CTX Clinical Trial - Study to Evaluate Patients With Cerebrotendinous

Clinical Trial Summary

The study is made up of two cohorts: a randomized double-blind crossover (placebo withdrawal with rescue) study among patients ≥ 16 years of age (adult cohort) and an open-label dose titration study among pediatric patients ≥1 month and <16 years of age (pediatric cohort)

See also
Status	Clinical Trial	Phase
Active, not recruiting	`NCT02699190` - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Recruiting	`NCT03047369` - The Myelin Disorders Biorepository Project
Completed	`NCT00935389` - Prospective Study of TW in the Treatment of LN Type V With Gross Proteinuria	N/A

See also

Status

Clinical Trial

Phase

Active, not recruiting

NCT02699190 - LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Recruiting

NCT03047369 - The Myelin Disorders Biorepository Project

Completed

NCT00935389 - Prospective Study of TW in the Treatment of LN Type V With Gross Proteinuria

N/A

Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.

Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.

Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

NCT number	NCT04270682
Study type	Interventional
Source	Mirum Pharmaceuticals, Inc.
Contact
Status	Completed
Phase	Phase 3
Start date	January 31, 2020
Completion date	October 4, 2023

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms