Trial · NCT01115582
Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
NCT01115582, Phase 3, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2016-12-09
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis. Trials, record NCT01115582. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01115582
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis
- NCT number
- NCT01115582
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 16 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Inborn Errors of Bile Acid Synthesis
- Interventions
- Cholic acid
- First posted
- 2010-05-04
- Last updated
- 2023-10-03
- Results first posted
- 2016-12-09
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Serum Transaminases | At baseline and after 30 days of treatment | Concentration of serum alanine transaminase (ALT) and aspartate transaminase (AST) |
| Primary | Serum and Urine Bile Acids | At baseline (BL) and after 30 days of treatment (D30) | Concentration of bile acids in serum (S) and urine (U). (abbreviations: chol.=cholenoic; monohydro=monohydroxy; dihydro=monohydro) |
| Secondary | Adverse Events | Total of 30 days, i.e. from the time point the patients entered into the study up to the end of treatment | Total number of patients with any adverse events |
| Secondary | Blood Pressure | At baseline and after 30 days of treatment | Systolic blood pressure (SBP) and diastolic blood pressure (DBP) |
| Secondary | Physical Examination | At baseline (BL) and after 30 days of treatment (D30) | Total number of patients with abnormal findings from general physical examination |
| Secondary | Total Bilirubin | At baseline and after 30 days of treatment | Concentration of total bilirubin in serum |
Source ClinicalTrials.gov
Outcomes6
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Serum Transaminases | At baseline and after 30 days of treatment | All patients entered and treated | U/L | MEAN |
| PRIMARY | Serum and Urine Bile Acids | At baseline (BL) and after 30 days of treatment (D30) | All patients entered and treated | mmol/L | MEAN |
| SECONDARY | Adverse Events | Total of 30 days, i.e. from the time point the patients entered into the study up to the end of treatment | All patients entered and treated | participants | NUMBER |
| SECONDARY | Blood Pressure | At baseline and after 30 days of treatment | All patients entered and treated | mmHg | MEAN |
| SECONDARY | Physical Examination | At baseline (BL) and after 30 days of treatment (D30) | All patients entered and treated | participants | NUMBER |
| SECONDARY | Total Bilirubin | At baseline and after 30 days of treatment | All patients entered and treated | mg/dL | MEAN |
Source ClinicalTrials.gov
Results by group33
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Serum Transaminases | Cholic Acid | ALT, baseline | 31.4 | Standard Deviation 21.9 | U/L |
| Serum Transaminases | Cholic Acid | ALT, Day 30 | 30.9 | Standard Deviation 24.0 | U/L |
| Serum Transaminases | Cholic Acid | AST, baseline | 62.7 | Standard Deviation 27.1 | U/L |
| Serum Transaminases | Cholic Acid | AST, Day 30 | 65.0 | Standard Deviation 39.0 | U/L |
| Serum and Urine Bile Acids | Cholic Acid | U, BL: 3β,7α-dihydroxy-Δ5 sulfate m/z 469 | 12.37 | Standard Deviation 35.31 | mmol/L |
| Serum and Urine Bile Acids | Cholic Acid | U, D30: 3β,7α-dihydroxy-Δ5 sulfate m/z 469 | 2.762 | Standard Deviation 3.955 | mmol/L |
| Serum and Urine Bile Acids | Cholic Acid | U, BL: 3β,7α,12α-trihydroxy-Δ5 sulfate m/z 485 | 14.11 | Standard Deviation 42.78 | mmol/L |
| Serum and Urine Bile Acids | Cholic Acid | U, D30: 3β,7α,12α-trihydroxy-Δ5 sulfate m/z 485 | 2.011 | Standard Deviation 2.995 | mmol/L |
| Serum and Urine Bile Acids | Cholic Acid | U, BL: 3β,7α-dihydroxy-Δ5 gluycosulfate m/z 526 | 159.85 | Standard Deviation 474.52 | mmol/L |
| Serum and Urine Bile Acids | Cholic Acid | U, D30: 3β,7α-dihydroxy-Δ5 gluycosulfate m/z 526 | 19.958 | Standard Deviation 29.341 | mmol/L |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cholic Acid | 16 | |
| Overall Study | COMPLETED | Cholic Acid | 16 | |
| Overall Study | NOT COMPLETED | Cholic Acid | 0 |
Source ClinicalTrials.gov
Adverse events10
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 | Decreased/low 250H/Vit D | General disorders | Cholic Acid | 4 | 16 | |
| other | Decreased Vitamin A | General disorders | Cholic Acid | 1 | 16 | |
| other | Nosebleed | Vascular disorders | Cholic Acid | 1 | 16 | |
| other | Diarrhoea | Gastrointestinal disorders | Cholic Acid | 1 | 16 | |
| other | Reflux | Gastrointestinal disorders | Cholic Acid | 1 | 16 | |
| other | Increased ALT | Hepatobiliary disorders | Cholic Acid | 1 | 16 | |
| other | Increased AST | Hepatobiliary disorders | Cholic Acid | 1 | 16 | |
| other | Muscle spasm | Musculoskeletal and connective tissue disorders | Cholic Acid | 1 | 16 | |
| serious | Vomiting | Gastrointestinal disorders | Cholic Acid | 1 | 16 | |
| serious | Fever | General disorders | Cholic Acid | 1 | 16 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cholic Acid | ||
| Total, other adverse events | Cholic Acid | 9 | 16 |
| Total, serious adverse events | Cholic Acid | 1 | 16 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
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/NCT01115582
GET https://conductscience.org/api/trials/v1/NCT01115582/results?page=2