Clinical trials: Pancytopenia
Immunodeficiency Syndrome, Severe Aplastic Anemia
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus…
- Conditions
- Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
- Trial name
- NCT00358657
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Fred Hutchinson Cancer Center
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)
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
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
Blood Stem Cell Transplant Failure, Leukemia
Mismatched Transplantation Using High-dose Post-transplant Cyclophosphamide
The goal of this clinical research study is to learn about the safety of giving a stem cell transplant from a tissue-mismatched donor, followed by cyclophosphamide, to patients with certain types of blood disorders or blood…
- Conditions
- Blood Stem Cell Transplant Failure, Leukemia, Hematologic Malignancies
- Trial name
- NCT01010217
- Testing
- Cyclophosphamide, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- M.D. Anderson Cancer Center
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
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
Severe Aplastic Anemia
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
- Trial name
- SOAR (NCT02998645)
- Testing
- eltrombopag, Cyclosporine
- Where
- 20 sites in Brazil, Hong Kong and 7 more
- Volunteers
- 54 people
- Sponsor
- Novartis Pharmaceuticals
Severe Aplastic Anemia
Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)
This study is a prospective, multicenter phase II study with patients receiving haploidentical transplantation for Severe Aplastic Anemia (SAA).
- Trial name
- CHAMP (NCT02918292)
- Testing
- Antithymocyte Globulin (ATG), Fludarabine, Cyclophosphamide
- Where
- 28 sites in United States
- Volunteers
- 32 people
- Sponsor
- Medical College of Wisconsin
Hematologic Malignancy, Myelodysplastic Syndrome (MDS)
Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies
Subjects will be diagnosed with a hematological malignancy (cancer of the blood), which is unlikely to be cured with conventional non-transplant therapy.
- Conditions
- Hematologic Malignancy, Myelodysplastic Syndrome (MDS), Aplastic Anemia
- Trial name
- NCT00673114
- Testing
- haplo/cord transplant
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Joanne Kurtzberg, MD
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
Hematologic Malignancies
G-CSF PMRD: Granulocyte Colony Stimulating Factor (G-CSF) Stimulated Bone Marrow and In Vivo T-Cell Depletion in Patients With Hematologic Malignancies or Bone Marrow Failure Syndrome
The purposes of this study are: To examine the engraftment rate in patients receiving in vivo T-cell-depleted G-CSF stimulated bone marrow from partially mismatched related donors.
- Trial name
- G-CSF PMRD (NCT00228813)
- Testing
- Granulocyte Colony Stimulating Factor
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Emory University
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
Fanconi Anemia
Pilot Study of Metformin for Patients With Fanconi Anemia
This is a single institution, open-label, single arm pilot study of Metformin in patients with Fanconi Anemia (FA) and cytopenias with the primary endpoint of hematologic response.
- Trial name
- NCT03398824
- Testing
- metformin HCl
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
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
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia, Relapse
Alemtuzumab to Treat Severe Aplastic Anemia
This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath ), in patients with severe aplastic anemia (SAA).
- Trial name
- NCT00195624
- Testing
- Alemtuzumab (Campath ), Cyclosporine
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)