Not applicableStudy completedNCT00043329What this trial is testingPost Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant OsteopetrosisWho this might be right forOsteopetrosis InterMune 6
Large-scale testing (Phase 3)Study completedNCT00775931What this trial is testingAllogeneic Transplantation For Severe OsteopetrosisWho this might be right forSevere Osteopetrosis Masonic Cancer Center, University of Minnesota 7
Not applicableNo Longer AvailableNCT01200017What this trial is testingExpanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell TransplantWho this might be right forAcute Lymphoblastic LeukemiaAcute Myeloid LeukemiaChronic Myeloid Leukemia+9 more Christopher Dvorak
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104What this trial is testingMT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisWho this might be right forMucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more Masonic Cancer Center, University of Minnesota 149
Testing effectiveness (Phase 2)Study completedNCT00730314What this trial is testingUnrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsWho this might be right forSickle Cell DiseaseThalassemiaAnemia+9 more Children's Hospital Los Angeles 25
Large-scale testing (Phase 3)Study completedNCT01019876What this trial is testingRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesWho this might be right forBone Marrow FailureOsteopetrosisFanconi Anemia+1 more Columbia University 38
Testing effectiveness (Phase 2)Ended earlyNCT00638820What this trial is testingReduced Intensity AlloTransplant For OsteopetrosisWho this might be right forOsteopetrosis Masonic Cancer Center, University of Minnesota 3
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
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
Early research (Phase 1)Ended earlyNCT04525352What this trial is testingA Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisWho this might be right forInfantile Malignant Osteopetrosis Rocket Pharmaceuticals Inc. 1
Large-scale testing (Phase 3)Study completedNCT00004402What this trial is testingPhase III Randomized Study of Interferon Gamma in Children With Severe, Congenital OsteopetrosisWho this might be right forOsteopetrosis FDA Office of Orphan Products Development 30