Clinical Trials on Congenital Aplastic Anemia

Total 12620 results

National Heart, Lung, and Blood Institute (NHLBI)

Completed

Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia

Anemia, Aplastic | Red-Cell Aplasia, Pure | Anemia, Diamond-Blackfan

United States
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure

Aplastic Anemia | Pure Red Cell Aplasia | Diamond Blackfan Anemia

United States
European Society for Blood and Marrow Transplantation
CHUGAI sanofi-aventis

Terminated

Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) (SAA-G-CSF)

Aplastic Anaemia

France, Netherlands, Germany, United Kingdom, Switzerland, Italy, Sweden, Greece, Czechia
Northwell Health

Completed

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia | Blackfan Diamond Syndrome | DBA | Congenital Hypoplastic Anemia

United States
Jiangsu HengRui Medicine Co., Ltd.

Completed

A Bioequivalence Study of Hetrombopag in Healthy Subjects

Sever Aplastic Anaemia

China
Jiangsu HengRui Medicine Co., Ltd.

Completed

A Trial of Hetrombopag in Healthy and Hepatic Impairment Subjects

Sever Aplastic Anaemia

China
King Faisal Specialist Hospital & Research Center

Completed

Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia

Acquired Aplastic Anaemia
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS) | Severe Aplastic Anemia | Hypo-Plastic MDS

United States
Jiangsu HengRui Medicine Co., Ltd.

Completed

A Trial of Hetrombopag in Healthy Subjects

Sever Aplastic Anaemia

China
Adrianna Vlachos, MD

Terminated

The Use of Trifluoperazine in Transfusion Dependent DBA (DBA)

Pure Red Cell Aplasia | Diamond Blackfan Anemia

United States
St. Jude Children's Research Hospital

Completed

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Anemia, Aplastic | Diamond-Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann Syndrome

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy

Severe Aplastic Anemia

United States
David Rizzieri, MD

Completed

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Sickle Cell Anemia | Pure Red Cell Aplasia | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Severe Aplastic Anemia

United States
National University Hospital, Singapore

Unknown

Study of Non-Myeloablative Haplo-identical Haematopoietic Stem Cell Transplantation in Patients With Haematological Malignancies or Acquired Aplastic Anaemia

Haematological Malignancies | Acquired Aplastic Anaemia

Singapore
Northwell Health

Terminated

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

Diamond Blackfan Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia

Diamond Blackfan Anemia

United States
Mitchell Cairo

Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin Lymphoma | Sickle Cell Disease | Acute Leukemia | Beta-Thalassemia | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Diamond Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann

United States
Federal Scientific Clinical Centre of Pediatric...

Unknown

L-leucine in Diamond Blackfan Anemia Patients

Diamond Blackfan Anemia

Russian Federation
Feinstein Institute for Medical Research
Diamond Blackfan Anemia Foundation, Inc.

Recruiting

Diamond Blackfan Anemia Registry (DBAR) (DBAR)

Anemia | Blood Disease

United States
Stanford University
University of Alabama at Birmingham; University of Minnesota

Completed

Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag

Anemia, Diamond-Blackfan

United States
Singapore General Hospital
Shanghai Yueyang Integrated Medicine Hospital; Singapore Bao Zhong Tang TCM...

Completed

Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders

Myelodysplastic Syndrome (MDS) | Myelofibrosis (MF) | Thalassemia Intermedia | Aplastic Anaemia (AA)

Singapore
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Up-front Matched Unrelated Donor Transplantation in Pediatric Patients With Idiopathic Aplastic Anemia (UPFRONT-MUD)

Severe Aplastic Anemia | Idiopathic Aplastic Anemia | Moderate Aplastic Anemia Requiring Transfusions
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

Steroid-refractory Diamond-Blackfan Anemia (DBA)

United States
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Pilot Study (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United Kingdom, United States, Germany
Paul Szabolcs

Recruiting

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Sickle Cell Anemia | Diamond-blackfan Anemia | Beta-thalassemia Major

United States
University of Utah
Novartis

Completed

Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

Severe Aplastic Anemia | Moderate Aplastic Anemia | Very Severe Aplastic Anemia

United States
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Aplastic Anemia | Dyskeratosis Congenita

United States
Masonic Cancer Center, University of Minnesota

Completed

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

Aplastic Anemia | Dyskeratosis Congenita

United States
Novartis

Completed

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Myelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare Anaemias

Australia
City of Hope Medical Center
National Cancer Institute (NCI)

Recruiting

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

Recurrent Severe Aplastic Anemia | Refractory Severe Aplastic Anemia

United States
Peking University People's Hospital

Recruiting

The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA

Severe Aplastic Anemia

China
Boston Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health... and other collaborators

Recruiting

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (TransIT)

Severe Aplastic Anemia

United States
Federal Research Institute of Pediatric Hematology...

Recruiting

Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA

Acquired Aplastic Anemia

Russian Federation
Shanghai General Hospital, Shanghai Jiao Tong University...
Ruijin Hospital; Xinhua Hospital, Shanghai Jiao Tong University School of Medicine and other collaborators

Completed

The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic Anemia

Severe Aplastic Anemia

China
Nagoya University

Unknown

Randomised Study Comparing Different Dosages of Rabbit ATG in Patients With SAA

Acquired Aplastic Anemia.

Japan
Navy General Hospital, Beijing
Peking Union Medical College Hospital; Cancer Institute and Hospital, Chinese... and other collaborators

Unknown

Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia

Severe Aplastic Anemia

China
Jiangsu HengRui Medicine Co., Ltd.

Recruiting

Safety and Effectiveness of Trappa Ethanolamine Tablets Combined With Ciclosporin in the Treatment of Primary Treatment of Non-severe Aplastic Anemia

Non-severe Aplastic Anemia

China
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Comparing Therapies for the Treatment of Severe Aplastic Anemia

Severe Aplastic Anemia (SAA)

United States
National Institutes of Health Clinical Center (CC)
National Heart, Lung, and Blood Institute (NHLBI)

Completed

A Description of Bacteria in the Mouths of Patients With Severe Aplastic Anemia

Severe Aplastic Anemia (SAA)

United States
UCSF Benioff Children's Hospital Oakland
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Medical... and other collaborators

Completed

Multi-Center Study of Iron Overload: Survey Study (MCSIO) (MCSIO)

Sickle Cell Disease | Thalassemia | Diamond-Blackfan Anemia

United States, United Kingdom, Germany
Institute of Hematology & Blood Diseases Hospital

Recruiting

Cyclophosphamide Added to Standard Immunosuppressive Therapy With Eltrombopag as Front-line Therapy in Patients With Severe Aplastic Anemia

Severe Aplastic Anemia

China
Institute of Hematology & Blood Diseases Hospital

Recruiting

Avatrombopag Combined With IST as First-line Treatment for SAA

Severe Aplastic Anemia

China
Institute of Hematology & Blood Diseases Hospital

Recruiting

A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency

Severe Aplastic Anemia

China
Institute of Hematology & Blood Diseases Hospital
Peking University People's Hospital; Xiyuan Hospital of China Academy of Chinese... and other collaborators

Recruiting

The Safety and Efficacy Study of Avatrombopag Switch in TPO-RA Refractory AA

Refractory Aplastic Anemia

China
Institute of Hematology & Blood Diseases Hospital

Recruiting

Efficacy and Safety of CSA and Avatrombopag for the Treatment of SAA in the Elderly (SAA)

Severe Aplastic Anemia

China
Novartis Pharmaceuticals

Completed

Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia (SOAR)

Severe Aplastic Anemia

Spain, Italy, Hong Kong, Thailand, Korea, Republic of, Mexico, Turkey, India, Brazil
Michael Pulsipher, MD

Active, not recruiting

Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia

Severe Aplastic Anemia

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy

Severe Aplastic Anemia

United States
Institute of Hematology & Blood Diseases Hospital

Terminated

Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (LODACA)

Refractory Aplastic Anemia

China

Clinical Trials on Congenital Aplastic Anemia

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Iran

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations