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Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

NCT04270682 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2024-10-28

Study results available
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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)

Conditions

  • CTX

Interventions

DRUG

Blinded CDCA 250 mg TID

Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

DRUG

Placebo

Adult cohort patients will receive blinded 250 mg CDCA TID or placebo during the double-blind periods based upon their treatment assignment.

DRUG

Open-Label CDCA 250 mg TID

Adult cohort patients will receive open-label 250 mg CDCA TID during the run-in and washout periods of the study or as rescue medication during the double-blind periods, if needed, based on clinical symptoms.

DRUG

Rescue Medication CDCA 250 mg TID

CDCA 250 mg TID will be provided as rescue medication during the double-blind periods, if needed, based on laboratory results.

DRUG

CDCA Weight-Based Dose TID

Patients in the pediatric cohort will complete a weight-based dose titration to a tolerated dose and will maintain that tolerated dose for the remainder of the study. Pediatric cohort dosing of CDCA will not exceed an equivalent dose of 750 mg/day.

Sponsors & Collaborators

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-31
Primary Completion
2023-07-13
Completion
2023-10-04
FDA Drug
Yes

Countries

  • United States
  • Brazil

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04270682 on ClinicalTrials.gov