Clinical trials: sponsored by National Heart, Lung, and Blood Institute (NHLBI)
- Lead sponsor (exact) National Heart, Lung, and Blood Institute (NHLBI)
- Age group Children (under 18)
- Has posted results yes
Severe Aplastic Anemia
Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells.
- Trial name
- NCT02979873
- Testing
- Sirolimus
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Congenital Hemolytic Anemia, Sickle Cell Disease
Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have been cured with bone marrow transplantation (BMT).
- Trial name
- NCT00061568
- Testing
- Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function
Background: Sickle cell disease (SCD) is a genetic disorder where red blood cells, that carry oxygen, are stiff and become stuck in small blood vessels.
- Trial name
- NCT06145282
- Testing
- Briquilimab, Filgrastim, Abatacept
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Sickle Cell Disease
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures.
- Trial name
- NCT03077542
- Testing
- haploidentical stem cell transplant, Sirolimus, Alemtuzumab
- Where
- United States
- Volunteers
- 57 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
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)
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)
Myeloproliferative Disorders, Acute Myelogenous Leukemia
Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
The are a variety of cancerous diseases of the blood and bone marrow that can be potentially cured by bone marrow transplantation (BMT).
- Conditions
- Myeloproliferative Disorders, Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00003838
- Testing
- T-cell replete PBPC allograft, Methotrexate, Cyclosporine
- Where
- United States
- Volunteers
- 202 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Acute Leukemia, Myelodysplasia
Calcineurin Inhibitor-Free Interventions BMT CTN 1301 for Prevention of Graft-versus-Host Disease (BMT CTN 1301)
The study is designed as a three arm randomized Phase III, multicenter trial comparing two calcineurin inhibitor (CNI)-free strategies for Graft-versus-Host Disease (GVHD) prophylaxis to standard tacrolimus and methotrexate…
- Trial name
- NCT02345850
- Testing
- Unmanipulated Bone Marrow Graft with Tacrolimus/Methotrexate, Mobilized CD34-selected Peripheral Blood Stem Cell graft, Unmanipulated Bone Marrow Graft with Cyclophosphamide
- Where
- 28 sites in United States
- Volunteers
- 346 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Graft vs Host Disease
Dexamethasone to Prevent Oral Chronic Graft-versus-Host Disease
This study will determine if a dexamethasone mouth rinse can reduce the risk of developing oral chronic graft-versus-host disease (cGVHD) in patients who have undergone a bone marrow (stem cell) transplant procedure.
- Trial name
- NCT00391170
- Testing
- Topical Dexamethasone
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA), Severe Aplastic Anemia (SAA)
Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
This study will evaluate the safety and effectiveness of treating patients with severe aplastic anemia (SAA) or myelodysplastic syndrome (MDS) with both peripheral blood stem cells from a family member and umbilical cord blood…
- Trial name
- NCT00604201
- Testing
- Umbilical Cord Blood
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Multiple Myeloma
Stem Cell Transplant With Lenalidomide Maintenance in Patients With Multiple Myeloma (BMT CTN 0702)
The study is designed as a Phase III, multicenter trial of tandem autologous transplants plus maintenance therapy versus the strategy of single autologous transplant plus consolidation therapy with lenalidomide, bortezomib and…
- Trial name
- NCT01109004
- Testing
- Lenalidomide, lenalidomide, bortezomib and dexamethasone
- Where
- 54 sites in United States
- Volunteers
- 758 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Neoplasm Metastasis
Stem Cell Transplantation for Metastatic Solid Tumors
The goal of this research study is to identify other types of cancer (malignant neoplasms) that may be treatable with stem cell transplantation (allogenic peripheral blood stem cell transplantation.
- Trial name
- NCT00001880
- Testing
- methotrexate, Cyclosporin
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Multiple Myeloma
Stem Cell Transplantation in Individuals With Multiple Myeloma (BMT CTN 0102)
The study is designed as a Phase III, multi-center trial of tandem autologous transplants versus the strategy of autologous followed by Human Leukocyte Antigen (HLA)-matched sibling non-myeloablative allogeneic transplant.
- Trial name
- NCT00075829
- Testing
- One Autologous Transplant, Non-Myeloablative Allogeneic Transplant, Second Autologous Transplant
- Where
- 36 sites in United States
- Volunteers
- 710 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Graft vs Host Disease, Immune System Disorders
Treatment for Acute Graft-Versus-Host Disease (BMT CTN 0302)
The study is a randomized Phase II, four arm treatment trial. The primary purpose of the study is to define new agents with promising activity against acute graft-versus-host disease (GVHD) suitable for testing against…
- Trial name
- NCT00224874
- Testing
- Etanercept, Mycophenolate Mofetil, Denileukin Diftitox
- Where
- 19 sites in United States
- Volunteers
- 180 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)