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Pure Red Cell Aplasia clinical trials

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NCT ID: NCT03364764 Completed - Clinical trials for Pure Red Cell Aplasia

Sirolimus Treatment for Refractory PRCA

Start date: February 1, 2018
Phase: Phase 4
Study type: Interventional

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of sirolimus for refractory PRCA.

NCT ID: NCT01362595 Completed - Clinical trials for Diamond Blackfan Anemia

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

LeucineDBA
Start date: June 2013
Phase: Phase 1/Phase 2
Study type: Interventional

This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions. The leucine is expected to produce a response in patients with DBA to the point where red blood cell production is increased. Red cell transfusions can then be less frequent or possibly discontinued. The investigators will study the side effects, if any, of giving leucine to DBA patients. Leucine levels of leucine will be obtained at baseline and during the study. The drug leucine will be provided in capsule form and taken 3 times a day for a total of 9 months.

NCT ID: NCT00391287 Completed - Clinical trials for Chronic Kidney Failure

Surveillance Study to Estimate the Incidence of Pure Red Blood Cell Aplasia Among Patients With Chronic Kidney Failure

PRIMS
Start date: June 2006
Phase: Phase 4
Study type: Observational

The purpose of this study is to estimate the incidence rate of pure red cell aplasia (PRCA; aplastic anemia) mediated by erythropoietin (EPO) antibodies in patients who are receiving subcutaneous (s.c.) epoetin alfa (polysorbate 80 formulation) for the treatment of anemia associated with chronic renal failure (CRF), and to compare this incidence rate to the incidence rate with s.c. exposure to other currently marketed recombinant erythropoietin products (epoetin alfa, epoetin beta, darbepoetin alfa), with adjustment of duration for which the drug is given to the patient. The study will also examine the impact of the pattern of using mixed s.c. exposure to multiple erythropoietin products occurring in this patients, and the impact of the time from which the treatment is started to the onset of PRCA.

NCT ID: NCT00314795 Completed - Anemia Clinical Trials

Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease

Start date: April 6, 2006
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the ability of peginesatide (AF37702) to increase and maintain increased hemoglobin levels in participants with chronic kidney disease (CKD) (either not on dialysis, receiving regular hemodialysis or peritoneal dialysis, or following renal transplant) with confirmed antibody-mediated pure red cell aplasia (PRCA).

NCT ID: NCT00211068 Completed - Clinical trials for Pure Red-cell Aplasia

A Study of Risk Factors for Anti-erythropoietin Antibody Positive Pure Red Cell Aplasia Among Patients With Chronic Kidney Disease Receiving Epoetin Alfa

Start date: March 2004
Phase: Phase 4
Study type: Observational

The purpose of this study is to collect historical occurrences of risk factors that are potentially associated with the development of anti-erythropoietin (EPO) antibody positive pure red cell aplasia (PRCA) in participants with chronic kidney disease who have been recently treated with epoetin alfa (EPREX).

NCT ID: NCT00211042 Completed - Clinical trials for Pure Red-cell Aplasia

A Study of Patients With Pure Red Cell Aplasia Associated With Recombinant Human Erythropoietin Treatment

Start date: February 2004
Phase: Phase 4
Study type: Observational

The purpose of this study is to investigate the relationship of anti-erythropoietin antibodies to the clinical course and outcome of pure red cell aplasia (PRCA) in participants currently or previously treated with recombinant human erythropoietin.

NCT ID: NCT00004378 Completed - Thrombocytopenia Clinical Trials

Stem Cell Transplantation (SCT) for Genetic Diseases

Start date: January 1995
Phase: N/A
Study type: Interventional

OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.

NCT ID: NCT00004143 Completed - Sickle Cell Anemia Clinical Trials

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Start date: September 1999
Phase: Phase 2
Study type: Interventional

RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood. Unfortunately, the conventional approach to allogeneic stem cell transplantation is a risky procedure. For some non-cancerous conditions, the risks of this procedure outweigh the potential benefits. This protocol is designed to test a new approach to allogeneic stem cell transplantation. It is hoped that this approach will be better suited for patients with non-cancerous blood and bone marrow disorders.