Clinical trials: Aplastic Anaemia
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective…
- Trial name
- ePRO-AA-PNH (NCT04128943)
- Testing
- symptom questionnaire, recording of vital signs, European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
- Where
- Switzerland
- Volunteers
- 9 people
- Sponsor
- University Hospital, Basel, Switzerland
Aplastic Anemia
Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea.
- Trial name
- NCT02773290
- Testing
- Romiplostim
- Where
- 3 sites in Japan, South Korea
- Volunteers
- 46 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Transfusion-dependent Non-severe Aplastic Anemia
Enarodustat+CsA vs CsA in the Treatment of Newly-diagnosed TD-NSAA
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine alone in the treatment of TD-NSAA.
- Trial name
- NCT07522996
- Testing
- Enarodustat, Cyclosporin (CSA)
- Sponsor
- Peking Union Medical College Hospital
Thrombocytopenia, Hematologic Diseases
Thrombosomes® in Bleeding Thrombocytopenic Patients
The study evaluates the safety and potential early signals of efficacy of allogeneic Thrombosomes in bleeding thrombocytopenic patients
- Conditions
- Thrombocytopenia, Hematologic Diseases, Bone Marrow Aplasia
- Trial name
- NCT03394755
- Testing
- Thrombosomes
- Where
- 7 sites in United States, Norway
- Volunteers
- 24 people
- Sponsor
- Cellphire Therapeutics, Inc.
Leukemia, Hodgkin's Lymphoma
Abdominal Obesity and Cardiovascular Risk Factors in Women Who Survived Cancer or a Related Illness Following Total Body Irradiation and Stem Cell Transplant
The purpose of this study is to better understand why some women who survived cancer or a related illness later develop diabetes, problems with their cholesterol, or other problems that may lead to heart disease.
- Conditions
- Leukemia, Hodgkin's Lymphoma, Non-Hodgkin's Lymphoma, Myelodysplastic Syndrome
- Trial name
- NCT00510315
- Testing
- Questionnaires, Laboratory tests, Abdominal MRI
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Aplastic Anemia
Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia
This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.
- Trial name
- NCT03055078
- Testing
- mesenchymal stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Hebei Medical University
Fanconi Anemia
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+…
- Trial name
- NCT00479115
- Testing
- AMD3100, AmCell CliniMACs
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Acquired Aplastic Anaemia
Clinico-Epidemiological Profile Of Acquired Aplastic Anemia Among Children At Assiut Governorate .
1. Assessment the clinical presentation and epidemiological profile of children with acquired aplastic anemia. 2. Determining the possible risk factors and associated conditions contributing to the development of acquired…
- Trial name
- NCT07259863
- Testing
- observational case series study no intervention
- Volunteers
- 35 people
- Sponsor
- Assiut University
Aplastic Anemia Idiopathic
A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia:
Severe and very severe aplastic anemia are life threatening disorders for which allogeneic stem cell transplant is only curative treatment. However, matched sibling donor (MSD) is available in only 25-35% cases.
- Trial name
- FluCAB-Prime (NCT03955601)
- Testing
- Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) +eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe…
- Trial name
- NCT07558434
- Testing
- Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Aplastic Anemia, CSA
Efficacy of IST Combined With TPO-RA in the Treatment of AA and Establishment of a Recurrence Prediction System
In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China.
- Conditions
- Aplastic Anemia, CSA, Drug Therapy
- Trial name
- NCT06009965
- Testing
- CsA+ATG+Herombopag, CsA+Herombopag
- Where
- China
- Volunteers
- 210 people
- Sponsor
- Peking Union Medical College Hospital
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
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match
The purpose of this study is to evaluate the multi-lineage hematopoietic chimerism for unrelated umbilical cord blood (UCB) grafts pooled from two to three cord blood units.
- Conditions
- Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia
- Trial name
- NCT01500161
- Testing
- Busulfan, Clofarabine, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Texas Oncology Cancer Center
Fanconi Anemia
Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia
The purpose of this research study is to evaluate the safety of the drug Etanercept (Enbrel) and to determine if this drug can help in the treatment of early bone marrow failure in patients with Fanconi anemia.
- Trial name
- NCT00965666
- Testing
- Etanercept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Haematological Malignancy, Acute Leukemia
Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes haemoglobinopathies, bone…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT05298930)
- Testing
- Physical activity program
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Centre Leon Berard
Fanconi Anemia
Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi…
- Trial name
- FANCOLEN-1 (NCT03157804)
- Testing
- IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs), Genetically Engineered Hematopoietic Stem/Progenitor Cells, Laboratory Biomarker Analysis
- Where
- 2 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Acquired Aplastic Anaemia
Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia
To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with…
- Trial name
- NCT01530555
- Testing
- Rabbit ATG, Thymoglobuline (Genzyme)
- Volunteers
- 35 people
- Sponsor
- King Faisal Specialist Hospital & Research Center
Fanconi Anemia
Quercetin in Children With Fanconi Anemia; a Pilot Study
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
- Trial name
- NCT01720147
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia. II.
- Trial name
- NCT00004464
- Testing
- cyclophosphamide, filgrastim
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Severe Aplastic Anemia
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive. 180 treated naive patients with SAA will be enrolled in the study.
- Trial name
- NCT03825744
- Testing
- Hetrombopag Olamine+Standard Therapy
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.