Trial · NCT04270682
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
NCT04270682, Phase 3, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2024-10-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE). Trials, record NCT04270682. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04270682
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
- NCT number
- NCT04270682
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 19 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- CTX
- Interventions
- Blinded CDCA 250 mg TID, Placebo, Open-Label CDCA 250 mg TID, Rescue Medication CDCA 250 mg TID, CDCA Weight-Based Dose TID
- First posted
- 2020-02-17
- Last updated
- 2024-10-28
- Results first posted
- 2024-10-28
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | Primary Analysis of Change from Baseline in Urine 23S-Pentol (Natural Log-Transformed) during the Two Double-blind Periods- Paired T-test |
| Secondary | Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | Primary Analysis of Change from Baseline Plasma Cholestanol (Natural Log-transformed) at the End of the Two Double-Blind Periods- Paired T-test |
| Secondary | Change From Baseline Plasma 7αC4 During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | Primary Analysis of Change from Baseline Plasma 7αC4 (Natural Log-Transformed) During the Two Double-Blind Periods |
| Secondary | Proportion of Participants Who Received Rescue Treatment During Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | Proportion of Participants Who Received Rescue Treatment during Two Double-Blind Periods - Prescott's Method |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | natural log of ng/mL | MEAN |
| SECONDARY | Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | All adult cohort participants who were randomized and took at least one dose of randomized study medication, and who had been on CDCA for at least 2 months prior to the open-label run-in period. | natural log of μg/mL | MEAN |
| SECONDARY | Change From Baseline Plasma 7αC4 During the Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | natural log of ng/mL | MEAN |
| SECONDARY | Proportion of Participants Who Received Rescue Treatment During Two Double-Blind Periods | Two double-blind periods: Week 0 to Week 4, Week 12 to Week 16 | All adult cohort participants who were randomized and took at least one dose of randomized study medication. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | CDCA TID | Pooled Double Blind Baseline | 7.18 | Standard Deviation 0.797 | natural log of ng/mL |
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Placebo TID | Pooled Double Blind Baseline | 6.96 | Standard Deviation 1.076 | natural log of ng/mL |
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | CDCA TID | Pooled last non missing postbaseline Double Blind record | 7.45 | Standard Deviation 0.675 | natural log of ng/mL |
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Placebo TID | Pooled last non missing postbaseline Double Blind record | 10.34 | Standard Deviation 0.788 | natural log of ng/mL |
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | CDCA TID | Pooled change from baseline to last non missing postbaseline Double Blind record | 0.20 | Standard Deviation 0.732 | natural log of ng/mL |
| Change From Baseline in Urine 23S-Pentol During the Two Double-Blind Periods | Placebo TID | Pooled change from baseline to last non missing postbaseline Double Blind record | 3.38 | Standard Deviation 1.111 | natural log of ng/mL |
| Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | CDCA TID | Pooled Double Blind baseline | 1.13 | Standard Deviation 0.791 | natural log of μg/mL |
| Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Placebo TID | Pooled Double Blind baseline | 1.14 | Standard Deviation 0.534 | natural log of μg/mL |
| Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | CDCA TID | Pooled last non missing post baseline Double Blind record | 1.10 | Standard Deviation 0.728 | natural log of μg/mL |
| Change From Baseline Plasma Cholestanol During the Two Double-Blind Periods | Placebo TID | Pooled last non missing post baseline Double Blind record | 1.88 | Standard Deviation 0 | natural log of μg/mL |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Sequence AB | 6 | |
| Overall Study | STARTED | Sequence BA | 7 | |
| Overall Study | STARTED | Pediatric Cohort | 5 | |
| Overall Study | COMPLETED | Sequence AB | 6 | |
| Overall Study | COMPLETED | Sequence BA | 7 | |
| Overall Study | COMPLETED | Pediatric Cohort | 4 | |
| Overall Study | NOT COMPLETED | Sequence AB | 0 | |
| Overall Study | NOT COMPLETED | Sequence BA | 0 | |
| Overall Study | NOT COMPLETED | Pediatric Cohort | 1 |
Source ClinicalTrials.gov
Adverse events105
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Diarrhea | Gastrointestinal disorders | CDCA | 0 | 13 | |
| other | Diarrhea | Gastrointestinal disorders | Placebo | 1 | 13 | |
| other | Diarrhea | Gastrointestinal disorders | Pediatric Cohort | 1 | 5 | |
| other | Constipation | Gastrointestinal disorders | CDCA | 0 | 13 | |
| other | Constipation | Gastrointestinal disorders | Placebo | 2 | 13 | |
| other | Constipation | Gastrointestinal disorders | Pediatric Cohort | 0 | 5 | |
| other | Headache | Nervous system disorders | CDCA | 1 | 13 | |
| other | Headache | Nervous system disorders | Placebo | 0 | 13 | |
| other | Headache | Nervous system disorders | Pediatric Cohort | 0 | 5 | |
| other | Upper Respiratory Tract Infection | Infections and infestations | CDCA | 1 | 13 |
1 to 10 of 105Source ClinicalTrials.gov
See all 105 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CDCA | 0 | 13 |
| Total, all-cause mortality | Placebo | 0 | 13 |
| Total, all-cause mortality | Pediatric Cohort | 0 | 5 |
| Total, other adverse events | CDCA | 13 | 13 |
| Total, other adverse events | Placebo | 13 | 13 |
| Total, other adverse events | Pediatric Cohort | 5 | 5 |
| Total, serious adverse events | CDCA | 0 | 13 |
| Total, serious adverse events | Placebo | 1 | 13 |
| Total, serious adverse events | Pediatric Cohort | 0 | 5 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04270682
GET https://conductscience.org/api/trials/v1/NCT04270682/results?page=2