Clinical trials: Severe Aplastic Anemia
Nonmalignant Neoplasm
Alemtuzumab and Low-Dose Cyclosporine in Treating Patients With Severe Aplastic Anemia or Acquired Marrow Failure
RATIONALE: Immunosuppressive therapies, such as alemtuzumab and cyclosporine, may improve bone marrow function and increase blood cell counts.
- Trial name
- NCT00895739
- Testing
- alemtuzumab, cyclosporine
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Federico II University
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
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Fanconi Anemia, Severe Aplastic Anemia
Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia
The purpose of this study is to discover whether children and adults with Fanconi anemia (FA) can be safely and effectively transplanted with Human Leukocyte Antigen (HLA) mismatched (up to one haplotype), HLA-matched sibling, or…
- Trial name
- Mafia (NCT00590460)
- Testing
- CAMPATH-1H, Anti-CD45, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Baylor College of Medicine
Immune Thrombocytopenia, Autoimmune Hemolytic Anemia
Severe Immune Cytopenia Registry Www.Sic-reg.Org
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the…
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Trial name
- sic-reg (NCT03576742)
- Testing
- potential biomarkers
- Where
- Austria
- Volunteers
- 80 people
- Sponsor
- Medical University of Graz
Severe Aplastic Anemia
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
- Trial name
- SOAR (NCT02998645)
- Testing
- eltrombopag, Cyclosporine
- Where
- 20 sites in Brazil, Hong Kong and 7 more
- Volunteers
- 54 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Basiliximab #2: In-Vivo Activated T-Cell Depletion to Prevent Graft-Versus_Host Disease (GVHD) After Nonmyeloablative Allotransplantation for the Treatment of Blood Cancer
The purpose of this study is to compare the effects (good and bad) of the medication basiliximab in combination with cyclosporine (investigational therapy) for the prevention of a complication of bone marrow transplantation known…
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Chronic Lymphocytic Leukemia
- Trial name
- NCT00975975
- Testing
- Basiliximab
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Indiana University
Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
Background: Immune bone marrow failure is a condition that occurs when a person s immune system attacks the cells of the bone marrow. This can lead to diseases including different types of anemias and blood cancers.
- Conditions
- Severe Aplastic Anemia, Single Lineage Cytopenias, T-LGL, Hypoplastic MDS
- Trial name
- NCT05998408
- Testing
- Ruxolitinib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Bone Marrow Transplantation in Treating Patients With Severe Aplastic Anemia or Rejection of Previous Bone Marrow Transplant
RATIONALE: Bone marrow from donors may be able to treat patients with severe aplastic anemia and patients whose bodies have rejected previous bone marrow transplantation.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00005852
- Testing
- anti-thymocyte globulin, filgrastim, muromonab-CD3
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Influenza Vaccine in Preventing Flu in Patients Who Have Undergone Stem Cell Transplant and in Healthy Volunteers
RATIONALE: The influenza vaccine may help prevent flu in patients who have undergone stem cell transplant. PURPOSE: This clinical trial is studying how well the influenza vaccine works in preventing flu in patients who have…
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00952185
- Testing
- cytology specimen collection procedure, fluorescent antibody technique, assessment of therapy complications
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Acute Leukemia, Chronic Leukemia
Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation
The purpose of this study is to analysis of the pharmacokinetics of fludarabine for hematopoietic stem cell transplantation in pediatric patients.
- Conditions
- Acute Leukemia, Chronic Leukemia, Severe Aplastic Anemia
- Trial name
- NCT01472055
- Testing
- Fludarabine
- Where
- South Korea
- Volunteers
- 46 people
- Sponsor
- Seoul National University Hospital
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Aplastic Anemia, Drug Effect
Avatrombopag Usage in NSAA
This is a multicenter, open-label, prospective one arm study to explore the efficacy and safety of Avatrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited.
- Trial name
- NCT04728789
- Testing
- Avatrombopag 20 MG
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Peking Union Medical College Hospital
Severe Aplastic Anemia, Very Severe Aplastic Anemia
Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia
The investigators hypothesis is that eltrombopag given to patients with moderate to very severe aplastic anemia will result in an increase in platelet counts.
- Conditions
- Severe Aplastic Anemia, Very Severe Aplastic Anemia, Moderate Aplastic Anemia
- Trial name
- NCT01703169
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of Utah
Aplastic Anemia
A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia
Severe acquired aplastic anaemia (SAA) is a bone marrow failure disease characterized by pancytopenia and a hypocellular bone marrow.
- Trial name
- OSP (NCT02203396)
- Testing
- rabbit ATG, Cyclosporine, Levamisole
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemoglobinopathy (Disorder), Severe Aplastic Anemia
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition.
- Conditions
- Hemoglobinopathy (Disorder), Severe Aplastic Anemia, Bone Marrow Failure Syndrome
- Trial name
- NCT04356469
- Testing
- Haploidentical Hematopoietic Cell Transplantation
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Johns Hopkins All Children's Hospital
Aplastic Anemia
Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Mesenchymal stem cells have been tested in many autoimmune disorders with encouraging results and may be an alternative to the treatment of immune-mediated severe acquired aplastic anemia.
- Trial name
- NCT01297972
- Testing
- Intravenous bone marrow mesenchymal stem cells infusion
- Where
- Brazil
- Volunteers
- 9 people
- Sponsor
- University of Sao Paulo
Severe Aplastic Anemia (SAA)
REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy
This study is researching an experimental drug called REGN7257 (called "study drug"). The study is focused on patients who have severe aplastic anemia (SAA), a disease of the bone marrow resulting in an impairment of the…
- Trial name
- NCT04409080
- Testing
- REGN7257
- Where
- 10 sites in United States, France and 2 more
- Volunteers
- 17 people
- Sponsor
- Regeneron Pharmaceuticals