Clinical trials: Cerebrotendinous Xanthomatosis
Cerebrotendinous Xanthomatosis
The Prevalence of CTX Disorder in Juvenile Cataract Cases in Turkey
Epidemiologic observational study. Study will include two phases: 1. Retrospective cross-sectional phase: At this stage of the study, retrospective screenings are carried out at study sites and all juvenile cataract cases are…
- Trial name
- GEN-EYE-I (NCT03584893)
- Testing
- Blood sampling for Cholestanol Analysis
- Where
- 36 sites in Turkey (Türkiye)
- Volunteers
- 1,000 people
- Sponsor
- TRPHARM
Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma
Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I)
The standard of care for children with DIPG includes focal radiotherapy (RT) but outcomes have remained dismal despite this treatment.
- Trial name
- BRAVO (NCT03396575)
- Testing
- TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Florida
Cerebrotendinous Xanthomatoses
Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
Retrospective study of CTX patients to investigate the safety and clinical efficacy of Chenodeoxycholic Acid
- Trial name
- NCT05499026
- Testing
- Chenodeoxycholic Acid
- Volunteers
- 28 people
- Sponsor
- Leadiant Biosciences Ltd.