Clinical trials: Bone Marrow Failure Syndrome
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
Steroid-refractory Diamond-Blackfan Anemia (DBA)
Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia
Background: Diamond-Blackfan anemia (DBA) is an inherited disease that affects the bone marrow. People with DBA have chronic anemia that can be severe. Many must have frequent transfusions of red blood cells.
- Trial name
- NCT05828108
- Testing
- Bitopertin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Acute Leukemia, Acute Myeloid Leukemia
UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen with modifications based on factors including diagnosis, disease status, and prior treatment.
- Conditions
- Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia/Lymphoma, Burkitt's Lymphoma
- Trial name
- NCT02722668
- Testing
- Fludarabine, Cyclophosphamide, MMF
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hematologic Malignancies
Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haplo-HCT
This is a single institution phase II study of a reduced intensity conditioning (RIC) followed by a haploidentical hematopoietic cell transplant (haplo-HCT) in persons with diagnosis of hematologic malignancy.
- Trial name
- NCT02988466
- Testing
- Haplo HCT <55 years old, Haplo HCT ≥55 years old, GVHD Prophylaxis
- Where
- United States
- Volunteers
- 78 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
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
Mycoses, Sezary Syndrome
Ph II of Non-myeloablative Allogeneic Transplantation Using TLI & ATG In Patients w/ Cutaneous T Cell Lymphoma
Non-myeloablative approach for allogeneic transplant is a reasonable option, especially given that the median age at diagnosis is 55-60 years and frequently present compromised skin in these patients, which increases the risk of…
- Conditions
- Mycoses, Sezary Syndrome, Lymphoma, T-Cell, Cutaneous, Bone Marrow Transplant Failure
- Trial name
- NCT00896493
- Testing
- anti-thymocyte globulin, cyclosporine, Lymphoid radiation
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Stanford University
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
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, Acute Myeloid Leukemia
T-cell Depleted Alternative Donor Transplantation
The primary purpose is to determine the ability of CD34+ selection and T cell depletion using the CliniMACS® device to prevent severe acute graft-versus-host disease (GVHD) in patients receiving a stem cell transplant from an…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00968864
- Testing
- CliniMACS® (T cell depletion)
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Wake Forest University Health Sciences
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
Myeloid Malignancy, Bone Marrow Failure Syndrome
Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
The purpose of this study is to find out what effects, good and/or bad, the addition of clofarabine, a new chemotherapy agent, to a standard busulfan and fludarabine conditioning treatment has.
- Conditions
- Myeloid Malignancy, Bone Marrow Failure Syndrome, Transfusion-dependent Red Blood Cell (RBC) Defect, Congenital Immunodeficiency
- Trial name
- NCT01596699
- Testing
- Alemtuzumab, Busulfan, Fludarabine
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of California, San Francisco
Acute Leukemias, Burkitt's Lymphoma
Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen.
- Conditions
- Acute Leukemias, Burkitt's Lymphoma, Chronic Myelogenous Leukemia
- Trial name
- NCT02145039
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Neural Tube Defects, Anemia
Post T-plant Infusion of Allogeneic Cytokine Induced Killer (CIK) Cells as Consolidative Therapy in Myelodysplastic Syndromes/Myeloproliferative Disorders
Allogeneic stem cell transplantation (transplant of blood cells from another individual) is a treatment option for patients with myelodysplasia or myeloproliferative Disorders.
- Conditions
- Neural Tube Defects, Anemia, Leukemia, Myeloid, Bone Marrow Transplant Failure
- Trial name
- NCT01392989
- Testing
- CIK cells, Cyclosporine, Mycophenolate Mofetil
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Everett Meyer
Bone Marrow Failure Syndromes, Immunodeficiencies
Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases
This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors.
- Conditions
- Bone Marrow Failure Syndromes, Immunodeficiencies, Immune Dysregulation Syndromes
- Trial name
- MiniClini (NCT02277639)
- Testing
- CliniMACs device
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Children's Hospital of Philadelphia
Diamond-Blackfan Anemia, Kostmann's Neutropenia
Stem Cell Transplant for Bone Marrow Failure Syndromes
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for…
- Conditions
- Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
- Trial name
- NCT00176878
- Testing
- Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota