Not applicableEnded earlyNCT04275479What this trial is testingDiabetes/ Endocrine Surveillance in SDSWho this might be right forShwachman-Diamond Syndrome Washington University School of Medicine 11
Testing effectiveness (Phase 2)Active Not RecruitingNCT02061800What this trial is testingCD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell TransplantWho this might be right forChronic Myeloid Leukemia (CML)Acute Myelogenous Leukemia (AML)Myelodysplastic Syndrome (MDS)+3 more Diane George 14
Large-scale testing (Phase 3)Looking for participantsNCT06056297What this trial is testingMavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsWho this might be right forNeutropenia X4 Pharmaceuticals 176
Not applicableEnded earlyNCT02179359What this trial is testingHematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesWho this might be right forSickle Cell DiseaseTransfusion Dependent Alpha- or Beta- ThalassemiaDiamond Blackfan Anemia+5 more Masonic Cancer Center, University of Minnesota 38
Not applicableStudy completedNCT00499070What this trial is testingAssessing Immune Function in Young Patients With Cytopenia That Did Not Respond to TreatmentWho this might be right forDyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+2 more University Hospital Freiburg 119
Not applicableLooking for participantsNCT06056908What this trial is testingShwachman Diamond Syndrome Registry and StudyWho this might be right forShwachman-Diamond SyndromeShwachman-Diamond Syndrome-Like Boston Children's Hospital 5,000
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)Study completedNCT00919503What this trial is testingTreosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited DisordersWho this might be right forNon-Neoplastic Hematologic and Lymphocytic Disorder Fred Hutchinson Cancer Center 98
Large-scale testing (Phase 3)Study completedNCT00176852What this trial is testingStem Cell Transplant for HemoglobinopathyWho this might be right forSickle Cell DiseaseThalassemiaSevere Congenital Neutropenia+2 more Masonic Cancer Center, University of Minnesota 22
Early research (Phase 1)Ended earlyNCT02231710What this trial is testingSafety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesWho this might be right forPrimary Immune Deficiency DisordersHemophagocytic LymphohistiocytosisInherited Bone Marrow Failure Syndrome+2 more Bellicum Pharmaceuticals 1
Testing effectiveness (Phase 2)Study completedNCT00004787What this trial is testingPhase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure SyndromesWho this might be right forShwachman SyndromeFanconi's AnemiaDyskeratosis Congenita+1 more National Center for Research Resources (NCRR) 20
Early research (Phase 1)Study completedNCT01917708What this trial is testingBone Marrow Transplant With Abatacept for Non-Malignant DiseasesWho this might be right forHurler SyndromeFanconi AnemiaGlanzmann Thrombasthenia+12 more Emory University 10
Testing effectiveness (Phase 2)Active Not RecruitingNCT04965597What this trial is testingTreosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)Who this might be right forBone Marrow Failure SyndromeCongenital Amegakaryocytic ThrombocytopeniaDiamond-Blackfan Anemia+6 more Fred Hutchinson Cancer Center 40
Not applicableStudy completedNCT02538653What this trial is testingDetermination of the Postprandial Responses to Two Cereal Foods Differing by Their SDS Content Consumed Alone.Who this might be right forMetabolic Disorder; Carbohydrate, Absorption, Intestine Mondelēz International, Inc. 20
Not applicableLooking for participantsNCT00027274What this trial is testingCancer in Inherited Bone Marrow Failure SyndromesWho this might be right forDiamond Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more National Cancer Institute (NCI) 4,000
Not applicableLooking for participantsNCT06999954What this trial is testingShwachman-Diamond Syndrome Global Patient Survey and Partnering PlatformWho this might be right forShwachman-Diamond SyndromeSDSIBMF+24 more Shwachman-Diamond Syndrome Alliance Inc 8,000
Testing effectiveness (Phase 2)Ended earlyNCT03333486What this trial is testingFludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood CancerWho this might be right forAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 PositiveAcute Leukemia in RemissionAcute Lymphoblastic Leukemia+34 more Roswell Park Cancer Institute 31
Large-scale testing (Phase 3)Study completedNCT00176878What this trial is testingStem Cell Transplant for Bone Marrow Failure SyndromesWho this might be right forDiamond-Blackfan AnemiaKostmann's NeutropeniaShwachman-Diamond Syndrome Masonic Cancer Center, University of Minnesota 10
Testing effectiveness (Phase 2)Active Not RecruitingNCT01966367What this trial is testingCD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationWho this might be right forBone Marrow Failure SyndromeSevere Aplastic AnemiaSevere Congenital Neutropenia+13 more Diane George 37
Not applicableEnded earlyNCT01319851What this trial is testingAlefacept and Allogeneic Hematopoietic Stem Cell TransplantationWho this might be right forThalassemiaSickle Cell DiseaseGlanzmann Thrombasthenia+10 more Emory University 3