Clinical trials: Bone Marrow Aplasia
Severe Aplastic Anemia
Efficacy and Safety of CSA and Avatrombopag for the Treatment of SAA in the Elderly
This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of CSA in combination with Avatrombopag in elderly patients with very/sever aplastic anemia treated for the first time.
- Trial name
- SAA (NCT05433922)
- Testing
- Avatrombopag
- Where
- China
- Volunteers
- 80 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Severe Aplastic Anemia
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant.
- Trial name
- TransIT (NCT05600426)
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 52 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- Boston Children's Hospital
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
Anemia, Aplastic
Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.
- Trial name
- NCT02857530
- Testing
- rhTPO
- Where
- China
- Volunteers
- 250 people
- Sponsor
- Tianjin Medical University General Hospital
Fanconi's Anemia
Phase I/II Study of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the probability of engraftment with total body irradiation, cyclophosphamide, fludarabine, and anti-thymocyte globulin followed by HLA nongenotypically identical donor, T-cell depleted hematopoietic cell…
- Trial name
- NCT00005898
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of Minnesota
Fanconi Anemia
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
The objective of this clinical trial is to develop a cell therapy for Fanconi Anemia which enables enhanced donor hematopoietic and immune reconstitution with decreased toxicity by transplanting depleted stem cells from a donor…
- Trial name
- NCT04784052
- Testing
- JSP191, CliniMACS Prodigy System, Depleted Stem Cell Transplant
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Porteus, Matthew, MD
Aplastic Anemia
Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant
This clinical trial is studying how well giving cyclophosphamide together with anti-thymocyte globulin followed by methotrexate and cyclosporine works in preventing chronic graft-vs-host disease (GVHD) in patients with severe…
- Trial name
- NCT00343785
- Testing
- cyclophosphamide, anti-thymocyte globulin, cyclosporine
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Fred Hutchinson Cancer Center
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia
- Trial name
- NCT02566304
- Testing
- Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Vitamin D Deficiency, Stem Cell Transplant Complications
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as…
- Conditions
- Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder
- Trial name
- NCT03176849
- Testing
- Vitamin D3, Standard Vitamin D3 Supplementation
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Phoenix Children's Hospital
Aplastic Anemia, Leukemia, Myeloid, Acute
King's Invasive Aspergillosis Study II
Invasive fungal disease (IFD) is an important cause of mortality and morbidity in patients undergoing intensive chemotherapy or transplantation.
- Conditions
- Aplastic Anemia, Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Bone Marrow Transplantation
- Trial name
- NCT02875743
- Testing
- Posaconazole
- Where
- United Kingdom
- Volunteers
- 120 people
- Sponsor
- King's College Hospital NHS Trust
Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia
Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Chronic Myeloproliferative Disorders, Diamond-blackfan Anemia, Fanconi Anemia, Graft Versus Host Disease
- Trial name
- NCT00290628
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 43 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
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Chronic Myeloid Leukemia, Acute Myelogenous Leukemia
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT
The primary objective of this study is to assess the safety and efficacy of performing unrelated stem cell transplants using intravenous busulfan and fludarabine as preparative therapy and tacrolimus plus methotrexate as the GVHD…
- Conditions
- Chronic Myeloid Leukemia, Acute Myelogenous Leukemia, Myelodysplasia, Acute Lymphocytic Leukemia
- Trial name
- NCT00516152
- Testing
- Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSF
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- University of California, San Francisco
Fanconi Anemia
Eltrombopag for Treatment of Fanconi Anemia
An open-label, phase II study to assess the efficacy and safety of eltrombopag for the treatment of children and adolescents with Fanconi anemia.
- Trial name
- NCT06045052
- Testing
- Eltrombopag
- Where
- Spain
- Volunteers
- 8 people
- Sponsor
- Julian Sevilla
Myelodysplastic Syndrome, Aplastic Anemia
Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic…
- Trial name
- NCT00806598
- Testing
- Thymoglobulin, Cyclosporine, Methylprednisolone
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- M.D. Anderson Cancer Center
Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death.
- Conditions
- Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
- Trial name
- NCT01659606
- Testing
- alemtuzumab, Fludarabine, Cyclosporins
- Where
- 13 sites in United States, Norway and 1 more
- Volunteers
- 40 people
- Sponsor
- Boston Children's Hospital
Acute Myeloid Leukemia, Acute Leukemia
Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies
New conditioning regimens are still needed to maximize efficacy and limit treatment-related deaths of allogeneic transplantation for advanced hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma
- Trial name
- NCT01499147
- Testing
- fludarabine/busulfan, fludarabine/ melphalan, ATG
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- University of Illinois Chicago
Severe Combined Immunodeficiency, Fanconi Anemia
Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative…
- Conditions
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita, Sickle Cell Disease
- Trial name
- NCT06731036
- Testing
- CD34+ cell enriched stem cell graft
- Sponsor
- University of Florida
Fanconi Anemia
Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia
The purpose of this research study is to evaluate the safety of the drug Etanercept (Enbrel) and to determine if this drug can help in the treatment of early bone marrow failure in patients with Fanconi anemia.
- Trial name
- NCT00965666
- Testing
- Etanercept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati