Not applicableLooking for participantsNCT01630421What this trial is testingGenetic and Functional Analysis of Aplasia Cutis Congenital (ACC)Who this might be right forAplasia Cutis Congenita UConn Health 600
Testing effectiveness (Phase 2)Active Not RecruitingNCT01659606What this trial is testingRadiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis CongenitaWho this might be right forDyskeratosis CongenitaHoyeraal Hreidarsson SyndromeRevesz Syndrome+1 more Boston Children's Hospital 40
Not applicableLooking for participantsNCT04528355What this trial is testingData Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more Paul Szabolcs 50
Not applicableUnknownNCT01470235What this trial is testingHypodontia and Ovarian CancerWho this might be right forOvarian CancerHypodontiaBreast Cancer+1 more University of Aarhus 200
Early research (Phase 1)Study completedNCT00692926What this trial is testingUnrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood CellsWho this might be right forMDSAnemia, AplasticInborn Errors of Metabolism+2 more Joanne Kurtzberg, MD 37
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 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)Ended earlyNCT01001598What this trial is testingSafety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaWho this might be right forFanconi AnemiaDyskeratosis Congenita Boston Children's Hospital 5
Not applicableStudy completedNCT02162420What this trial is testingHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaWho this might be right forDyskeratosis CongenitaAplastic Anemia Masonic Cancer Center, University of Minnesota 61
Testing effectiveness (Phase 2)Looking for participantsNCT04232085What this trial is testingRegenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow FailuresWho this might be right forPrimary Immune Deficiency DisorderImmune Deficiency DiseaseBone Marrow Failure+7 more Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 27
Not applicableStudy completedNCT03576742What this trial is testingSevere Immune Cytopenia Registry Www.Sic-reg.OrgWho this might be right forImmune ThrombocytopeniaAutoimmune Hemolytic AnemiaEvans Syndrome+1 more Medical University of Graz 80
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
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Testing effectiveness (Phase 2)Looking for participantsNCT01962415What this trial is testingReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTWho this might be right forPrimary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more Paul Szabolcs 100
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
Not applicableAvailableNCT06731036What this trial is testingExpanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell BoostsWho this might be right forSevere Combined ImmunodeficiencyFanconi AnemiaDyskeratosis Congenita+1 more University of Florida
Not applicableStudy completedNCT05544448What this trial is testingIn Vitro Effect Study of Interleukin-2 Muteins on Regulatory T Cells of Patients With Different Autoimmune, Allo-immune or Inflammatory DiseasesWho this might be right forAutoimmune DiseasesInflammatory DiseaseAcquired Bone Marrow Aplasia+8 more Assistance Publique - Hôpitaux de Paris 67
Early research (Phase 1)Study completedNCT00744692What this trial is testingReduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant DisordersWho this might be right forNon Malignant DisordersImmunodeficienciesCongenital Marrow Failures+5 more Duke University 22
Large-scale testing (Phase 3)Study completedNCT00455312What this trial is testingStem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAWho this might be right forDyskeratosis CongenitaAplastic Anemia Masonic Cancer Center, University of Minnesota 36
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