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Anemia, Aplastic clinical trials

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NCT ID: NCT03246178 Enrolling by invitation - Clinical trials for Severe Aplastic Anemia

Haploidentical Transplantation in Severe Aplastic Anemia

Start date: July 10, 2017
Phase: N/A
Study type: Interventional

This is a prospective case-control study on SAA patients treated with HSCT, order to further discuss and assess the safety, feasibility and effectiveness of HFD-HSCT which performed with reduced-intensity fludarabine-based conditioning regimen.Our findings would indicate that SAA patients who lack MSD benefited most if HFD-HSCT was performed with reduced-intensity fludarabine-based conditioning regimen, and our improved outcomes with HFD-HSCT may lead to a salvaged therapy and an expanded direct role for SAA in the future.

NCT ID: NCT03243656 Completed - Eltrombopag Clinical Trials

Eltrombopag & Cyclosporine in Children With Sever Aplastic Anemia

Eltroplastic
Start date: December 20, 2017
Phase: Phase 4
Study type: Interventional

Aplastic anemia is a rare disorder characterized by pancytopenia and a hypo cellular bone marrow.but,It is very serious disease causing morbidity and mortality. Aplastic anemia can be treated effectively with haematopoietic stem cell transplantation and immunosuppressive drug regimens but haematopoietic stem cell transplantation has limitations due to its cost and many patient are unsuitable. Immunosuppressive drug has a significant number of patients have persistent cytopenias. Currently, the treatment of these patients is regular transfusion, which are expensive, inconvenient, and associated with serious side effects related to iron overload and transfusion. Eltrombopag is an oral thrombopoietin mimetic that selectively binds at the transmembrane and juxtamembrane domains of the thrombopoietin receptor, at sites distinct from the binding site of thrombopoietin therefore it does not compete for binding with the native molecule. It promoting thrombopoiesis and release of platelets from mature megakaryocytes. Also, promote other hematopoietic stem cell as well as in thrombopoiesis .

NCT ID: NCT03218657 Recruiting - Aplastic Anemia Clinical Trials

Clinical Study of Non Severe Aplastic Anemia Treated With Cyclosporine, Androgen and Levamisole

Start date: January 1, 2018
Phase: N/A
Study type: Interventional

The clinical symptoms of non transfusion dependent non severe aplastic anemia (NSAA) are often lighter than that of severe aplastic anemia. Clinical observation is often used and the treatment should be given according to the follow-up results of peripheral blood routine and the survival condition of the patients. In recent years, a number of studies at home or abroad have tended to intervene earlier. The risk of observation and waiting for disease progression is higher. Early immunosuppression should be considered. For the treatment of non transfusion dependent non severe aplastic anemia, the commonly used treatment regimen is androgen combined with CSA. But the investigators find that Levamisole hydrochloride (LMS) as a commonly used immunomodulatory drugs may be helpful to improving immune disorder symptoms in NSAA patients. Therefore,the investigators are conducting a prospective, randomized controlled study to compare the rate, side effects and long-term survival in non transfusion dependent patients with NSAA between the androgen+CSA group and the androgen+CSA+LMS group.

NCT ID: NCT03208647 Completed - Aplastic Anemia Clinical Trials

Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia

MCSAA
Start date: January 1, 2003
Phase: N/A
Study type: Observational

To ascertain the risk factors of AA in ZheJiang Province of China at the moment, especially the comprehensive multiple factors risks. The author conducted a case-control research included 338 AA cases and 1464 controls. Subjects were surveyed using same standard questionnaire including personal data, past histories of diseases, family history and histories of occupational and daily exposure to harmful substances. Single and multiple Logistic regression analyses were made using SPSS17.0 on the dates to study potential factors in the development of AA.

NCT ID: NCT03192397 Active, not recruiting - Clinical trials for Myelodysplastic Syndrome

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

Start date: August 9, 2017
Phase: Phase 1/Phase 2
Study type: Interventional

This phase Ib/2 trial studies how well chemotherapy, total body irradiation, and post-transplant cyclophosphamide work in reducing rates of graft versus host disease in patients with hematologic malignancies undergoing a donor stem cell transplant. Drugs used in the chemotherapy, such as fludarabine phosphate and melphalan hydrochloride, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy and total-body irradiation before a donor stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells (called graft versus host disease). Giving cyclophosphamide after the transplant may stop this from happening.

NCT ID: NCT03176849 Completed - Clinical trials for Myelodysplastic Syndromes

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

Start date: November 1, 2017
Phase: Phase 4
Study type: Interventional

Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as well as overall improved survival. However, supplementation in children undergoing HSCT has shown to be a challenge using standard or aggressive supplementation strategies. The primary objective of this study is to determine the safety and efficacy of a single, high dose oral vitamin D (Stoss Therapy) at the start of transplant followed by maintenance supplementation in children undergoing HSCT.

NCT ID: NCT03173937 Recruiting - Clinical trials for Myelodysplastic Syndrome (MDS)

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Start date: June 13, 2017
Phase: Phase 1/Phase 2
Study type: Interventional

Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective. Objective: To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective. Eligibility: Recipients ages 4-60 with SAA or MDS Donors ages 4-75 Design: Recipients will be screened with: - Blood, lung, and heart tests - Bone marrow biopsy - CT scan Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose. Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits: - First 3-4 months: 1-2 times weekly - Then every 6 months for 5 years<TAB> Donors will be screened with: - Physical exam - Medical history - Blood tests Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein. Donors will get filgrastim injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.

NCT ID: NCT03086252 Withdrawn - Leukemia Clinical Trials

Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study

Start date: July 24, 2017
Phase: N/A
Study type: Interventional

This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.

NCT ID: NCT03055078 Recruiting - Aplastic Anemia Clinical Trials

Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia

Start date: January 2017
Phase: Phase 1
Study type: Interventional

This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.

NCT ID: NCT03047746 Recruiting - Clinical trials for Paroxysmal Nocturnal Hemoglobinuria

Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF

Start date: February 1, 2017
Phase: N/A
Study type: Interventional

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.