9 completed, 7 unknown, 4 recruiting, 2 active not recruiting. 8 Not applicable, 3 Phase 3, 2 Phase 1, 2 Phase 2, 1 Phase 4.
Follow Xanthomatosis: new trials by email Literature Review of Xanthomatosis trials Research Report on Xanthomatosis
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 |
|---|---|---|---|---|---|---|
| Topical 2% Simvastatin for Xanthelasma Palpebrarum Topical Simvastatin 2% Cream, Vehicle cream - Xanthelasma Palpebrarum | Phase 2 | Not yet recruiting | Klira Skin | 30 | 2026-08-01 | 2026-06-23 |
| Observation of the Effect and Mechanism of Multi Tunnel Minimally Invasive Treatment for Xanthelasma Palpebrarum Multi level and multi tunnel puncture treatment, Heparin Sodium Injection, Multi Level and Multi Tunnel Puncture Treatment + Heparin Sodium Injection - Xanthelasma Palpebrarum | Not applicable | Enrolling by invitation | Fuzhou First General Hospital Skin Disease Prevention and Control Hospital | 270 | 2025-02-01 | 2025-03-11 |
| ''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 |
| Efficacy of 100% Trichloroacetic Acid Vs. Cryotherapy in Xanthelasma Palpebrarum 100% Trichloroacetic Acid (TCA), Cryotherapy (Liquid Nitrogen) - Xanthelasma Palpebrarum | Not applicable | Active not recruiting | Nishtar Medical University | 60 | 2024-09-07 | 2025-02-21 |
| 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Xanthomatosis (CTX) Chenodeoxycholic acid, Chenodeoxycholic acid Placebo - Cerebrotendinous Xanthomatoses | Phase 3 | Withdrawn | Leadiant Biosciences, Inc. | 2024-05-27 | 2024-11-18 | |
| Efficacy and Safety of Ultrapulse Carbon Dioxide Laser Treatment in Eyelid Tumors Ultra-pulsed Carbon Dioxide Laser - Eyelid Tumor, Xanthelasma Palpebrarum | Unknown | Sun Yat-sen University | 1000 | 2022-11-01 | 2024-03-07 | |
| 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 |
| Atorvastatin Therapy on Xanthoma in Alagille Syndrome atorvastatin - Alagille Syndrome, Atorvastatin | Phase 4 | Completed | Children's Hospital of Fudan University | 15 | 2022-03-22 | 2026-03-27 |
| 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 |
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Xanthomatosis, Cerebrotendinous (23) · 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)
Lipid Metabolism Disorders (29) · Nutritional and Metabolic Diseases (29) · Metabolic Diseases (28) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Genetic Diseases, Inborn (23) · Lipid Metabolism, Inborn Errors (23) · Metabolism, Inborn Errors (22) · Pathologic Processes (9) · Pathological Conditions, Signs and Symptoms (9) · Brain Diseases (6) · Cardiovascular Diseases (6) · Central Nervous System Diseases (6) · Endocrine System Diseases (6) · Skin and Connective Tissue Diseases (6)
Chenodeoxycholic Acid (5) · Blood Specimen Collection (2) · Atorvastatin (1) · Bite Force (1) · Cryotherapy (1) · Cyclophosphamide (1) · Diphtheria-Tetanus Vaccine (1) · Granulocyte-Macrophage Colony-Stimulating Factor (1) · Hematologic Tests (1) · Heparin (1) · Lasers, Gas (1) · Lovastatin (1) · Simvastatin (1) · Sodium Chloride (1) · Teriparatide (1)
TRPHARM (3) · Children's Hospital of Philadelphia (2) · Albert Einstein College of Medicine (1) · Alexandria University (1) · Children's Hospital of Fudan University (1) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (1) · Fuzhou First General Hospital Skin Disease Prevention and Control Hospital (1) · Humanis Saglık Anonim Sirketi (1) · Joint Authority for Päijät-Häme Social and Health Care (1) · Klira Skin (1) · Leadiant Biosciences Ltd. (1) · Leadiant Biosciences, Inc. (1) · Mirum Pharmaceuticals, Inc. (1) · Nanhua University (1) · National Center for Research Resources (NCRR) (1)
United States (11) · China (3) · Turkey (Türkiye) (3) · Germany (2) · Israel (2) · Italy (2) · Spain (2) · Argentina (1) · Belgium (1) · Brazil (1) · Canada (1) · Czechia (1) · Denmark (1) · Egypt (1) · Finland (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) · Muhammad Irfan Jamil (1) · Omar Tareq (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)