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
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 completedNCT01391637What this trial is testingLong-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)Who this might be right forPelizaeus-Merzbacher DiseasePMD StemCells, Inc. 4
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Not applicableLooking for participantsNCT07290114What this trial is testingMesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator TapeWho this might be right forStress Urinary Incontinence (SUI) Mehmet Incebıyik 147
Early research (Phase 1)Looking for participantsNCT06150716What this trial is testingOrbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)Who this might be right forPelizaeus-Merzbacher Disease Ionis Pharmaceuticals, Inc. 24
Early research (Phase 1)Study completedNCT01005004What this trial is testingStudy of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) SubjectsWho this might be right forPelizaeus-Merzbacher Disease StemCells, Inc. 4
Not applicableLooking for participantsNCT04880356What this trial is testingLongitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.Who this might be right forInherited DiseaseRare DiseasesMetabolic Disease+3 more Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Early research (Phase 1)Looking for participantsNCT02254863What this trial is testingUCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsWho this might be right forAdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more Joanne Kurtzberg, MD 40
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
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
Not applicableLooking for participantsNCT03047369What this trial is testingThe Myelin Disorders Biorepository ProjectWho this might be right forLeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more Children's Hospital of Philadelphia 12,000
Not applicableLooking for participantsNCT05659901What this trial is testingRocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher DiseaseWho this might be right forPelizaeus-Merzbacher Disease Ionis Pharmaceuticals, Inc. 32