Clinical trials: Aplastic Anaemia
Aplastic Anemia
Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia
Aplastic anemia (AA) is a rare bone marrow failure disease characterized by bone marrow hypocellularity and peripheral blood pancytopenia.
- Trial name
- NCT05399732
- Testing
- Luspatercept, Cyclosporine
- Where
- China
- Volunteers
- 58 people
- Sponsor
- Bing Han
Neutropenia, Sickle Cell Anemia
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II.
- Conditions
- Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
- Trial name
- NCT00005893
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Shwachman Syndrome, Fanconi's Anemia
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure…
- Conditions
- Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, Thrombocytopenia
- Trial name
- NCT00004787
- Testing
- filgrastim
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Aplastic Anaemia
Conditioning Regimen Containing Low-Dose ATG for the Treatment of AA 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 Aplastic Anemia (AA)
- Trial name
- NCT07885982
- Testing
- Anti-Thymocyte Globulin (Rabbit) (Thymoglobulin®)
- Where
- China
- Volunteers
- 59 people
- Sponsor
- Anhui Provincial Hospital
Hematological Malignancies
Safety and Efficacy of Obatoclax Mesylate (GX15-070MS) for the Treatment of Hematological Malignancies
Defects in the apoptotic process can lead to the onset of cancer by allowing cells to grow unchecked when an oncogneic signal is present.
- Trial name
- NCT00438178
- Testing
- Obatoclax mesylate (GX15-070MS)
- Where
- 3 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Gemin X
Severe Aplastic Anemia
Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia
The purpose of this study is to evaluate the effectiveness of allogeneic transplant after a reduced-intensity preparative regimen for patient, to evaluate survival, and to evaluate the side effects of this treatment.
- Trial name
- NCT01129323
- Testing
- Cyclophosphamide, Fludarabine, Rabbit ATG
- Sponsor
- City of Hope Medical Center
Severe Aplastic Anemia, Refractory Aplastic Anemia
Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia
This study is designed as a Phase Ib/II trial. The phase Ib cohort will enroll patients with severe aplastic anemia (SAA) who have failed to respond to intensified immunosuppressive therapy.
- Conditions
- Severe Aplastic Anemia, Refractory Aplastic Anemia, Newly Diagnosed Aplastic Anemia
- Trial name
- NCT07297550
- Testing
- Golidocitinib Capsules
- Volunteers
- 42 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
Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia.
The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of bone marrow derived mesenchymal stem cells from related donor to patients with relapsed/refractory aplastic anemia.
- Trial name
- MSC (NCT01305694)
- Testing
- bone marrow derived mesenchymal stem cells
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Guangzhou General Hospital of Guangzhou Military Command
Hereditary Diseases, Acquired Aplastic Anemia
Cord Blood Transplantation in Severe Aplastic Anemia
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;
- Conditions
- Hereditary Diseases, Acquired Aplastic Anemia, Relapse, Absence of an HLA Identical Donor
- Trial name
- APCORD (NCT01343953)
- Testing
- Cord Blood Transplantation
- Where
- France
- Volunteers
- 26 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sever Aplastic Anaemia
A Trial of Hetrombopag in Healthy and Hepatic Impairment Subjects
This is a single-dose, open-label, phase I clinical study evaluating the PK of hetrombopag in subjects with mild hepatic impairment (Child-Pugh Class A), subjects with moderate hepatic impairment (Child-Pugh Class B), as well as…
- Trial name
- NCT05088642
- Testing
- Hetrombopag Olamine Tablet
- Where
- China
- Volunteers
- 24 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Blood Disease, Anemia, Aplastic
Aplastic Anemia Epidemiology: Incidence and Case-control
To conduct incidence and case-control studies of aplastic anemia in metropolitan Bangkok, Thailand
- Trial name
- NCT00005682
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Leukemia, Myeloid, Chronic, Anemia, Aplastic
Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor
The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to…
- Conditions
- Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis, Lymphoma
- Trial name
- NCT00393380
- Testing
- Parathyroid Hormone (teriparatide)
- Where
- 4 sites in United States
- Volunteers
- 13 people
- Sponsor
- The Emmes Company, LLC
Hematologic Malignancies, Nonmalignant Diseases
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609827
- Testing
- Melphalan
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Aplastic Anemia
Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission
This study will examine 1) whether it is possible to collect enough stem cells (cells produced by the bone marrow that mature into white and red blood cells and platelets) from patients with aplastic anemia to use for future…
- Trial name
- NCT00011830
- Testing
- G-CSF, Apheresis
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia
This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia
- Trial name
- NCT06633328
- Testing
- CD7 CAR-T cells injection, Allogeneic hematopoietic stem cell transplantation
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Zhejiang University
Anemia, Aplastic, Blood Disease
Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study Data
To quantify the role of drugs and other factors in the etiology of agranulocytosis and aplastic anemia.
- Conditions
- Anemia, Aplastic, Blood Disease, Agranulocytosis
- Trial name
- NCT00005307
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA)…
- Trial name
- NCT07523009
- Testing
- Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01
- Sponsor
- Peking Union Medical College Hospital