7 completed, 2 terminated, 1 active not recruiting, 1 recruiting. 6 Phase 2, 4 Phase 1, 2 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 6 | Completed 4, Active not recruiting 1, Unknown 1 |
| Phase 1 | 4 | Completed 3, Active not recruiting 1 |
| Not applicable | 2 | Terminated 2 |
| Phase 3 | 1 | Completed 1 |
Follow Severe Congenital Neutropenia: new trials by email Literature Review of Severe Congenital Neutropenia trials Research Report on Severe Congenital Neutropenia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Total Lymphoid Irradiation Pre-HSCT in Severe Congenital Neutropenia conditioning with TLI - Severe Congenital Neutropenia, GATA2 Deficiency | Phase 2 | Unknown | Federal Research Institute of Pediatric Hematology, Oncology and Immunology | 10 | 2021-04-14 | 2021-04-14 |
| Investigation of the Genetics of Hematologic Diseases Bone Marrow Failure Syndromes, Erythrocyte Disorder | Recruiting | St. Jude Children's Research Hospital | 1716 | 2016-06-17 | 2026-06-09 | |
| Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen - Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia | Not applicable | Terminated | Masonic Cancer Center, University of Minnesota | 38 | 2014-09-02 | 2025-03-03 |
| Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Abatacept - Hurler Syndrome, Fanconi Anemia | Phase 1 | Completed | Emory University | 10 | 2014-01-01 | 2019-12-26 |
| CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation CD34 Stem Cell Selection Therapy - Bone Marrow Failure Syndrome, Severe Aplastic Anemia | Phase 1 / Phase 2 | Active not recruiting | Diane George | 37 | 2013-03-01 | 2025-08-14 |
| Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies fludarabine phosphate, melphalan, total-body irradiation - Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission | Phase 2 | Completed | Roswell Park Cancer Institute | 94 | 2012-02-28 | 2019-09-24 |
| Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation Alefacept - Thalassemia, Sickle Cell Disease | Not applicable | Terminated | Emory University | 3 | 2010-09-01 | 2017-07-27 |
| Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease anti-thymocyte globulin, therapeutic allogeneic lymphocytes, fludarabine phosphate - Congenital Amegakaryocytic Thrombocytopenia, Leukemia | Phase 1 | Completed | University of California, San Francisco | 21 | 2005-04-01 | 2012-11-12 |
| Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders alemtuzumab, busulfan, cyclosporine - Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia | Phase 2 | Completed | University of California, San Francisco | 35 | 2005-01-01 | 2017-09-28 |
| Stem Cell Transplant for Hemoglobinopathy Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide - Sickle Cell Disease, Thalassemia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 22 | 2002-06-01 | 2020-02-27 |
| Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission anti-thymocyte globulin, busulfan, fludarabine phosphate - Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia | Phase 1 / Phase 2 | Completed | University of California, San Francisco | 40 | 2000-08-01 | 2012-11-12 |
Neutropenia, Severe Congenital, Autosomal Recessive 3 (12) · Anemia, Diamond-Blackfan (10) · Anemia, Sickle Cell (7) · Shwachman-Diamond Syndrome (7) · Fanconi Anemia (5) · Anemia, Aplastic (4) · Congenital amegakaryocytic thrombocytopenia (4) · Leukemia, Myeloid, Chronic-Phase (4) · Wiskott-Aldrich Syndrome (4) · Chediak-Higashi Syndrome (3) · Dyskeratosis Congenita (3) · Granulomatous Disease, Chronic (3) · Leukemia (3) · Lymphohistiocytosis, Hemophagocytic (3) · Myelodysplastic Syndromes (3)
Bone Marrow Diseases (12) · Hematologic Diseases (12) · Hemic and Lymphatic Diseases (12) · Anemia (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Genetic Diseases, Inborn (11) · Anemia, Hypoplastic, Congenital (10) · Congenital Bone Marrow Failure Syndromes (10) · Red-Cell Aplasia, Pure (10) · Metabolic Diseases (9) · Nutritional and Metabolic Diseases (9) · Anemia, Hemolytic (8) · Bone Marrow Failure Disorders (8) · Anemia, Hemolytic, Congenital (7) · Chronic Disease (7)
fludarabine phosphate (5) · Antilymphocyte Serum (3) · Busulfan (3) · Peripheral Blood Stem Cell Transplantation (3) · Whole-Body Irradiation (3) · Alemtuzumab (2) · Cord Blood Stem Cell Transplantation (2) · Methotrexate (2) · Abatacept (1) · Alefacept (1) · Bone Marrow Transplantation (1) · Cyclophosphamide (1) · Cyclosporine (1) · Granulocyte Colony-Stimulating Factor (1) · Melphalan (1)
University of California, San Francisco (3) · Emory University (2) · Masonic Cancer Center, University of Minnesota (2) · Centre Hospitalier Universitaire de Liege (1) · Diane George (1) · Federal Research Institute of Pediatric Hematology, Oncology and Immunology (1) · Roswell Park Cancer Institute (1) · St. Jude Children's Research Hospital (1)
United States (10) · Belgium (1) · Russia (1)
Morton J. Cowan, MD (3) · Angela Smith, MD (1) · Ashish Gupta, MBBS, MPH (1) · Diane George, MD (1) · George Chen (1) · John Horan, MD (1) · John T Horan, MD (1) · Laurent Servais (1) · Marcin Wlodarski, MD, PhD (1)