Large-scale testing (Phase 3)Study completedNCT02478840What this trial is testingEvaluation of Long-term Efficacy of Treatment With LamazymWho this might be right forAlpha-Mannosidosis Zymenex A/S 18
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
Not applicableLooking for participantsNCT06184503What this trial is testingAnalysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3Who this might be right forAlpha-Mannosidosis Chiesi Farmaceutici S.p.A. 5
Testing effectiveness (Phase 2)Study completedNCT02998879What this trial is testingTrial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-MannosidosisWho this might be right forAlpha-Mannosidosis Chiesi Farmaceutici S.p.A. 5
Not applicableWithdrawnNCT03264040What this trial is testingBiomarker for Mannosidosis Disease (BioMannosidosis)Who this might be right forAlpha-Mannosidase B DeficiencyLysosomal Alpha B MannosidosisAlpha-Mannosidase Deficiency CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)UnknownNCT01285700What this trial is testingDose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosisWho this might be right forAlpha Mannosidosis Zymenex A/S 10
Not applicableStudy completedNCT00498420What this trial is testingThe Natural History of Alpha-MannosidosisWho this might be right forAlpha Mannosidosis Zymenex A/S 45
Testing effectiveness (Phase 2)Study completedNCT00176917What this trial is testingStem Cell Transplantation for HurlerWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more Masonic Cancer Center, University of Minnesota 41
Not applicableUnknownNCT00005900What this trial is testingStudy of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationWho this might be right forI Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more Fairview University Medical Center 10
Not applicableStudy completedNCT02141503What this trial is testingClinical Biomarkers in Alpha-mannosidosisWho this might be right forAlpha-Mannosidosis Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 11
Not applicableEnded earlyNCT03651245What this trial is testingEuropean Alpha-Mannosidosis ParticipantWho this might be right forImmunodeficiencySkeletal AbnormalitiesDeafness+3 more CENTOGENE GmbH Rostock 667
Large-scale testing (Phase 3)Study completedNCT01908712What this trial is testingLamazym Aftercare Study FR Designed to Provide Treatment for French PatientsWho this might be right forAlpha-Mannosidosis Chiesi Farmaceutici S.p.A. 13
Testing effectiveness (Phase 2)Study completedNCT01681940What this trial is testingLong-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-MannosidosisWho this might be right forAlpha-Mannosidosis Zymenex A/S 10
Not applicableLooking for participantsNCT03333200What this trial is testingLongitudinal Study of Neurodegenerative DisordersWho this might be right forMLDKrabbe DiseaseALD+24 more University of Pittsburgh 1,500
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
Testing effectiveness (Phase 2)Ended earlyNCT01372228What this trial is testingPhase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersWho this might be right forHurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more Talaris Therapeutics Inc. 3
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
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
Not applicableStudy completedNCT01891422What this trial is testingLongitudinal Studies of the GlycoproteinosesWho this might be right forAspartylglucosaminuriaFucosidosisGalactosialidosis+6 more Greenwood Genetic Center 100
Testing effectiveness (Phase 2)Study completedNCT03513328What this trial is testingConditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationWho this might be right forBone Marrow Failure SyndromeThalassemiaSickle Cell Disease+13 more University of Florida 6