Ctexli (chenodeoxycholic acid) / Leadiant Biosci - LARVOL DELTA

CTEXLI (chenodiol). (PubMed, Clin Ther) - No abstract available

Journal

Mirum’s CTEXLI (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX) (Businesswire) - "Mirum Pharmaceuticals, Inc...today announced that the U.S. Food and Drug Administration (FDA) has approved CTEXLI (chenodiol) tablets, a bile acid, for the treatment of adults with cerebrotendinous xanthomatosis (CTX). CTEXLI is the first and only treatment approved for this rare, progressive and debilitating disease. The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures."

FDA approval • Genetic Disorders • Metabolic Disorders • Rare Diseases

FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease (PRNewswire) - "Today, the U.S. Food and Drug Administration approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease....The recommended dosage is 250 milligrams, taken orally three times a day."

FDA approval • Rare Diseases

LBI-CDCA-001: A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) (clinicaltrials.gov) - P3 | N=0 | Withdrawn | Sponsor: Leadiant Biosciences, Inc. | N=10 ➔ 0 | Active, not recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal

LBI-CDCA-001: A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) (clinicaltrials.gov) - P3 | N=10 | Active, not recruiting | Sponsor: Leadiant Biosciences, Inc. | Not yet recruiting ➔ Active, not recruiting

Enrollment closed

LBI-CDCA-001: A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) (clinicaltrials.gov) - P3 | N=10 | Not yet recruiting | Sponsor: Leadiant Biosciences, Inc. | Initiation date: Mar 2024 ➔ Jun 2024

Trial initiation date

A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) (clinicaltrials.gov) - P3 | N=10 | Not yet recruiting | Sponsor: Leadiant Biosciences, Inc.

New P3 trial

Bioequivalence Study to Compare Chenodeoxycholic Acid Capsules (250mg Chenodeoxycholic Acid) Versus Chenodeoxycholic Acid Leadiant 250 mg Hard Capsules (250mg Chenodeoxycholic Acid) (clinicaltrials.gov) - P1 | N=24 | Completed | Sponsor: Humanis Saglık Anonim Sirketi

New P1 trial

Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX) (clinicaltrials.gov) - P=N/A | N=28 | Completed | Sponsor: Leadiant Biosciences Ltd.

New trial

"Na #CDCA #Leadiant nu #trastuzumab @RocheNL en straks #Humira @abbvie ? @AutoriteitCM @StichtingFTV https://t.co/N072KVUo8E" (@StichtingFTV)

Pharmacology of pharmacy prepared chenodeoxycholic acid. (clinicaltrialsregister.eu) - P4; N=12; Ongoing; Sponsor: Amsterdam UMC

New P4 trial • Metabolic Disorders

Expert opinion on diagnosing, treating and managing patients with cerebrotendinous xanthomatosis (CTX): a modified Delphi study. (PubMed, Orphanet J Rare Dis) - "The results of this study provide a basis for standardisation of care and highlight key areas where further research is needed to inform best practices for the diagnosis, treatment and management of patients with CTX."

Clinical • Journal • CNS Disorders • Pediatrics

TOWARDS THE BIOCHEMICAL DIAGNOSIS OF BILE ACID DIARRHEA (DDW 2020) - P4 | "Compared with SeHCAT, C4 performed better than FGF19 for the diagnosis of bile acid diarrhea. With 76% diagnostic accuracy, C4 seemed an adequate alternative to SeHCAT. Of note, half of the patients with an indeterminate C4 result responded to treatment with a bile acid binder."

Rare Diseases • FGF • FGF19