# Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

> **NCT04270682** · PHASE3 · COMPLETED · sponsor: **Mirum Pharmaceuticals, Inc.** · enrollment: 19 (actual)

## Conditions studied

- CTX

## Interventions

- **DRUG:** 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

## Key facts

- **NCT ID:** NCT04270682
- **Lead sponsor:** Mirum Pharmaceuticals, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2020-01-31
- **Primary completion:** 2023-07-13
- **Final completion:** 2023-10-04
- **Target enrollment:** 19 (ACTUAL)
- **Last updated:** 2024-10-28


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04270682

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04270682, "Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)". Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/clinical/NCT04270682. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*