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 applicableStudy completedNCT01626092What this trial is testingReduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersWho this might be right forLysosomal Storage DiseasePeroxisomal Disorder Masonic Cancer Center, University of Minnesota 3
Not applicableStudy completedNCT04041102What this trial is testingNatural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) PatientsWho this might be right forGM1 Gangliosidosis University of Pennsylvania 31
Large-scale testing (Phase 3)Ended earlyNCT04395339What this trial is testingGM1 Prophylaxis for WBRT Related Cognitive DysfunctionWho this might be right forBrain MetastasesRadiotherapy Side EffectCognitive Impairment+2 more Sun Yat-sen University 51
Not applicableStudy completedNCT04310163What this trial is testingInterviews and Video Capture in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 25
Not applicableLooking for participantsNCT05574673What this trial is testingCharacterization of Epithelial Ovarian Cancer Patients in Terms of Homologous Recombination PhenotypeWho this might be right forOvarian CancerPrimary Peritoneal CancerFallopian Tube Cancer Nordic Society of Gynaecological Oncology - Clinical Trials Unit 350
Large-scale testing (Phase 3)Looking for participantsNCT07082543What this trial is testingEvaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 GangliosidosisWho this might be right forGangliosidoses, GM2Gangliosidosis, GM1 Azafaros A.G. 75
Testing effectiveness (Phase 2)Looking for participantsNCT03952637What this trial is testingIntravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 GangliosidosisWho this might be right forLysosomal DiseasesGangliosidosisGM1 National Human Genome Research Institute (NHGRI) 54
Testing effectiveness (Phase 2)UnknownNCT05732129What this trial is testingThe Efficacy and Safety of Fluzoparib Plus Irinotecan as Second-line Treatment in Patients With Homologous Recombination Deficiency (HRD) Metastatic Colorectal Cancer.Who this might be right forHomologous Recombination Deficiency Alterations Metastatic Colorectal Cancer Fudan University 29
Not applicableLooking for participantsNCT00668187What this trial is testingA Natural History Study of the GangliosidosesWho this might be right forTay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more University of Minnesota 52
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04713475What this trial is testingStudy of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 GangliosidosisWho this might be right forGM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2+1 more Gemma Biotherapeutics 26
Large-scale testing (Phase 3)Looking for participantsNCT07082725What this trial is testingEvaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)Who this might be right forNiemann-Pick Type C Disease Azafaros A.G. 72
Post-approval studies (Phase 4)Ended earlyNCT02030015What this trial is testingSynergistic Enteral Regimen for Treatment of the GangliosidosesWho this might be right forGM1 GangliosidosesGM2 GangliosidosesTay-Sachs Disease+1 more University of Minnesota 16
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Not applicableStudy completedNCT04470713What this trial is testingNatural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2Who this might be right forGM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more Idorsia Pharmaceuticals Ltd. 226
Not applicableEnrolling By InvitationNCT05368038What this trial is testingScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramWho this might be right forAcid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Testing effectiveness (Phase 2)Ended earlyNCT04273269What this trial is testingA Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 GangliosidosisWho this might be right forGM1 Gangliosidosis LYSOGENE 5
Not applicableStudy completedNCT05109793What this trial is testingGM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)Who this might be right forGM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease Azafaros A.G. 31