Clinical trials: Bone Marrow Failure
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
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
Hematologic Malignancy, Bone Marrow Transplant
Tocilizumab for the Prevention of Graft Failure and GVHD in Haplo-Cord Transplantation
The purpose of this study is to evaluate the safety of reducing and ultimately eliminating anti-thymocyte globulin (ATG) from the haplo-cord transplant conditioning regimen and replacing it with tocilizumab, an IL-6 receptor…
- Trial name
- NCT04395222
- Testing
- Tocilizumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Weill Medical College of Cornell University
Telomere Disease
Low-Dose Danazol for the Treatment of Telomere Related Diseases
Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA.
- Trial name
- NCT03312400
- Testing
- Danazol
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
Primary Objectives: 1. To determine the feasibility and toxicity of employing purine-analog based conditioning for allogeneic donor stem cell transplantation in patients with severe aplastic anemia (AA). 2.
- Trial name
- NCT00427336
- Testing
- Fludarabine, Cyclophosphamide, Antithymocyte Globulin
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- M.D. Anderson Cancer Center
Aplastic Anemia
A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the…
- Trial name
- NCT02094417
- Testing
- AMG531
- Where
- South Korea
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
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
Sars-CoV2, Acute Respiratory Distress Syndrome
Mesenchymal Stromal Cells for the Treatment of SARS-CoV-2 Induced Acute Respiratory Failure (COVID-19 Disease)
*At this time, we are only enrolling at Houston Methodist Hospital (HMH)/Baylor College of Medicine (BCM) and are not shipping cells outside of BCM/HMH.* This is a study for patients who have respiratory infection caused by…
- Conditions
- Sars-CoV2, Acute Respiratory Distress Syndrome, COVID-19
- Trial name
- NCT04345601
- Testing
- Mesenchymal Stromal Cells, Supportive Care
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Baylor College of Medicine
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00755040
- Testing
- cyclosporine ophthalmic emulsion
- Where
- 5 sites in United States
- Volunteers
- 164 people
- Sponsor
- Vanderbilt-Ingram 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)
Fanconi Anemia, Severe Aplastic Anemia
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
The purpose of this study is to discover whether children and adults with Fanconi anemia (FA) can be safely and effectively transplanted with Human Leukocyte Antigen (HLA) mismatched (up to one haplotype), HLA-matched sibling, or…
- Trial name
- Mafia (NCT00590460)
- Testing
- CAMPATH-1H, Anti-CD45, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
Severe Aplastic Anemia, Aplastic Anemia
NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
Our primary objective is to determine if it is feasible for previously untreated severe aplastic anemia (SAA) patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide.
- Conditions
- Severe Aplastic Anemia, Aplastic Anemia, Bone Marrow Failure, Immunosuppression
- Trial name
- NCT02833805
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Sickle Cell Anemia, Severe Aplastic Anemia
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
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.
- Conditions
- Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
- Trial name
- NCT00004143
- Testing
- Campath, Chemo and/or TBI Allo SCT
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- David Rizzieri, 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
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Aplastic Anemia, Menorrhagia
Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones
This study will explore the role of oral contraceptive pills in managing uterine bleeding in women who have low blood platelet counts as a result of aplastic anemia.
- Conditions
- Aplastic Anemia, Menorrhagia, Amenorrhea
- Trial name
- NCT00556400
- Testing
- Lo-Ovral Oral Contraceptive Pills
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
Myelodysplastic Syndromes
Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes
The objectives of this trial are to evaluate the safety, tolerability, maximum tolerated dose (MTD), pharmacokinetics and preliminary efficacy of volasertib in two dosing schedules of intravenous volasertib as monotherapy or in…
- Trial name
- NCT02721875
- Testing
- Volasertib, Azacitidine
- Where
- Japan
- Volunteers
- 1 people
- Sponsor
- Boehringer Ingelheim
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