Xanthomatosis, Cerebrotendinous clinical trials
The registry lists 23 studies for Xanthomatosis, Cerebrotendinous across more than 10 countries. 4 are recruiting or about to start. 12 of them are mainly about Xanthomatosis, Cerebrotendinous (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
TRPHARM (3) · Children's Hospital of Philadelphia (2) · Alexandria University (1) · Leadiant Biosciences, Inc. (1) · National Center for Research Resources (NCRR) (1) · Sheba Medical Center (1) · University of Florida (1) · Postgraduate Institute of Dental Sciences Rohtak (1)
What is being tested here
Drugs under test in Xanthomatosis, Cerebrotendinous studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Chenodeoxycholic Acid | 0 | 1 | 4 |
| Zoledronic Acid | 0 | 1 | 1 |
| Lovastatin | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The study
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
Who these studies take
Of the 3 Xanthomatosis, Cerebrotendinous studies recruiting now, 2 (67%) set no upper age limit and 1 (33%) stop at 75 or younger. 1 (33%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (33%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (33%) | 1 (33%) |
| 18 | 3 (100%) | 3 (100%) |
| 30 | 3 (100%) | 3 (100%) |
| 40 | 3 (100%) | 3 (100%) |
| 50 | 2 (67%) | 2 (67%) |
| 65 | 2 (67%) | 2 (67%) |
| 75 | 2 (67%) | 2 (67%) |
| 85 | 2 (67%) | 2 (67%) |
Across all 23 studies, recruiting or not: 43% stop at 75 or younger and 39% at 65; 70% take children; 4% are for women only and 0% for men only; 30% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 23 of 23. search them.
- The Myelin Disorders Biorepository ProjectRecruitingNCT030473691 country
- Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.RecruitingNCT048803561 country
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT053680381 country
- ''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 ''RecruitingNCT06456827Not applicable1 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesCompletedNCT026991901 country
- Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I)TerminatedNCT03396575Phase 11 country
- A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)WithdrawnNCT06260748Phase 31 country
- Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)CompletedNCT04270682Phase 32 countries
- Cerebrotendinous Xanthomatosis (CTX) Prevalence StudyCompletedNCT026382201 country
- Bioequivalence Study to Compare Chenodeoxycholic Acid Capsules (250mg Chenodeoxycholic Acid) Versus Chenodeoxycholic Acid Leadiant 250 mg Hard Capsules (250mg Chenodeoxycholic Acid)CompletedNCT06180057Phase 11 country
- Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)CompletedNCT05499026
- An Observational Study for Evaluation of for the Prevalence of Cerebrotendinous Xanthomatosis (CTX) DiseaseTerminatedNCT041130831 country
- Type I Collagen Fragments in the GCF and Saliva During OTMStatus unknownNCT05020431Not applicable1 country
- A Study on the Prevalence of Mutation of Cerebrotendinous Xanthomatosis (CTX)Status unknownNCT042180061 country
- The Prevalence of CTX Disorder in Juvenile Cataract Cases in TurkeyStatus unknownNCT035848931 country
- Effects of a Liquid Meal and Gut Hormones (GIP and GLP-2) on Bone Remodeling in Participants With Hypoparathyroidism.CompletedNCT03728959Not applicable1 country
- Investigation for Health Efficacy of Infrared-C Radiation on Diabetes Patients in Senior's Activity CenterStatus unknownNCT04177420Not applicable1 country
- The Role of Narrowband Ultraviolet B Exposure in the Maintenance of Vitamin D Levels During WinterCompletedNCT02275650Not applicable1 country
- Cholestanol in HumansWithdrawnNCT00018694Not applicable1 country
- Evaluation of Carotid IMT and Atherogenic Risk Factors in Patients With Cerebrotendinous XanthomatosisStatus unknownNCT016138981 country
- Efficacy Study of Zoledronic Acid and Teriparatide Combination Therapy in Women With OsteoporosisCompletedNCT00439244Phase 34 countries
- Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous XanthomatosisStatus unknownNCT00004346Phase 21 country