Clinical trials: Marrow Aplasia
Severe Aplastic Anemia
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
Background: Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells.
- Trial name
- NCT01623167
- Testing
- Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
- Where
- United States
- Volunteers
- 207 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia
- Trial name
- NCT02566304
- Testing
- Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Severe Aplastic Anemia
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells.
- Trial name
- NCT04304820
- Testing
- Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Myeloproliferative Disorders, Leukemia
Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor stem cell helps stop the growth of cancer or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00003816
- Testing
- anti-thymocyte globulin, busulfan, carboplatin
- Where
- United States
- Volunteers
- 361 people
- Sponsor
- Roswell Park Cancer Institute
Aplastic Anemia
Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects
This was an open label, non-randomized, phase II study of eltrombopag in combination with rabbit ATG/CsA in subjects with moderate or more severe AA who did not received prior ATG/ALG-based immunosuppressive therapy.
- Trial name
- NCT02404025
- Testing
- Eltrombopag, Rabbit ATG, CsA
- Where
- 11 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients
Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients…
- Trial name
- NCT01818726
- Testing
- ICL670, Chelation, No chelation
- Where
- Russia
- Volunteers
- 15 people
- Sponsor
- Novartis Pharmaceuticals
Diamond Blackfan Anemia, Pure Red Cell Aplasia
The Use of Trifluoperazine in Transfusion Dependent DBA
Diamond Blackfan anemia (DBA) is a rare inherited pure red cell aplasia. The two main non-stem cell transplant therapeutic options are corticosteroids and red blood cell (RBC) transfusions.
- Trial name
- DBA (NCT03966053)
- Testing
- Trifluoperazine
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Adrianna Vlachos, MD
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This phase II trial studies how well giving fludarabine phosphate, melphalan, and low-dose total-body irradiation (TBI) followed by donor peripheral blood stem cell transplant (PBSCT) works in treating patients with hematologic…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01529827
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- Roswell Park Cancer Institute
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission
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
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…
- Conditions
- Acute Myeloid Leukemia in Remission, Adult Acute Lymphoblastic Leukemia in Complete Remission, Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission, Chronic Myelomonocytic Leukemia in Remission
- Trial name
- NCT03192397
- Testing
- Allogeneic Hematopoietic Stem Cell Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Roswell Park Cancer Institute
Severe Aplastic Anemia, Very Severe Aplastic Anemia
Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts.
- Conditions
- Severe Aplastic Anemia, Very Severe Aplastic Anemia, Moderate Aplastic Anemia
- Trial name
- NCT01703169
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of Utah
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
Diamond Blackfan Anemia
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.
- Trial name
- ACE-011-DBA (NCT01464164)
- Testing
- Sotatercept, Sotatercept with prednisone boost
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Northwell Health
Fanconi Anemia, Myelodysplastic Syndrome (MDS)
A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia
The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control.
- Conditions
- Fanconi Anemia, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
- Trial name
- NCT03600909
- Testing
- Busulfan, Fludarabine, Cyclophosphamide
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
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
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
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Severe Aplastic Anemia (SAA)
Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Background: Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients.
- Trial name
- NCT01891994
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)