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Completed Phase 3 Interventional Results available

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

ClinicalTrials.gov ID: NCT04270682

Public ClinicalTrials.gov record NCT04270682. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Jul 22, 2026, 8:53 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 3 Study to Evaluate the Effects of Chenodeoxycholic Acid in Adult and Pediatric Patients With Cerebrotendinous Xanthomatosis

Study identification

NCT ID
NCT04270682
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Lead sponsor
Mirum Pharmaceuticals, Inc.
Industry
Enrollment
19 participants

Conditions and interventions

Conditions

Interventions

  • Blinded CDCA 250 mg TID Drug
  • Placebo Drug
  • Open-Label CDCA 250 mg TID Drug
  • Rescue Medication CDCA 250 mg TID Drug
  • CDCA Weight-Based Dose TID Drug

Drug

Eligibility (public fields only)

Age range
1 Month and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jan 30, 2020
Primary completion
Jul 12, 2023
Completion
Oct 3, 2023
Last update posted
Oct 27, 2024

2020 – 2023

United States locations

U.S. sites
9
U.S. states
8
U.S. cities
8
Facility City State ZIP Site status
Travere Investigational Site Aurora Colorado 80045
Travere Investigational Site Orlando Florida 32806
Travere Investigational Site Iowa City Iowa 52242
Travere Investigational Site New Orleans Louisiana 70112
Travere Investigational Site New Orleans Louisiana 70121
Travere Investigational Site Great Neck New York 11021
Travere Investigational Site Columbus Ohio 43221
Travere Investigational Site Austin Texas 78723
Travere Investigational Site Seattle Washington 98195

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT04270682, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Oct 27, 2024 · Synced Jul 22, 2026

Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.

Open the official record

The complete protocol, eligibility criteria, and contact information for NCT04270682 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →