Clinical trials: Aplastic Anemia
Dyskeratosis Congenita, Aplastic Anemia
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who…
- Trial name
- NCT02162420
- Testing
- Alemtuzumab, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Dyskeratosis Congenita, Aplastic Anemia
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant…
- Trial name
- NCT00455312
- Testing
- Campath 1H, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Anemia, Chronic Kidney Disease
Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease
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…
- Conditions
- Anemia, Chronic Kidney Disease, Chronic Renal Failure, Pure Red Cell Aplasia
- Trial name
- NCT00314795
- Testing
- Peginesatide
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 22 people
- Sponsor
- Takeda
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA).
- Conditions
- Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
- Trial name
- NCT00244010
- Testing
- Allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Fanconi Anemia
Stem Cell Transplantation for Fanconi Anemia
The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched…
- Trial name
- NCT00167206
- Testing
- Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Moderate Aplastic Anemia, Unilineage Bone Marrow Failure Disorders
Eltrombopag for Moderate Aplastic Anemia
Background: Moderate aplastic anemia is a blood disease which may require frequent blood and platelet transfusions. Sometimes patients with this disease can be treated with immunosuppressive drugs.
- Trial name
- NCT01328587
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Leukemia
Alemtuzumab in Myelodysplastic Syndrome (MDS), Aplastic Anemia, and T-Cell Large Granular Lymphocytic Leukemia (T-GL)
The goal of this clinical research study is to determine the effectiveness of alemtuzumab in patients with aplastic anemia, MDS, or T-Cell large granular lymphocytic leukemia. The safety of alemtuzumab will also be studied.
- Trial name
- NCT01191749
- Testing
- Alemtuzumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- M.D. Anderson Cancer Center
Anemia, Diamond-Blackfan
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work.
- Trial name
- NCT04269889
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia
Patients 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).
- Trial name
- NCT00578903
- Testing
- Cytoxan, Campath, Total Body Irradiation (TBI)
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Baylor College of Medicine
Anemia, Aplastic
Fludarabine-based Conditioning for Severe Aplastic Anemia (BMT CTN 0301)
The purpose of the current study is to continue to optimize conditioning regimens in high-risk patients with severe aplastic anemia transplanted with marrow from HLA-compatible unrelated donors.
- Trial name
- NCT00326417
- Testing
- Fludarabine, Cyclophosphamide 150mg, Cyclophosphamide 100mg
- Where
- 22 sites in United States
- Volunteers
- 97 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease, Thalassemia
Stem Cell Transplant for Hemoglobinopathy
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia
- Trial name
- NCT00176852
- Testing
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia, Bone Marrow Failure Syndromes
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Our primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.
- Trial name
- NCT02224872
- Testing
- Bone marrow transplant, Thymoglobulin, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Leukemia, Lymphoma
Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis
The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD).
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndrome, Myelofibrosis
- Trial name
- NCT02065154
- Testing
- Cyclophosphamide
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- University of Alabama at Birmingham
Aplastic Anemia
Danazol for Genetic Bone Marrow and Lung Disorders
Background: Some people have bone marrow and lung disorders that are caused by genetic problems. These problems often involve damage to the ends of the chromosomes that pass down genes. One of these disorders is aplastic anemia.
- Trial name
- NCT01441037
- Testing
- Danazol
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia
Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA).
- Trial name
- NCT00065260
- Testing
- Campath-1H, r-ATG, CsA
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)