8 completed, 6 unknown, 3 recruiting, 2 terminated. 6 Not applicable, 3 Phase 3, 2 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 6 | Completed 2, Unknown 2, Recruiting 1, Withdrawn 1 |
| Phase 3 | 3 | Completed 2, Withdrawn 1 |
| Phase 1 | 2 | Completed 1, Terminated 1 |
| Phase 2 | 1 | Unknown 1 |
Follow Xanthomatosis, Cerebrotendinous: new trials by email Literature Review of Xanthomatosis, Cerebrotendinous trials Research Report on Xanthomatosis, Cerebrotendinous
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 |
|---|---|---|---|---|---|---|
| ''Comparative Evaluation of Change in Bite Force and Levels of Bone Turnover Markers CTX and BALP in Hyperdivergent and Hypodivergent Cases During Retention Phases-A Prospective Clinical Trial '' Bite force and Gcf - Relapse, Bite Force | Not applicable | Recruiting | Postgraduate Institute of Dental Sciences Rohtak | 40 | 2024-12-25 | 2024-12-10 |
| 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Xanthomatosis (CTX) Chenodeoxycholic acid, Chenodeoxycholic acid Placebo - Cerebrotendinous Xanthomatoses | Phase 3 | Withdrawn | Leadiant Biosciences, Inc. | 2024-05-27 | 2024-11-18 | |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 |
| Type I Collagen Fragments in the GCF and Saliva During OTM Biological measuring of collagen fragments biomarker during orthodontic tooth movement - Collagen Degeneration | Not applicable | Unknown | Alexandria University | 14 | 2021-05-15 | 2021-08-27 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| 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 | |
| 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 |
| A Study on the Prevalence of Mutation of Cerebrotendinous Xanthomatosis (CTX) Mutation Analysis - Xanthomatosis, Cerebrotendinous | Unknown | TRPHARM | 800 | 2019-12-01 | 2021-04-01 | |
| An Observational Study for Evaluation of for the Prevalence of Cerebrotendinous Xanthomatosis (CTX) Disease Blood sampling for cholestanol analysis - CTX - Cerebrotendinous Xanthomatosis | Terminated | TRPHARM | 22 | 2019-10-19 | 2022-06-15 | |
| 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 | |
| Effects of a Liquid Meal and Gut Hormones (GIP and GLP-2) on Bone Remodeling in Participants With Hypoparathyroidism. Liquid meal (Nutridrink), GIP, GLP-2 - Hypoparathyroidism | Not applicable | Completed | University of Copenhagen | 7 | 2018-09-12 | 2019-12-12 |
| Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I) TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning - Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma | Phase 1 | Terminated | University of Florida | 11 | 2018-07-17 | 2025-10-29 |
| The Prevalence of CTX Disorder in Juvenile Cataract Cases in Turkey Blood sampling for Cholestanol Analysis - Cerebrotendinous Xanthomatosis | Unknown | TRPHARM | 1000 | 2018-06-01 | 2021-03-29 | |
| Investigation for Health Efficacy of Infrared-C Radiation on Diabetes Patients in Senior's Activity Center FIR-C mattress, FIR-C abdominal pad, fake FIR-C mattress - Diabetes Mellitus, Type 2 | Not applicable | Unknown | Nanhua University | 64 | 2018-03-23 | 2019-11-26 |
| 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 | |
| Cerebrotendinous Xanthomatosis (CTX) Prevalence Study Cerebrotendinous Xanthomatosis (CTX) | Completed | Travere Therapeutics, Inc. | 442 | 2015-09-01 | 2024-09-19 | |
| 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 | |
| The Role of Narrowband Ultraviolet B Exposure in the Maintenance of Vitamin D Levels During Winter nbUVB - Vitamin D Deficiency | Not applicable | Completed | Joint Authority for Päijät-Häme Social and Health Care | 37 | 2013-09-01 | 2014-10-27 |
| Evaluation of Carotid IMT and Atherogenic Risk Factors in Patients With Cerebrotendinous Xanthomatosis Blood tests - Cerebrotendinous Xanthomatosis (CTX) | Unknown | Sheba Medical Center | 17 | 2010-08-01 | 2012-06-07 |
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Leukodystrophy, Metachromatic (5) · Leukodystrophy, Globoid Cell (4) · Adrenoleukodystrophy (3) · Alexander Disease (3) · Canavan Disease (3) · Leukoencephalopathies (3) · Pelizaeus-Merzbacher Disease (3) · Peroxisomal Disorders (3) · Rare Diseases (3) · Aicardi-Goutieres syndrome (2) · Alexanders leukodystrophy (2) · Allan-Herndon-Dudley syndrome (2) · Attention Deficit Disorder with Hyperactivity (2) · CADASIL (2)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (23) · Lipid Metabolism Disorders (23) · Lipid Metabolism, Inborn Errors (23) · Nutritional and Metabolic Diseases (23) · Genetic Diseases, Inborn (22) · Metabolic Diseases (22) · Metabolism, Inborn Errors (22) · Xanthomatosis (22) · Pathologic Processes (8) · Pathological Conditions, Signs and Symptoms (8) · Brain Diseases (6) · Central Nervous System Diseases (6) · Endocrine System Diseases (6) · Bone Diseases (5) · Brain Diseases, Metabolic (5)
Chenodeoxycholic Acid (5) · Blood Specimen Collection (2) · Bite Force (1) · Cyclophosphamide (1) · Diphtheria-Tetanus Vaccine (1) · Granulocyte-Macrophage Colony-Stimulating Factor (1) · Hematologic Tests (1) · Lovastatin (1) · Sodium Chloride (1) · Teriparatide (1) · Zoledronic Acid (1)
TRPHARM (3) · Children's Hospital of Philadelphia (2) · Albert Einstein College of Medicine (1) · Alexandria University (1) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1) · Humanis Saglık Anonim Sirketi (1) · Joint Authority for Päijät-Häme Social and Health Care (1) · Leadiant Biosciences Ltd. (1) · Leadiant Biosciences, Inc. (1) · Mirum Pharmaceuticals, Inc. (1) · Nanhua University (1) · National Center for Research Resources (NCRR) (1) · Novartis (1) · Postgraduate Institute of Dental Sciences Rohtak (1) · RTI International (1)
United States (10) · Turkey (Türkiye) (3) · Israel (2) · Belgium (1) · Brazil (1) · Denmark (1) · Egypt (1) · Finland (1) · Germany (1) · India (1) · Italy (1) · Jordan (1) · Spain (1) · Taiwan (1)
Adeline Vanderver, MD (2) · Ching Yong Chin, Master (1) · Curt Scharfe, MD, PhD (1) · Elias Sayour, MD, PhD (1) · Erna Snellman, professor (1) · Gerald Salen (1) · Hasan Onal, Asc. Prof. (1) · Hofit Cohen, MD (1) · Jin-Bang Shang, Master (1) · Melissa Wasserstein, MD (1) · Mette Rosenkilde, MD (1) · Omar Tareq (1) · Toni T Karppinen, MD (1)
PMID 19602651, PMID 31315600, PMID 32342562, PMID 32749716, PMID 35142618, PMID 37782421, PMID 41667276, PMID 9492234
2019 (1), 2023 (1), R01HD073292 (1), U01NS106845 (1), U54NS115052 (1)