3 completed, 1 active not recruiting, 1 recruiting, 1 withdrawn. 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Completed 1 |
| Phase 3 | 1 | Withdrawn 1 |
Follow Cerebrotendinous Xanthomatoses: new trials by email Literature Review of Cerebrotendinous Xanthomatoses trials Research Report on Cerebrotendinous Xanthomatoses
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 Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) Chenodeoxycholic acid, Chenodeoxycholic acid Placebo - Cerebrotendinous Xanthomatoses | Phase 3 | Withdrawn | Leadiant Biosciences, Inc. | 2024-05-27 | 2024-11-18 | |
| Bioequivalence Study to Compare Chenodeoxycholic Acid Capsules (250mg Chenodeoxycholic Acid) Versus Chenodeoxycholic Acid Leadiant 250 mg Hard Capsules (250mg Chenodeoxycholic Acid) Chenodeoxycholic acid, Chenodeoxycholic acid leadiant - Cerebrotendinous Xanthomatoses | Phase 1 | Completed | Humanis Saglık Anonim Sirketi | 24 | 2022-06-12 | 2023-12-28 |
| 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 | |
| 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 | |
| Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX) Chenodeoxycholic Acid - Cerebrotendinous Xanthomatoses | Completed | Leadiant Biosciences Ltd. | 28 | 2014-12-09 | 2022-08-12 |
Xanthomatosis, Cerebrotendinous (6) · Canavan Disease (3) · Leukodystrophy, Globoid Cell (3) · Leukodystrophy, Metachromatic (3) · Peroxisomal Disorders (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) · Charcot-Marie-Tooth Disease (2) · Cockayne Syndrome (2) · Gangliosidoses (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6) · Genetic Diseases, Inborn (6) · Lipid Metabolism Disorders (6) · Lipid Metabolism, Inborn Errors (6) · Metabolic Diseases (6) · Metabolism, Inborn Errors (6) · Nutritional and Metabolic Diseases (6) · Xanthomatosis (6) · Abnormalities, Multiple (3) · Bone Diseases (3) · Bone Diseases, Developmental (3) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Carbohydrate Metabolism, Inborn Errors (3)
Children's Hospital of Philadelphia (2) · Humanis Saglık Anonim Sirketi (1) · Leadiant Biosciences Ltd. (1) · Leadiant Biosciences, Inc. (1) · RTI International (1)
United States (3) · Israel (1) · Jordan (1)
Adeline Vanderver, MD (2) · Curt Scharfe, MD, PhD (1)
PMID 31315600, PMID 32342562, PMID 32749716, PMID 35142618
2023 (1), U01NS106845 (1), U54NS115052 (1)