Clinical trials: sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers.
- Conditions
- Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL, Hypoplastic MDS
- Trial name
- NCT05998408
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Psoriasis, Atherosclerotic Cardiovascular Disease
Pilot Study to Evaluate the Effect of Nicotinamide Riboside on Immune Activation in Psoriasis
Background: Psoriasis causes chronic inflammation in the body. Researchers want to see if a kind of vitamin B3 dietary supplement can help. This might lead to more treatment options.
- Conditions
- Psoriasis, Atherosclerotic Cardiovascular Disease, Obesity, Dyslipidemia
- Trial name
- NCT04271735
- Testing
- Niagen
- Where
- United States
- Volunteers
- 29 people
- 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)
Pulmonary Artery Hypertension, Congenital Heart Disease
Heart Catheterization Using Magnetic Resonance Imaging (MRI) Fluoroscopy and Passive Guidewires
Background: A heart catheterization is a diagnostic heart procedure used to measure pressures and take pictures of the blood flow through the heart chambers.
- Conditions
- Pulmonary Artery Hypertension, Congenital Heart Disease, Structural Heart Disease
- Trial name
- NCT03152773
- Testing
- MRI Heart guidewire catheterization
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
Healthy, Latent Tuberculosis Infection (LTBI)
Characterization of Tissue-Specific Immune Responses to Bronchoscopic Instillation of Mycobacterial Antigens Into the Human Lung
Background: Tuberculosis (TB) is a lung disease. It is caused by inhaling a type of airborne bacterium. Tuberculin Purified Protein Derivative (PPD) is used to test for TB exposure. It is usually injected under a person s skin.
- Trial name
- NCT05027958
- Testing
- Tuberculin Purified Protein Derivative
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Functional Mitral Regurgitation
NHLBI DIR Transcatheter Mitral Cerclage Annuloplasty Early Feasibility Study
This research protocol tests a new technique and devices that we have developed to treat functional mitral valve regurgitation, called transcatheter mitral valve cerclage annuloplasty, otherwise known as "cerclage".
- Trial name
- NCT03929913
- Testing
- Transmural Systems Transcatheter Mitral Cerclage Annuloplasty (TMCA)
- Where
- 3 sites in United States
- Volunteers
- 19 people
- 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)
Leukemia, Leukemia, Lymphocytyc
PCI-32765 for Special Cases of Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
Background: Chronic lymphocytic leukemia and small lymphocytic lymphoma (CLL/SLL) are types of blood or lymph node cancers that mostly affect the elderly.
- Conditions
- Leukemia, Leukemia, Lymphocytyc, CLL (Chronic Lymphocytic Leukemia), SLL (Small Lymphocytic Lymphoma)
- Trial name
- NCT01500733
- Testing
- PCI 32765
- Where
- United States
- Volunteers
- 86 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Kidney Cancer
HERV-E TCR Transduced Autologous T Cells in People With Metastatic Clear Cell Renal Cell Carcinoma
Background: Gene transfer is a new cancer therapy takes white blood cells from a person and grows them in a lab. The cells are changed with a virus to attack tumor cells, then returned to the person.
- Trial name
- NCT03354390
- Testing
- cell infusion
- Where
- United States
- Volunteers
- 17 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)
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
A Phase II Study Using Ibrutinib and Short-Course Fludarabine in Treatment-Naive CLL
This is a pilot phase 2 study investigating the safety and efficacy of ibrutinib combined with short-course fludarabine in previously untreated CLL patients.
- Trial name
- NCT02514083
- Testing
- Ibrutinib, Fludarabine
- Where
- United States
- Volunteers
- 29 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)
Lymphangioleiomyomatosis
Discovery of Sirolimus Sensitive Biomarkers in Blood
Background: Lymphangioleiomyomatosis (LAM) is a rare, progressive disease. It usually affects women in the prime of their lives. It typically results in lung destruction.
- Trial name
- NCT03304678
- Testing
- Sirolimus 2mg
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)