor 23 (FGF23), a cardiovascular risk factor, has been demonstrated. In addition, a number of the I.V. iron presentations mostly used to treat anemia show unwanted side effects related to phosphate alterations and increased FGF23. Objectives. The General Objective of this project is to evaluate, through in vivo and in vitro studies, the cardiovascular alterations related to the anemia-induced increase in FGF23 production; as well as the identification of possible molecular targets that may be useful in its prevention and/or palliation. Specific Objectives are: 1) To determine in a population with anemia (due to iron deficiency), with and without CKD, an association between the parameters related to iron metabolism, FGF23 and markers of cardiovascular damage. 2) To evaluate in vivo, in a murine experimental model of anemia, with and without CKD, the effects of the modulation (inhibition) of triggers of iron deficiency (hepcidin) and of the increase in FGF23 (HF1α), on markers of cardiovascular damage. 3) To compare in vivo, in an experimental model of anemia with and without CKD, the effect of different I.V. iron presentations (ferrous sulphate, ferric carboxymaltose and ferric citrate) on FGF23 levels and their cardiovascular impact. 4) To evaluate in vitro, in cardiomyocytes cultures, in the presence of iron deficiency, the direct effect of FGF23 on the induction of cardiac damage. 5) To evaluate in vitro, in osteoblasts cultures, the direct effect of ferrous sulphate, ferric carboxymaltose, ferric citrate and hepcidin. Methodology. The levels of intact and C-terminal FGF23 (FGF23i and FGF23c), the differential expression profile of plasma miRNAS and of proteomic, markers of cardiovascular disease, mineral metabolism, inflammation and oxidative stress and intracellular signalling pathways will be evaluated.

Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion...
AnemiaMyelodysplastic SyndromeThis phase II trial studies how well deferasirox works in treating patients with very low, low, or intermediate-risk anemia or myelodysplastic syndrome that depends on red blood cell transfusions. Deferasirox may treat too much iron in the blood caused by blood transfusions.

Ferrous Sulphate Supplement (V0355) in Women With Iron Deficiency Anaemia
Iron Deficiency AnemiaThe purpose of this study is to investigate the pharmacokinetics of serum iron after a single oral administration of 160 mg (2 tablets of 80 mg) V0355 in women with iron deficiency anaemia.