Not applicableUnknownNCT04319796What this trial is testingEuropean Registry on Rare Neurological DiseasesWho this might be right forRare Diseases University Hospital Tuebingen 5,000
Not applicableUnknownNCT04324775What this trial is testingComparison of Acute Effect of Different Manual Techniques on Muscle Fascia StiffnessWho this might be right forManual Lymphatic DrainageSwedish MassageMuscle Fascia Stiffness Marmara University 30
Testing effectiveness (Phase 2)Study completedNCT01043640What this trial is testingAllogeneic Bone Marrow Transplant for Inherited Metabolic DisordersWho this might be right forMucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more Masonic Cancer Center, University of Minnesota 46
Not applicableWithdrawnNCT05755568What this trial is testingLearn About Metachromatic Leukodystrophy (MLD) in Children in SpainWho this might be right forMetachromatic Leukodystrophy (MLD) Takeda
Not applicableAvailableNCT07487389What this trial is testingZilganersen Expanded Access Program for Individuals With Alexander DiseaseWho this might be right forAlexander Disease Ionis Pharmaceuticals, Inc.
Not applicableStudy completedNCT02843555What this trial is testingNatural History of the LeukodystrophiesWho this might be right forLeukodystrophies Baylor Research Institute 10
Testing effectiveness (Phase 2)Study completedNCT01560182What this trial is testingGene Therapy for Metachromatic Leukodystrophy (MLD)Who this might be right forLysosomal Storage DiseaseMetachromatic Leukodystrophy Orchard Therapeutics 20
Early research (Phase 1)WithdrawnNCT01003912What this trial is testingFetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage DiseasesWho this might be right forLysosomal Storage DiseasesInborn Errors of Metabolism Joanne Kurtzberg, MD
Not applicableStudy completedNCT01938014What this trial is testingLysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenWho this might be right forMucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more University of Chicago 19
Not applicableStudy completedNCT00889174What this trial is testingThe Nosology and Etiology of Leukodystrophies of Unknown CausesWho this might be right forLeukodystrophyLeukoencephalopathy National Human Genome Research Institute (NHGRI) 76
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Testing effectiveness (Phase 2)Enrolling By InvitationNCT06369974What this trial is testingSingle Participant Study of an Experimental ASO Treatment for TUBB4A-related LeukodystrophyWho this might be right forGenetic Disease Massachusetts General Hospital 1
Testing effectiveness (Phase 2)Study completedNCT01510028What this trial is testingMulticenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)Who this might be right forMetachromatic Leukodystrophy (MLD) Shire 24
Testing effectiveness (Phase 2)Study completedNCT01801709What this trial is testingIntracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic LeukodystrophyWho this might be right forMetachromatic Leukodystrophy Institut National de la Santé Et de la Recherche Médicale, France 5
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)Active Not RecruitingNCT05739643What this trial is testingGene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTWho this might be right forKrabbe Disease Forge Biologics, Inc 9
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)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 applicableNo Longer AvailableNCT02084121What this trial is testingAllogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)Who this might be right forMetachromatic Leukodystrophy Talaris Therapeutics Inc.
Early research (Phase 1)Study completedNCT00418561What this trial is testingMetazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)Who this might be right forMetachromatic Leukodystrophy (MLD) Shire 13