10 completed, 7 recruiting, 5 terminated, 4 active not recruiting. 11 Phase 2, 5 Phase 1, 2 Phase 3, 1 Not applicable.
Follow Leukodystrophy, Globoid Cell: new trials by email Literature Review of Leukodystrophy, Globoid Cell trials Research Report on Leukodystrophy, Globoid Cell
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients FBX-101 - Krabbe Disease | Terminated | Forge Biologics, Inc | 2 | 2024-08-06 | 2025-09-05 | |
| Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT FBX-101 - Krabbe Disease | Phase 1 / Phase 2 | Active not recruiting | Forge Biologics, Inc | 9 | 2023-02-03 | 2025-11-20 |
| Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease PBKR03 - Leukodystrophy, Globoid Cell | Phase 1 / Phase 2 | Suspended | Gemma Biotherapeutics | 24 | 2022-02-24 | 2026-06-30 |
| Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases. collection of data - Inherited Disease, Rare Diseases | Recruiting | Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta | 100 | 2021-03-01 | 2024-11-19 | |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 Safety and efficacy assessments - Inherited Metabolic Disorders (IMD) | Terminated | Magenta Therapeutics, Inc. | 3 | 2019-05-21 | 2021-03-15 | |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Krabbe Disease (BioKrabbe) Krabbe Disease | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) MGTA-456 - Inherited Metabolic Disorders (IMD) | Phase 2 | Completed | Magenta Therapeutics, Inc. | 8 | 2018-02-09 | 2021-11-03 |
| LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Leukodystrophy, White Matter Disease | Completed | Children's Hospital of Philadelphia | 236 | 2017-01-06 | 2025-11-10 | |
| The Myelin Disorders Biorepository Project Leukodystrophy, White Matter Disease | Recruiting | Children's Hospital of Philadelphia | 12000 | 2016-12-08 | 2025-10-23 | |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Krabbe Disease Global Patient Registry Krabbe Disease | Recruiting | State University of New York at Buffalo | 60 | 2014-09-01 | 2026-10-28 | |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Campath-1H, Clofarabine, Melphalan - Lysosomal Storage Disease, Peroxisomal Disorder | Not applicable | Completed | Masonic Cancer Center, University of Minnesota | 3 | 2012-07-11 | 2017-12-05 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
Showing 20 of 31. Sign in to see all of them.
Leukodystrophy, Metachromatic (19) · Adrenoleukodystrophy (14) · Mucopolysaccharidosis I (13) · alpha-Mannosidosis (9) · Mucopolysaccharidosis II (8) · Mucopolysaccharidosis VI (8) · Sandhoff Disease (8) · Tay-Sachs Disease (8) · Fucosidosis (7) · Lysosomal Storage Diseases (7) · Metabolism, Inborn Errors (7) · Mucopolysaccharidoses (7) · Mucopolysaccharidosis III (7) · Peroxisomal Disorders (7)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (31) · Nutritional and Metabolic Diseases (31) · Brain Diseases (30) · Brain Diseases, Metabolic (30) · Central Nervous System Diseases (30) · Demyelinating Diseases (30) · Hereditary Central Nervous System Demyelinating Diseases (30) · Lipid Metabolism Disorders (30) · Lipid Metabolism, Inborn Errors (30) · Lipidoses (30) · Lysosomal Storage Diseases, Nervous System (30) · Brain Diseases, Metabolic, Inborn (29) · Metabolic Diseases (29) · Nervous System Diseases (29) · Genetic Diseases, Inborn (28)
Alemtuzumab (5) · Stem Cell Transplantation (5) · Busulfan (3) · Cyclophosphamide (3) · Melphalan (3) · Bone Marrow Transplantation (2) · Clofarabine (2) · Cyclosporine (2) · Hematopoietic Stem Cell Transplantation (2) · Hydroxyurea (2) · Mycophenolic Acid (2) · Whole-Body Irradiation (2) · Antilymphocyte Serum (1) · Data Collection (1) · Gemfibrozil (1)
Masonic Cancer Center, University of Minnesota (6) · Children's Hospital of Philadelphia (2) · Forge Biologics, Inc (2) · Magenta Therapeutics, Inc. (2) · Paul Szabolcs (2) · University of Pittsburgh (2) · Aldagen (1) · CENTOGENE GmbH Rostock (1) · Fairview University Medical Center (1) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1) · Gemma Biotherapeutics (1) · Joanne Kurtzberg, MD (1) · National Center for Research Resources (NCRR) (1) · New York Medical College (1) · Polaryx Therapeutics, Inc. (1)
United States (27) · Brazil (1) · Canada (1) · Egypt (1) · Germany (1) · India (1) · Israel (1) · Italy (1) · Netherlands (1) · Sri Lanka (1) · United Kingdom (1)
Paul Orchard, MD (4) · Adeline Vanderver, MD (2) · Joanne Kurtzberg, MD (2) · Paul Szabolcs, MD (2) · Chester B. Whitley, Ph.D., M.D. (1) · Curt Scharfe, MD, PhD (1) · Deepa S Rajan, MD (1) · James Hinson, MD (1) · Judith P. Willner (1) · K. Scott Baker (1) · Maria L Escolar, MD (1) · May Orfali, MD (1) · Michael Msall, M.D. (1) · Mitchell S Cairo, MD (1)
PMID 21586746, PMID 36196048, PMID 31315600, PMID 32342562, PMID 32634238, PMID 32749716, PMID 32750127, PMID 35142618, PMID 35333110, PMID 37782421, PMID 39499628, PMID 41667276
R01HD104814 (1), R01NS061965 (1), U01NS106845 (1), U54NS065768 (1), U54NS115052 (1)