Clinical trials: Severe Aplastic Anemia
Severe Aplastic Anemia
Avatrombopag Combined With IST as First-line Treatment for SAA
This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).
- Trial name
- NCT05720234
- Testing
- avatrombopag
- Where
- China
- Volunteers
- 53 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Anemia, Aplastic
Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia
Efficacy and Safety of Recombinant human thrombopoietin in patients with severe aplastic anemia and very severe aplastic anemia, a randomized, double-blind, placebo-controlled, II phase, multi-center clinical research.
- Trial name
- NCT02857530
- Testing
- rhTPO
- Where
- China
- Volunteers
- 250 people
- Sponsor
- Tianjin Medical University General Hospital
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
Severe Aplastic Anemia
Haploidentical Transplantation in Severe Aplastic Anemia
This is a prospective case-control study on SAA patients treated with HSCT, order to further discuss and assess the safety, feasibility and effectiveness of HFD-HSCT which performed with reduced-intensity fludarabine-based…
- Trial name
- NCT03246178
- Testing
- MSD-HSCT, HFD-HSCT
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Wu Xiaoxiong
Severe Aplastic Anemia
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding.
- Trial name
- NCT02828592
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Northside Hospital, Inc.
Aplastic Anaemia, Transplantation Conditioning
Modified Transplantation Regimen and aGVHD Prophylaxis for Severe Aplastic Anemia in the Setting of Allogeneic HSCT.
The investigators retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in 4 transplantation centers.
- Trial name
- aGVHD (NCT06837987)
- Testing
- Modified transplantation system
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Hematology department of the 920th hospital
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Aplastic Anemia, Bone Marrow Failure Syndrome
Haploidentical HCT for Severe Aplastic Anemia
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation.
- Trial name
- NCT04558736
- Testing
- Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- St. Jude Children's Research Hospital
Dyskeratosis Congenita, Aplastic Anemia
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant…
- Trial name
- NCT00455312
- Testing
- Campath 1H, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Severe Aplastic Anemia, Refractory, Severe Aplastic Anemia, Relapse
Alemtuzumab to Treat Severe Aplastic Anemia
This study will evaluate the safety and usefulness of a new immunosuppressive drug, alemtuzumab (Campath ), in patients with severe aplastic anemia (SAA).
- Trial name
- NCT00195624
- Testing
- Alemtuzumab (Campath ), Cyclosporine
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Non-severe Aplastic Anemia
Safety and Effectiveness of Trappa Ethanolamine Tablets Combined With Ciclosporin in the Treatment of Primary Treatment of Non-severe Aplastic Anemia
This is a multicenter, randomized, double-blind, and open-label, placebo-controlled phase II clinical study. In order to evaluate the effectiveness and safety of trappa ethanolamine tablets combined with ciclosporin in the…
- Trial name
- NCT05797623
- Testing
- Trappa ethanolamine tablets ; ciclosporin
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Human Placental-Derived Stem Cell Transplantation
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Adrenoleukodystrophy, Niemann-Pick Disease
- Trial name
- HPDSC (NCT01586455)
- Testing
- Human Placental Derived Stem Cell
- Where
- 3 sites in United States
- Volunteers
- 43 people
- Sponsor
- New York Medical College
Acquired Aplastic Anemia.
Randomised Study Comparing Different Dosages of Rabbit ATG in Patients With SAA
The objective of this study is to evaluate the feasibility and effectiveness of immunosuppressive therapy (IST) using rabbit anti-thymocyte globulin (ATG) (Thymoglobuline, Genzyme) for patients with very severe aplastic anemia…
- Trial name
- NCT01844635
- Testing
- Thymoglobulin
- Where
- Japan
- Volunteers
- 320 people
- Sponsor
- Nagoya University