Cerebrotendinous Xanthomatosis

Top drugs studied for this disease

All Approvals Safety alerts Label changes Trial results Research Guidance Withdrawals

CLINICAL_TRIALS research · 2025-09-12
Clinical Trial Update: ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
This clinical trial update describes ScreenPlus, a comprehensive, multi-disorder newborn screening program that is currently enrolling by in…
EMA approval · 2025-08-13
EMA Authorised: Chenodeoxycholic acid Leadiant (previously Chenodeoxycholic acid sigma-tau) (chenodeoxycholic acid) — Leadiant GmbH
The EMA authorised Chenodeoxycholic acid Leadiant for Cerebrotedinous xanthomatosis (a rare inborn error of metabolism) with orphan designat…
CLINICAL_TRIALS withdrawal · 2024-11-18
Clinical Trial Update: A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) [Phase 3]
A Phase 3 study evaluating chenodeoxycholic acid for cerebrotendinous xanthomatosis has been withdrawn.
CLINICAL_TRIALS trial_result · 2024-10-28
Clinical Trial Update: Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE) [Phase 3]
A Phase 3 RESTORE trial evaluating a Mirum Pharmaceuticals therapy for Cerebrotendinous Xanthomatosis has completed, with results posted.
CLINICAL_TRIALS trial_result · 2024-09-19
Clinical Trial Update: Cerebrotendinous Xanthomatosis (CTX) Prevalence Study
A clinical trial update shows that the prevalence study for cerebrotendinous xanthomatosis (CTX) has completed and results were posted.
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CLINICAL_TRIALS withdrawal · 2013-10-14
Clinical Trial Update: Cholestanol in Humans [NA]
A clinical trial evaluating cholestanol in humans for Cerebrotendinous Xanthomatosis has been withdrawn. The sponsor is listed as the US Dep…
CLINICAL_TRIALS research · 2012-06-07
Clinical Trial Update: Evaluation of Carotid IMT and Atherogenic Risk Factors in Patients With Cerebrotendinous Xanthomatosis
An observational clinical trial evaluating carotid intima-media thickness and other atherogenic risk factors in patients with Cerebrotendino…