Clinical trials: Aplastic Anaemia
Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria
Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria is an acquired chronic hemolytic anemia,this study is designed to evaluate the safety and efficacy of Levamisole combined with cyclosporine A in patients with classic paroxysmal nocturnal…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, Hemoglobinuria, Hemoglobinuria, Paroxysmal, Proteinuria
- Trial name
- PNH-2012 (NCT01642979)
- Testing
- Levamisole+cyclosporin A+Glucocorticoids, cyclosporin A+Glucocorticoids, Glucocorticoids
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
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
Thrombocytopenia, Hematologic Malignancy
Thrombosomes® in Bleeding Thrombocytopenic Patients Study
This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate…
- Conditions
- Thrombocytopenia, Hematologic Malignancy, Bone Marrow Aplasia, Myeloproliferative Disorders
- Trial name
- NCT04631211
- Testing
- Thrombosomes, Liquid Stored Platelets (LSP)
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 21 people
- Sponsor
- Cellphire Therapeutics, Inc.
Pure Red-cell Aplasia
A Study of Risk Factors for Anti-erythropoietin Antibody Positive Pure Red Cell Aplasia Among Patients With Chronic Kidney Disease Receiving Epoetin Alfa
The purpose of this study is to collect historical occurrences of risk factors that are potentially associated with the development of anti-erythropoietin (EPO) antibody positive pure red cell aplasia (PRCA) in participants with…
- Trial name
- NCT00211068
- Testing
- No intervention
- Where
- 22 sites in Brazil, France and 5 more
- Volunteers
- 124 people
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Severe Aplastic Anemia (SAA)
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to…
- Trial name
- NCT05323617
- Testing
- Romiplostim, Antithymocyte Globulin, Cyclosporine A
- Sponsor
- Amgen
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
Immune Thrombocytopenia, Chemotherapy Induced Thrombocytopenia
Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative Thrombocytopenias.
To utilise extended platelet parameters in order to individuate Immune Thrombocytopenia (ITP) from hypo-proliferative causes of thrombocytopenia.
- Conditions
- Immune Thrombocytopenia, Chemotherapy Induced Thrombocytopenia, Myelodysplasia, Aplastic Anaemia
- Trial name
- NCT01933035
- Testing
- Immune Thrombocytopenics, Hypo-proliferative thrombocytopenics, Control Population
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Beth Israel Medical Center
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
Aplastic Anemia
GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in SAA With Treatment of Allo-HSCT
Objectives 2.1 Primary objectives 1) To observe and compare incidence and severity of aGVHD and cGVHD between the two arms within 2 years after transplantation. 2) To observe and compare the engraftment rate between the two arms.
- Trial name
- NCT05510505
- Testing
- CD20 monoclonal antibody, ATG
- Where
- China
- Volunteers
- 100 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Aplastic Anemia
Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may improve bone marrow function and increase blood cell counts.
- Trial name
- NCT01231841
- Testing
- cyclosporine, anti-thymocyte globulin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The Cleveland Clinic
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia
- Trial name
- NCT02566304
- Testing
- Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Myelodysplastic Syndrome, Aplastic Anemia
Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM or NeulastaTM ) can help to control severe aplastic anemia (AA) or hypoplastic…
- Trial name
- NCT00806598
- Testing
- Thymoglobulin, Cyclosporine, Methylprednisolone
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- M.D. Anderson Cancer 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
Aplastic Anemia Idiopathic
Outcomes in Bone Marrow Aplasia.
Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and…
- Trial name
- NCT06493981
- Testing
- Eltrombopag
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Assiut University
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Sargramostim in Reducing Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplantation for Hematologic Cancer or Aplastic Anemia
RATIONALE: Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy or…
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00053157
- Testing
- sargramostim
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Roswell Park Cancer Institute
Myelodysplastic Syndrome (MDS) With Refractory Anemia (RA), Severe Aplastic Anemia (SAA)
Stem Cell Transplant Using Peripheral and Cord Blood Stem Cells to Treat Severe Aplastic Anemia and Myelodysplastic Syndrome
This study will evaluate the safety and effectiveness of treating patients with severe aplastic anemia (SAA) or myelodysplastic syndrome (MDS) with both peripheral blood stem cells from a family member and umbilical cord blood…
- Trial name
- NCT00604201
- Testing
- Umbilical Cord Blood
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Fanconi Anemia, Head and Neck Squamous Cell Carcinoma
Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
This research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC…
- Trial name
- AFAN (NCT06648096)
- Testing
- Afatinib
- Where
- 2 sites in Germany, Spain
- Volunteers
- 25 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Fanconi Anemia
TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
- Trial name
- NCT00352976
- Testing
- Cyclophosphamide, Fludarabine, Total Body Irradiation
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease, Thalassemia
Stem Cell Transplant for Hemoglobinopathy
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia
- Trial name
- NCT00176852
- Testing
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota