2 completed, 2 recruiting. 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 1 | Recruiting 1 |
Follow PMD: new trials by email Literature Review of PMD trials Research Report on PMD
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 |
|---|---|---|---|---|---|---|
| A Phase 2 Study of Radiotherapy-induced Immune Priming to Enhance Elranatamab (Elra) in Relapsed Refractory Multiple Myeloma (RRMM) With Extramedullary Disease (EMD) and Paramedullary Disease (PMD) "PRIME-EMD-PMD" Elranatamab - Phase 2, Radiotherapy-Induced Immune Priming | Phase 2 | Recruiting | M.D. Anderson Cancer Center | 34 | 2026-03-31 | 2026-09-18 |
| 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 | |
| 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 |
Pelizaeus-Merzbacher Disease (3) · Adrenoleukodystrophy (2) · Aicardi-Goutieres syndrome (2) · Alexander Disease (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2) · Canavan Disease (2) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2) · Gangliosidoses (2) · Gangliosidoses, GM2 (2) · Leukodystrophy, Globoid Cell (2) · Leukodystrophy, Hypomyelinating, 5 (2)
Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Cardiovascular Diseases (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Demyelinating Diseases (3) · Genetic Diseases, Inborn (3) · Genetic Diseases, X-Linked (3) · Hereditary Central Nervous System Demyelinating Diseases (3) · Immune System Diseases (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3)
Children's Hospital of Philadelphia (2) · M.D. Anderson Cancer Center (1) · StemCells, Inc. (1)
United States (4)
Adeline Vanderver, MD (2) · Christine Ye, MD (1) · Stephen Huhn, MD (1)
U01NS106845 (1), U54NS115052 (1)