10 completed, 5 terminated, 4 recruiting, 1 active not recruiting. 9 Phase 2, 4 Phase 1, 3 Not applicable, 3 Phase 3.
Follow Shwachman-Diamond Syndrome: new trials by email Literature Review of Shwachman-Diamond Syndrome trials Research Report on Shwachman-Diamond Syndrome
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 |
|---|---|---|---|---|---|---|
| A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections Mavorixafor, Placebo - Neutropenia | Phase 3 | Recruiting | X4 Pharmaceuticals | 176 | 2024-06-06 | 2026-08-06 |
| Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform Shwachman-Diamond Syndrome, SDS | Recruiting | Shwachman-Diamond Syndrome Alliance Inc | 8000 | 2024-02-07 | 2025-05-31 | |
| 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 | |
| Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) Treosulfan, Fludarabine Phosphate, Tacrolimus - Bone Marrow Failure Syndrome, Congenital Amegakaryocytic Thrombocytopenia | Phase 2 | Completed | Fred Hutchinson Cancer Center | 40 | 2022-04-19 | 2026-03-09 |
| Diabetes/ Endocrine Surveillance in SDS Oral Glucose Tolerance Test, Modified Oral Glucose Tolerance Test, Modified Mixed Meal Tolerance Test - Shwachman-Diamond Syndrome | Terminated | Washington University School of Medicine | 11 | 2020-01-10 | 2024-08-19 | |
| Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis - Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission | Phase 2 | Terminated | Roswell Park Cancer Institute | 31 | 2017-12-07 | 2025-07-04 |
| Shwachman Diamond Syndrome Registry and Study Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like | Recruiting | Boston Children's Hospital | 5000 | 2016-01-19 | 2026-04-06 | |
| Determination of the Postprandial Responses to Two Cereal Foods Differing by Their SDS Content Consumed Alone. Sandwich biscuit high in SDS, Co-extruded cereal low in SDS - Metabolic Disorder; Carbohydrate, Absorption, Intestine | Not applicable | Completed | Mondelēz International, Inc. | 20 | 2015-09-01 | 2017-09-14 |
| Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases BPX-501 and Rimiducid - Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis | Phase 1 | Terminated | Bellicum Pharmaceuticals | 1 | 2015-02-01 | 2024-03-25 |
| 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+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide - Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML) | Phase 1 / Phase 2 | Unknown | Diane George | 14 | 2013-06-03 | 2024-06-04 |
| 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 |
| Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine - Non-Neoplastic Hematologic and Lymphocytic Disorder | Phase 2 | Completed | Fred Hutchinson Cancer Center | 98 | 2009-07-31 | 2021-08-13 |
| Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment polymerase chain reaction, flow cytometry, immunologic technique - Dyskeratosis Congenita, Fanconi Anemia | Completed | University Hospital Freiburg | 119 | 2007-01-01 | 2015-01-16 | |
| 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 |
| Cancer in Inherited Bone Marrow Failure Syndromes Diamond Blackfan Anemia, Dyskeratosis Congenita | Recruiting | National Cancer Institute (NCI) | 4000 | 2001-11-28 | 2026-10-16 | |
| Stem Cell Transplant for Bone Marrow Failure Syndromes Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation - Diamond-Blackfan Anemia, Kostmann's Neutropenia | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 10 | 2000-06-01 | 2017-12-28 |
Showing 20 of 21. Sign in to see all of them.
Anemia, Diamond-Blackfan (14) · Neutropenia, Severe Congenital, Autosomal Recessive 3 (11) · Anemia, Sickle Cell (10) · Anemia, Aplastic (9) · Dyskeratosis Congenita (7) · Fanconi Anemia (7) · Bone Marrow Failure Disorders (6) · Lymphohistiocytosis, Hemophagocytic (6) · Wiskott-Aldrich Syndrome (6) · Granulomatous Disease, Chronic (5) · Primary Immunodeficiency Diseases (5) · Thalassemia (5) · Congenital Bone Marrow Failure Syndromes (4) · Hemoglobinopathies (4)
Bone Marrow Diseases (21) · Digestive System Diseases (21) · Hemic and Lymphatic Diseases (21) · Lipid Metabolism Disorders (21) · Lipid Metabolism, Inborn Errors (21) · Lipomatosis (21) · Nutritional and Metabolic Diseases (21) · Pancreatic Diseases (21) · Hematologic Diseases (20) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (19) · Exocrine Pancreatic Insufficiency (19) · Genetic Diseases, Inborn (18) · Metabolic Diseases (18) · Congenital Bone Marrow Failure Syndromes (17) · Anemia (16)
fludarabine phosphate (7) · Antilymphocyte Serum (4) · Peripheral Blood Stem Cell Transplantation (4) · Tacrolimus (4) · Whole-Body Irradiation (4) · Busulfan (3) · Cyclophosphamide (3) · Methotrexate (3) · Alemtuzumab (2) · Bone Marrow Transplantation (2) · Melphalan (2) · Mycophenolic Acid (2) · fludarabine (2) · merphos (2) · thymoglobulin (2)
Masonic Cancer Center, University of Minnesota (3) · Diane George (2) · Emory University (2) · Fred Hutchinson Cancer Center (2) · Roswell Park Cancer Institute (2) · Bellicum Pharmaceuticals (1) · Boston Children's Hospital (1) · Centre Hospitalier Universitaire de Liege (1) · Mondelēz International, Inc. (1) · National Cancer Institute (NCI) (1) · National Center for Research Resources (NCRR) (1) · Shwachman-Diamond Syndrome Alliance Inc (1) · University Hospital Freiburg (1) · Washington University School of Medicine (1) · X4 Pharmaceuticals (1)
United States (17) · Belgium (3) · Australia (2) · Czechia (2) · Germany (2) · Italy (2) · Spain (2) · Switzerland (2) · Argentina (1) · Austria (1) · Canada (1) · Colombia (1) · Denmark (1) · France (1) · Georgia (1)
Diane George, MD (2) · Angela Smith, MD (1) · Ashish Gupta, MBBS, MPH (1) · David A. Williams (1) · Garry Tobin, MD (1) · George Chen (1) · Jennie Brand Miller (1) · John Horan, MD (1) · John T Horan, MD (1) · Laurent Servais (1) · Lauri Burroughs, MD (1) · Lauri M. Burroughs (1) · Lisa J McReynolds, M.D. (1) · Margaret MacMillan, MD (1)
PMID 24786393, PMID 25304614, PMID 25405500, PMID 25425683, PMID 25963299, PMID 26095669, PMID 28280134, PMID 31836590, PMID 32790018, PMID 33810313, PMID 34162668, PMID 34852175, PMID 35322185, PMID 35360980, PMID 37354000, PMID 39279436, PMID 40762130
2023 (1), P30CA015704 (1), U10HL069294 (1)