7 recruiting, 3 completed, 1 withdrawn. 4 Phase 1, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Recruiting 2, Completed 1, Withdrawn 1 |
| Not applicable | 1 | Recruiting 1 |
Follow Pelizaeus-Merzbacher Disease: new trials by email Literature Review of Pelizaeus-Merzbacher Disease trials Research Report on Pelizaeus-Merzbacher Disease
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 |
|---|---|---|---|---|---|---|
| Mesh-Free Versus Mesh-Based Surgery for Female Stress Urinary Incontinence: A Prospective Comparison of Pubo-Urethral Ligament Plication and Transobturator Tape Pubo-Urethral Ligament Plication (PLP), Transobturator Tape (TOT) - Stress Urinary Incontinence (SUI) | Not applicable | Recruiting | Mehmet Incebıyik | 147 | 2025-01-13 | 2025-12-18 |
| Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) ION356 - Pelizaeus-Merzbacher Disease | Phase 1 | Recruiting | Ionis Pharmaceuticals, Inc. | 24 | 2024-04-10 | 2025-12-12 |
| Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease Pelizaeus-Merzbacher Disease | Recruiting | Ionis Pharmaceuticals, Inc. | 32 | 2022-10-03 | 2026-10-02 | |
| 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 | |
| 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 |
| 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 | |
| Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) HuCNS-SC transplant in the lead-in phase - Pelizaeus-Merzbacher Disease, PMD | Completed | StemCells, Inc. | 4 | 2011-06-01 | 2016-05-13 | |
| Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects HuCNS-SC cells - Pelizaeus-Merzbacher Disease | Phase 1 | Completed | StemCells, Inc. | 4 | 2009-11-01 | 2015-01-15 |
| Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases ALD-601 - Lysosomal Storage Diseases, Inborn Errors of Metabolism | Phase 1 | Withdrawn | Joanne Kurtzberg, MD | 2009-10-01 | 2017-06-29 |
Leukodystrophy, Globoid Cell (5) · Adrenoleukodystrophy (4) · Leukodystrophy, Metachromatic (4) · Leukoencephalopathies (4) · Alexander Disease (3) · Gangliosidoses, GM2 (3) · Multiple Sulfatase Deficiency Disease (3) · Xanthomatosis, Cerebrotendinous (3) · Aicardi-Goutieres syndrome (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2)
Brain Diseases (11) · Brain Diseases, Metabolic (11) · Central Nervous System Diseases (11) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Demyelinating Diseases (11) · Genetic Diseases, X-Linked (11) · Hereditary Central Nervous System Demyelinating Diseases (11) · Nutritional and Metabolic Diseases (11) · Brain Diseases, Metabolic, Inborn (10) · Genetic Diseases, Inborn (10) · Metabolic Diseases (10) · Metabolism, Inborn Errors (10) · Nervous System Diseases (10) · Leukoencephalopathies (7) · Lipid Metabolism Disorders (6)
Palliative Care (1) · Suburethral Slings (1)
Children's Hospital of Philadelphia (2) · Ionis Pharmaceuticals, Inc. (2) · Joanne Kurtzberg, MD (2) · StemCells, Inc. (2) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1) · Mehmet Incebıyik (1) · University of Pittsburgh (1)
United States (9) · France (2) · Germany (2) · Italy (2) · Netherlands (2) · Israel (1) · Japan (1) · Turkey (Türkiye) (1) · United Kingdom (1)
Adeline Vanderver, MD (2) · Joanne Kurtzberg, MD (2) · Stephen Huhn, MD (2)
PMID 32342562, PMID 32749716, PMID 37782421, PMID 41667276
U01NS106845 (1), U54NS115052 (1)