Clinical trials: Bone Marrow Failure Syndromes
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
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, Blackfan Diamond Syndrome
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.
- Conditions
- Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia
- Trial name
- LeucineDBA (NCT01362595)
- Testing
- leucine
- Where
- 11 sites in United States
- Volunteers
- 55 people
- Sponsor
- Northwell Health
Myelodysplastic Syndrome
Addition of Vorinostat to Azacitidine in Higher Risk MDS a Phase II add-on Study in Patients With Azacitidine Failure
Azacytidine (AZA) is the current standard of care for frontline patient treated with high-risk MDS and is clinically active in all type of MDS, however, 50% of the patients will never respond.
- Trial name
- GFM-AZA-VOR (NCT01748240)
- Testing
- Azacitidine and oral vorinostat
- Where
- 24 sites in France
- Volunteers
- 21 people
- Sponsor
- Groupe Francophone des Myelodysplasies
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
Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia
Tipifarnib in Treating Patients With Acute Myeloid Leukemia in Remission
This randomized phase III trial studies tipifarnib in treating patients with acute myeloid leukemia (AML) in remission. Tipifarnib may stop the growth of cancer cells by blocking the enzymes necessary for their growth.
- Conditions
- Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia, Adult Acute Monocytic Leukemia, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00093470
- Testing
- Clinical Observation, Tipifarnib
- Where
- 341 sites in United States, Israel and 1 more
- Volunteers
- 144 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer
The purpose of this study is to compare the effects (good and bad) of the medication basiliximab in combination with cyclosporine (investigational therapy) for the prevention of a complication of bone marrow transplantation known…
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia
- Trial name
- NCT00975975
- Testing
- Basiliximab
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Indiana University
Fanconi Anemia
Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow…
- Trial name
- NCT00595127
- Testing
- Cyclophosphamide, Fludarabine, Total body irradiation (TBI)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
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)
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Severe Aplastic Anemia, Very Severe Aplastic Anemia
Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts.
- Conditions
- Severe Aplastic Anemia, Very Severe Aplastic Anemia, Moderate Aplastic Anemia
- Trial name
- NCT01703169
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of Utah
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
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
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.
Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia
This phase II trial studies how well total-body irradiation (TBI) works when given together with fludarabine phosphate and cyclophosphamide followed by donor bone marrow transplant, mycophenolate mofetil, and cyclosporine in…
- Conditions
- Acute Myeloid Leukemia in Remission, de Novo Myelodysplastic Syndrome, Fanconi Anemia, Previously Treated Myelodysplastic Syndrome
- Trial name
- NCT00453388
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Cyclosporine
- Where
- 5 sites in United States, Brazil
- Volunteers
- 6 people
- Sponsor
- Fred Hutchinson Cancer Center
Myelodysplastic Syndrome
Decitabine and Clofarabine in Higher Risk Myelodysplastic Syndromes (MDS)
The goal of this clinical research study is to learn if sequential administration of decitabine and clofarabine can help to control MDS better than decitabine alone. The safety of this drug combination will also be studied.
- Trial name
- NCT00903760
- Testing
- Decitabine, Clofarabine
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- M.D. Anderson Cancer Center
Leukemia
Alemtuzumab in Myelodysplastic Syndrome (MDS), Aplastic Anemia, and T-Cell Large Granular Lymphocytic Leukemia (T-GL)
The goal of this clinical research study is to determine the effectiveness of alemtuzumab in patients with aplastic anemia, MDS, or T-Cell large granular lymphocytic leukemia. The safety of alemtuzumab will also be studied.
- Trial name
- NCT01191749
- Testing
- Alemtuzumab
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- M.D. Anderson 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)