Clinical trials: Bone Marrow Failure Syndromes
Aplastic Anemia
Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia
To ascertain the risk factors of AA in ZheJiang Province of China at the moment, especially the comprehensive multiple factors risks. The author conducted a case-control research included 338 AA cases and 1464 controls.
- Trial name
- MCSAA (NCT03208647)
- Testing
- AA or not
- Volunteers
- 1,802 people, healthy volunteers welcome
- Sponsor
- Shengyun Lin
Immune Thrombocytopenia, Chemotherapy Induced Thrombocytopenia
Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative Thrombocytopenias.
To utilise extended platelet parameters in order to individuate Immune Thrombocytopenia (ITP) from hypo-proliferative causes of thrombocytopenia.
- Conditions
- Immune Thrombocytopenia, Chemotherapy Induced Thrombocytopenia, Myelodysplasia, Aplastic Anaemia
- Trial name
- NCT01933035
- Testing
- Immune Thrombocytopenics, Hypo-proliferative thrombocytopenics, Control Population
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Beth Israel Medical Center
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Haematological Malignancy, Acute Leukemia
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT05298930)
- Testing
- Physical activity program
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Centre Leon Berard
Recurrent Severe Aplastic Anemia, Refractory Severe Aplastic Anemia
A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia
This phase I trial evaluates the safety and feasibility of using a reduced-intensity regimen of cyclophosphamide, pentostatin, and anti-thymocyte globulin prior to a CD4+ T-cell depleted haploidentical hematopoietic cell…
- Trial name
- NCT05757310
- Testing
- Anti-Thymocyte Globulin, Biospecimen Collection, Bone Marrow Aspirate
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
AML, ALL
Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders
Allogeneic stem cell transplantation may provide long-term remissions for some patients with hematological malignancies. However, allogeneic transplantation is associated with a significant risk of potentially life threatening…
- Conditions
- AML, ALL, CLL, Myelodysplastic Syndrome
- Trial name
- NCT00533923
- Testing
- Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Alcohol-Related Carcinoma, Fanconi Anemia
Role of Acetaldehyde in the Development of Oral Cancer
This is a minimal risk intervention study where healthy volunteers and individuals with Fanconi anemia will consume a single dose of alcohol and provide primarily non-invasive biological samples at various time points.
- Conditions
- Alcohol-Related Carcinoma, Fanconi Anemia, Oral Cavity Carcinoma
- Trial name
- NCT05973656
- Testing
- Alcohol, Biospecimen Collection, Breath Test
- Where
- United States
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Masonic Cancer Center, University of Minnesota
Transfusional Iron Overload, Thalassemia Major
High-Tc Susceptometer to Monitor Transfusional Iron Overload
The proposed research project will continue the application and development of a new method (biomagnetic susceptometry) that measures magnetic fields to determine how much iron is in the liver.
- Conditions
- Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes
- Trial name
- NCT01241357
- Testing
- Hepatic biomagnetic susceptibility measurement
- Where
- United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Columbia University
Neutropenia, Thrombocytopenia
Effects of Levetiracetam Monotherapy on Various Hematological Parameters in Adult Patients Suffering of Epilepsy
Low therapeutic index of established antiepileptic drugs (AEDs) coupled with better understanding of the pathophysiology of seizure has encouraged the development of several novel AEDs.
- Conditions
- Neutropenia, Thrombocytopenia, Aplastic Anemia, Anemia
- Trial name
- NCT06491303
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Assiut University
Leukemia, Aplastic Anemia
Protection Against Benzene Toxicity
We are investigating the role of an enzyme (NQO1) in protection against the bone marrow toxicity of the occupational and environmental toxicant benzene.
- Trial name
- NCT00011453
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Aplastic Anemia
Alefacept in Patients With Relapsed/Refractory Aplastic Anemia
Aplastic Anemia (AA) is an autoimmune hematologic stem cell disease mediated by activated T-lymphocytes that leads to pancytopenia. The disease related morbidity and mortality if left untreated can approach 90%.
- Trial name
- NCT01267643
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- The Cleveland Clinic
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
Aplastic Anemia, Drug Effect
A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary TD-NSAA
Aplastic anemia (AA) is a group of clinical syndromes. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but…
- Trial name
- NCT06004791
- Testing
- Herombopag + CsA, rhTPO combined with Herombopag + CsA
- Where
- China
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Peking Union Medical College Hospital
Chronic Myeloproliferative Disorders, Fanconi Anemia
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of…
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00900055
- Testing
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction
- Where
- United States
- Volunteers
- 213 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
FANCONI ANEMIA
Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT
While stem cell transplantation has proven an effective means of treating a wide variety of diseases involving hematopoietic stem cells and their progeny, a shortage of donors has proved a major impediment to the widest…
- Trial name
- RAFHAS (NCT00586274)
- Testing
- CD34 selected haploidentical PBSCT, Fludarabine, T cell infusion
- Where
- 2 sites in United States
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Inherited BMF Syndrome
Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes
Inherited bone marrow failure syndromes (IBMFSs) are a diverse collection of genetic illnesses characterized by various degrees of peripheral cytopenias due to defective single-lineage or multi-lineage hematopoiesis, it can…
- Trial name
- NCT05436587
- Testing
- The whole-exome sequencing
- Where
- Egypt
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Sohag University
Aplastic Anemia, Leukemia
Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
This is a genetic disease (transmitted through the parents' genes) called Fanconi Anemia. Because of that genetic disease, the bone marrow has changed and now has failed, or has given rise to a preleukemia called myelodysplastic…
- Conditions
- Aplastic Anemia, Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT00987480
- Testing
- Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine., CliniMACS device
- Where
- 6 sites in United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
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
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant
This phase I/II trial studies how well cytokine-treated veto cells work in treating patients with hematologic malignancies following stem cell transplant.
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Aplastic Anemia, Bone Marrow Failure
- Trial name
- NCT03622788
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Cytokine-treated Veto Cells
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center