Clinical trials: Anemia, Aplastic
Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with…
- Conditions
- Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Hereditary Sideroblastic Anemia
- Trial name
- NCT04965597
- Testing
- Treosulfan, Fludarabine Phosphate, Tacrolimus
- Where
- 24 sites in United States
- Volunteers
- 40 people
- Sponsor
- Fred Hutchinson Cancer Center
Severe Aplastic Anemia
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding.
- Trial name
- NCT02828592
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Northside Hospital, Inc.
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Aplastic Anemia
Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia
Acquired Aplastic anemia is one of the most frequent reason of bone marrow failure in East (Pakistan). The first treatment option is Allogenic Bone Marrow transplantation which is an expansive treatment option and also require a…
- Trial name
- ATG (NCT02028416)
- Testing
- ATG-fresenius
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- National Institute of Blood Disease Center, Pakistan
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
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
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
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Acquired Aplastic Anemia.
Randomised Study Comparing Different Dosages of Rabbit ATG in Patients With SAA
The objective of this study is to evaluate the feasibility and effectiveness of immunosuppressive therapy (IST) using rabbit anti-thymocyte globulin (ATG) (Thymoglobuline, Genzyme) for patients with very severe aplastic anemia…
- Trial name
- NCT01844635
- Testing
- Thymoglobulin
- Where
- Japan
- Volunteers
- 320 people
- Sponsor
- Nagoya University
Refractory Aplastic Anemia
Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia
This is a phase 2, single-center, single-arm, open-label trial. Simon's two-stage design is performed to evaluate the efficacy of low-dose Decitabine in refractory aplastic anemia.
- Trial name
- LODACA (NCT04854889)
- Testing
- Decitabine
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA)
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and…
- Conditions
- Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia
- Trial name
- NCT01224496
- Testing
- Chinese herbal concoction twice a day for 6 months
- Where
- Singapore
- Volunteers
- 38 people
- Sponsor
- Singapore General Hospital
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Aplastic Anemia
Revolade Tablets Specified Drug-use Survey
This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).
- Trial name
- NCT06287268
- Testing
- eltrombopag
- Where
- 17 sites in Japan
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Bone Marrow Failure Disorders, Aplastic Anemia
TPO-Mimetic Use in Children for Hematopoietic Failure
This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure…
- Conditions
- Bone Marrow Failure Disorders, Aplastic Anemia, Thrombocytopenia, Refractory Cytopenia of Childhood
- Trial name
- NCT04478227
- Testing
- Romiplostim
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Anjali Sharathkumar
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia
This study is for patients with Severe Aplastic Anemia (SAA). A stem cell transplant from a genetically matched sibling donor can help or cure this disease in 85 to 100 percent of patients.
- Trial name
- AACD34 (NCT00186797)
- Testing
- Allogeneic stem cell transplant, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemia, Cytopenia
A Study of Emapalumab for Pediatric Aplastic Anemia
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
- Conditions
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- Trial name
- NCT06430788
- Testing
- Emapalumab
- Where
- 6 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Fanconi Anemia
Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi Anemia
The goal of this observational study is to analyze the data included in the Spanish Registry of Patients with Fanconi anemia to better understand the natural history of the disease, identify genetic risk and prognostic factors…
- Trial name
- NCT06490510
- Where
- Spain
- Volunteers
- 227 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Lymphoma, Leukemia
Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation
RATIONALE: Following stem cell transplantation, a major risk is graft-versus-host disease (GVHD). This occurs when donor immune cells that have been infused recognise the host's cells as 'foreign' and attack these cells.
- Conditions
- Lymphoma, Leukemia, Myeloma, Myelodysplastic Syndromes
- Trial name
- ToTem (NCT03836690)
- Testing
- CD62L- Tem
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University College, London