3 completed, 1 recruiting. 1 Not applicable, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Completed 1 |
| Phase 3 | 1 | Completed 1 |
Follow CTX: new trials by email Literature Review of CTX trials Research Report on CTX
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 |
|---|---|---|---|---|---|---|
| Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE) Blinded CDCA 250 mg TID, Placebo, Open-Label CDCA 250 mg TID - CTX | Phase 3 | Completed | Mirum Pharmaceuticals, Inc. | 19 | 2020-01-31 | 2024-10-28 |
| 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 | |
| Prospective Study of TW in the Treatment of LN Type V With Gross Proteinuria tripterygium glycosides - Lupus Nephritis, Proteinuria | Not applicable | Completed | Nanjing University School of Medicine | 44 | 2009-04-01 | 2011-08-02 |
Xanthomatosis, Cerebrotendinous (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)
Autoimmune Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Connective Tissue Diseases (3) · Female Urogenital Diseases (3) · Female Urogenital Diseases and Pregnancy Complications (3) · Genetic Diseases, Inborn (3) · Immune System Diseases (3) · Kidney Diseases (3) · Lipid Metabolism Disorders (3) · Lipid Metabolism, Inborn Errors (3) · Male Urogenital Diseases (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nutritional and Metabolic Diseases (3) · Pathological Conditions, Signs and Symptoms (3)
Children's Hospital of Philadelphia (2) · Mirum Pharmaceuticals, Inc. (1) · Nanjing University School of Medicine (1)
United States (3) · Brazil (1) · China (1)
Adeline Vanderver, MD (2) · Hu Weixin, Doctor (1)
U01NS106845 (1), U54NS115052 (1)