Clinical trials: Bone Marrow Failure Syndrome
Hematologic Malignancies, Nonmalignant Diseases
Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)
Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609814
- Testing
- Clofarabine, Fludarabine Injection
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, San Francisco
Iron Overload
Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
- Trial name
- NCT01546415
- Testing
- Desferasirox
- Where
- 7 sites in China
- Volunteers
- 64 people
- Sponsor
- Novartis Pharmaceuticals
Neutropenic Enterocolitis, Myeloablative Chemotherapy Induced Bone Marrow Aplasia
Effectiveness and Safety of MMSCs for Enhancing Hematopoietic Recovery and Prophylaxis of Neutropenic Enterocolitis
Subjects will undergo peripheral blood stem cell mobilisation and collection with subsequent high-dose chemotherapy. After finalization of high-dose chemotherapy subjects will receive bone marrow derived allogeneic multipotent…
- Trial name
- NCT02145923
- Testing
- Peripheral blood stem cell mobilisation and collection, High-dose chemotherapy, Bone marrow derived allogeneic MMSCs infusion
- Where
- Russia
- Volunteers
- 16 people
- Sponsor
- Burnasyan Federal Medical Biophysical Center
Fanconi Anemia
Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants
RATIONALE: Studying samples of bone marrow from patients with Fanconi anemia and from healthy participants in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to Fanconi…
- Trial name
- NCT00896740
- Testing
- microarray analysis, biopsy
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Aplastic Anemia
Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia
This study will examine the safety and effectiveness of a new drug combination for treating patients with severe aplastic anemia.
- Trial name
- NCT00005935
- Testing
- Mycophenolate mofetil, Cyclosporine
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Telomere Disease
Low-Dose Danazol for the Treatment of Telomere Related Diseases
Background: DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA.
- Trial name
- NCT03312400
- Testing
- Danazol
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Aplastic
Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia
Our hypothesis is that ex vivo activated immune cells would produce multiple known and unknown potent hematopoietic cytokines, working in concert, these cytokines help stem cell growth and differentiation.
- Trial name
- NCT00399971
- Testing
- Ex Vivo Immunotherapy
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Zhongxing Yangfan Biotech Co. Ltd.
Severe Aplastic Anemia
Combination Therapy of Severe Aplastic Anemia
This study will test the safety and effectiveness of a combination of three drugs in treating severe aplastic anemia and preventing its recurrence.
- Trial name
- NCT00001964
- Testing
- Cyclosporine A, ATG, MMF
- Where
- United States
- Volunteers
- 104 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma
- Trial name
- NCT00084695
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Milton S. Hershey Medical Center
Aplastic Anemia
Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia
OBJECTIVES: I. Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin…
- Trial name
- NCT00004474
- Testing
- Anti-thymocyte globulin, Cyclophosphamide, Bone marrow transplantation
- Where
- 4 sites in United States
- Volunteers
- 224 people
- Sponsor
- Roswell Park Cancer Institute
Dyskeratosis Congenita, Fanconi Anemia
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Dyskeratosis Congenita, Fanconi Anemia, Myelodysplastic Syndromes, Pearson Marrow-pancreas Syndrome
- Trial name
- NCT00499070
- Testing
- polymerase chain reaction, flow cytometry, immunologic technique
- Where
- 10 sites in Austria, Belgium and 8 more
- Volunteers
- 119 people
- Sponsor
- University Hospital Freiburg
Graft Versus Host Disease
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents…
- Trial name
- ASCENT (NCT03924401)
- Testing
- Abatacept
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Emory University
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Blasts 10-19 Percent of Bone Marrow Nucleated Cells, Blasts More Than 5 Percent of Bone Marrow Nucleated Cells
CPX-351 in Treating Patients With Relapsed or Refractory High Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
This phase I trial studies best dose and side effects of liposome-encapsulated daunorubicin-cytarabine (CPX-351) and how well it works in treating patients with high risk myelodysplastic syndrome or chronic myelomonocytic…
- Conditions
- Blasts 10-19 Percent of Bone Marrow Nucleated Cells, Blasts More Than 5 Percent of Bone Marrow Nucleated Cells, High Risk Chronic Myelomonocytic Leukemia, Recurrent Chronic Myelomonocytic Leukemia
- Trial name
- NCT03896269
- Testing
- Liposome-encapsulated Daunorubicin-Cytarabine
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- M.D. Anderson Cancer Center
Haematological Malignancy, Acute Leukemia
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts
To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT04331483)
- Testing
- Physical activity program
- Where
- France
- Sponsor
- Centre Leon Berard
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)
Severe Aplastic Anemia, Bone Marrow Failure Syndromes
Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia
Our primary objective is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors.
- Trial name
- NCT02224872
- Testing
- Bone marrow transplant, Thymoglobulin, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 18 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Severe Aplastic Anemia
Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).
- Trial name
- NCT06039436
- Testing
- Anti-Thymocyte Globulin (Rabbit)
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Anhui Provincial Hospital