Clinical trials: Aplastic Anaemia
Severe Aplastic Anemia
Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients
Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia.The theoretical basis for marrow failure includes primary defects in or damage to the stem cell or the marrow…
- Trial name
- NCT03295058
- Testing
- Non ATG Conditioning regimen, GVHD Prophylaxis, Allogenic Stem Cell Transplantation
- Volunteers
- 50 people
- Sponsor
- Assiut University
Transfusional Iron Overload, Transfusional Hemosiderosis
Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions. The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a…
- Trial name
- CORDELIA (NCT00600938)
- Testing
- Core Study: Deferasirox, Core Study: Deferoxamine, Extension: deferoxamine to deferasirox
- Where
- 21 sites in Canada, China and 9 more
- Volunteers
- 197 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia, Hypoplastic Myelodysplastic Syndrome
Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
An open label single arm study to assess efficacy and safety of BL-8040 on top of standard immunotherapy regimen of hATG, cyclosporine and steroids in patients with Hypoplastic MDS and AA over the course of a six month (180 day)…
- Trial name
- NCT02462252
- Testing
- BL-8040, horse anti-thymocyte globulin (hATG), Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- BioLineRx, Ltd.
Anemia, Aplastic, Anemia, Hypoplastic
Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment
Background: Severe plastic anemia can lead to problems with bone marrow platelet production and result in low blood platelet counts, which require frequent platelet transfusions to improve blood clotting.
- Trial name
- NCT00944749
- Testing
- h-ATG (ATGAM ), Cyclosporine (Gengraf )
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Lymphoblastic Leukemia, Cancer Survivors
Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors.
- Conditions
- Lymphoblastic Leukemia, Cancer Survivors, Aplastic Anemia, Myelodysplastic Syndromes
- Trial name
- IAMFIT (NCT05194397)
- Testing
- Nicotinamide Riboside, Exercise Intervention
- Where
- 3 sites in United States
- Volunteers
- 80 people
- Sponsor
- Children's Hospital of Philadelphia
Aplastic Anemia
Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA
This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of relapsed/refractory chemoradiotherapy-induced aplastic anemia
- Trial name
- NCT07532408
- Testing
- Romiplostim N01
- Sponsor
- Peking Union Medical College 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
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myeloproliferative Disorders, Kidney Cancer
Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic Anemia
RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant using stem cells that closely match the patient's stem cells, helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Kidney Cancer, Leukemia, Lymphoma
- Trial name
- NCT00295997
- Testing
- anti-thymocyte globulin, filgrastim, graft-versus-tumor induction therapy
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Aplastic Anemia
Autologous Tregs for Aplastic Anaemia
This Phase I study will determine the safety and optimal dose of expanded autologous Tregs to treat patients with Aplastic Anaemia (AA) (who have failed, or are considered ineligible for IST (immunosuppressive therapy) / other…
- Trial name
- TIARA (NCT05386264)
- Testing
- Expanded autologous T regulatory cells
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- King's College Hospital NHS Trust
Complications of Organ Transplant Stem Cells Umbilical Cord
Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）
Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment.
- Trial name
- MSC-SAA (NCT02218437)
- Testing
- MSC+ATG
- Where
- China
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Chinese Academy of Medical Sciences
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Aplastic Anemia, Neutropenia
Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
Background: Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions.
- Conditions
- Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
- Trial name
- NCT01193283
- Testing
- Cyclophosphamide, Cyclosporine
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Aplastic Anemia
Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia
Severe aplastic anemia (SAA)is characterized by the depletion of hematopoietic precursors associated with life-threatening complications.
- Trial name
- NCT01995331
- Testing
- cyclophosphamide,cyclosporine A
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Xiaofan Zhu
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
Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Dyskeratosis congenita is a disease that affects numerous parts of the body, most typically causing failure of the blood system. Lung disease, liver disease and cancer are other frequent causes of illness and death.
- Conditions
- Dyskeratosis Congenita, Hoyeraal Hreidarsson Syndrome, Revesz Syndrome, Aplastic Anemia
- Trial name
- NCT01659606
- Testing
- alemtuzumab, Fludarabine, Cyclosporins
- Where
- 13 sites in United States, Norway and 1 more
- Volunteers
- 40 people
- Sponsor
- Boston Children's Hospital
Diamond-Blackfan Anemia, Kostmann's Neutropenia
Stem Cell Transplant for Bone Marrow Failure Syndromes
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for…
- Conditions
- Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
- Trial name
- NCT00176878
- Testing
- Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Fanconi Anemia
Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia
RATIONALE: Giving low doses of chemotherapy, such as cyclophosphamide, before a donor bone marrow transplant helps stop the growth of abnormal cells.
- Trial name
- NCT00317876
- Testing
- cyclophosphamide, cyclosporine, methotrexate
- Where
- 3 sites in United States, Brazil
- Volunteers
- 25 people
- Sponsor
- Fred Hutchinson Cancer Center
Pure Red Cell Aplasia, Acquired
Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA
Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased.
- Trial name
- PRCA (NCT04470804)
- Testing
- Sirolimus, Cyclosporine A
- Where
- China
- Volunteers
- 56 people
- Sponsor
- Bing Han