Clinical trials: Aplasia
Corpus Callosum Agenesis
BECOME CHILDREN OF HOLDERS Corpus Callosum Agenesis Screened IN PERIOD Antenatal
The corpus callosum agenesis is an interesting malformation between 0.05% and 1.5% of the general population. This malformation is mostly diagnosed prenatally by ultrasound, usually at 22 weeks of gestation, and the use of…
- Trial name
- NCT02826824
- Where
- France
- Volunteers
- 60 people
- Sponsor
- University Hospital, Strasbourg, France
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
Severe Aplastic Anemia (SAA)
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to…
- Trial name
- NCT05323617
- Testing
- Romiplostim, Antithymocyte Globulin, Cyclosporine A
- Sponsor
- Amgen
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Primary Immune Thrombocytopenic Purpura, Amegakaryocytic Aplasia
Ultrasound to Detect Splenomegally in Patients With Isolated Thrombocytopenia
Investigate prevalence of splenomegally in patients with isolated thrombocytopenia. Asess effect of splenomegally in clinical picture ,Complication, treatment response and disease outcome To explain possible causes of…
- Conditions
- Primary Immune Thrombocytopenic Purpura, Amegakaryocytic Aplasia, Unilineage Myelodysplastic Syndrome ( Megakaryocyte Dysplasia), Lymphoproliferative Disorder With Secondary ITP
- Trial name
- NCT07182760
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
Myelodysplastic Syndromes
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Background: Myelodysplastic syndrome (MDS) and myelodysplastic/myeloproliferative neoplasm (MDS/MPN) are blood disorders that can cause serious complications in children and adults.
- Trial name
- NCT06303193
- Testing
- pacritinib
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease, Thalassemia
Multi-Center Study of Iron Overload: Pilot Study
The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions…
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- MCSIO (NCT01114776)
- Where
- 3 sites in United States, Germany and 1 more
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Hematologic Malignancies, Nonmalignant Diseases
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies
- Trial name
- NCT03609840
- Testing
- Thiotepa
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
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
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor
The purpose of this study is to assess the feasibility of Plerixafor used in combination with G-CSF (Granulocyte Colony Stimulating Factor) in 5 Fanconi anemia patients to mobilize and collect a sufficient number of peripheral…
- Trial name
- FancoMob (NCT02678533)
- Testing
- G-CSF, Plerixafor
- Where
- France
- Volunteers
- 4 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7)
Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia
This research study is studying identification of de novo Fanconi anemia in younger patients with newly diagnosed acute myeloid leukemia.
- Conditions
- Childhood Acute Erythroleukemia (M6), Childhood Acute Megakaryocytic Leukemia (M7), Childhood Acute Minimally Differentiated Myeloid Leukemia (M0), Childhood Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT01146210
- Testing
- laboratory biomarker analysis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Oncology Group
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies
This phase II trial studies how well reduced intensity donor stem cell transplant works in treating patients with hematologic malignancies.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q)
- Trial name
- NCT01384513
- Testing
- Fludarabine, Busulfan, Total Body Irradiation (TBI)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Fanconi Anemia
Blood Cell Collection for Future Use in Individuals With Fanconi Anemia
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells.
- Trial name
- NCT00271089
- Testing
- PBSC Collection, Bone Marrow Harvest, CliniMacs Cell Selection System
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia, Platelet Transfusion Refractoriness
Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness
Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR).
- Trial name
- NCT06254560
- Testing
- Rituximab
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Aplastic Anaemia
Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)
The purpose of this study is to examine the effect of G-CSF on disease free survival and overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.
- Trial name
- SAA-G-CSF (NCT01163942)
- Testing
- G-CSF, Early retreatment with ATG
- Where
- 71 sites in Czechia, France and 7 more
- Volunteers
- 205 people
- Sponsor
- European Society for Blood and Marrow Transplantation
Aplastic Anemia
Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia
Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation.
- Trial name
- NCT01105273
- Testing
- anti-thymocyte globulin, filgrastim, Fludarabine
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Asan Medical Center
Anemia, Aplastic, Anemia, Hypoplastic
A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients
Severe aplastic anemia (SAA) is a life-threatening blood disease which can be effectively treated with immunosuppressive drug regimens or allogeneic stem cell transplantation.
- Conditions
- Anemia, Aplastic, Anemia, Hypoplastic, Thrombocytopenia
- Trial name
- NCT00922883
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Chronic Kidney Disease
Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease
The purpose of this study is to evaluate the ability of peginesatide (AF37702) to increase and maintain increased hemoglobin levels in participants with chronic kidney disease (CKD) (either not on dialysis, receiving regular…
- Conditions
- Anemia, Chronic Kidney Disease, Chronic Renal Failure, Pure Red Cell Aplasia
- Trial name
- NCT00314795
- Testing
- Peginesatide
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 22 people
- Sponsor
- Takeda
Sever Aplastic Anaemia, Elderly (People Aged 65 or More)
Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia
This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.
- Conditions
- Sever Aplastic Anaemia, Elderly (People Aged 65 or More), Immunosuppressive Treatment
- Trial name
- NCT07010237
- Testing
- Dose-attenuated IST and Hetrombopag
- Where
- China
- Volunteers
- 65 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Sargramostim in Reducing Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplantation for Hematologic Cancer or Aplastic Anemia
RATIONALE: Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy or…
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00053157
- Testing
- sargramostim
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Roswell Park Cancer Institute