Trial · NCT00007020
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
NCT00007020, Phase 3, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2020-07-15
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid. Trials, record NCT00007020. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00007020
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
- NCT number
- NCT00007020
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 85 participants, actual
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- First posted
- 2000-12-07
- Last updated
- 2023-10-03
- Results first posted
- 2020-07-15
- Limitations and caveats
CAC-91-10-10 includes data from patients enrolled from 1992-2009. The study end date was arbitrarily chosen as data cut-off point; the disease requires life-long treatment, no final study visit was done. Many patients continued onto study CAC-002-01
Source ClinicalTrials.gov
Registered outcomes6
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients with excretion of atypical bile acids in urine by category, from worst status before treatment (baseline, BL) to best status on treatment (OT) |
| Secondary | Change in Liver Function Tests (LFTs) Measured in Serum | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients with elevations of liver function tests (alanine transaminase \[ALT\], aspartate transaminase \[AST\]) measured as multiples of the upper limit of normal (ULN) at baseline (worst value) and on treatment (best value) |
| Secondary | Liver Histology | At baseline (if no historical data were available) and between 1 and 6 months following treatment start. | Patients (number, percentage) with pathological findings for qualitative (the presence of inflammation, fibrosis, necrosis, giant cells and cholestasis) and quantitative (the degrees of the aforementioned histologic features) liver histopathology at baseline (BL) and on treatment (OT). |
| Secondary | Height and Weight | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Change in height/weight percentiles from baseline (worst value) to the best on-treatment value, based on CDC (Centres for Disease Control and Prevention, US) growth chart percentiles |
| Secondary | Adverse Events | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Number of patients with any adverse event |
| Other | Change in Bilirubin Measured in Serum | Baseline and on treatment (every 1, 3, or 6 months, depending on protocol version, for an average of 2.8 years) | Bilirubin concentration in serum at baseline and on treatment |
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 | Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Change in Liver Function Tests (LFTs) Measured in Serum | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients treated and with at least 1 pre-treatment and 1 post-Treatment assessment | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Liver Histology | At baseline (if no historical data were available) and between 1 and 6 months following treatment start. | All patients entered into the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Height and Weight | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients treated and with at least 1 pre-treatment and 1 post-treatment assessment | Percentile | MEAN |
| SECONDARY | Adverse Events | Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years | Patients entered into the study and treated | Participants | COUNT_OF_PARTICIPANTS |
| OTHER_PRE_SPECIFIED | Change in Bilirubin Measured in Serum | Baseline and on treatment (every 1, 3, or 6 months, depending on protocol version, for an average of 2.8 years) | All available bilirubin laboratory data are collected then grouped by bilirubin test type and collection time points (baseline vs. on treatment). Not all participant had available data, and each participant may have more than one type of bilirubin collected at a time point, and may have multiple on-treatment collections. | mg/dL | MEAN |
Source ClinicalTrials.gov
Results by group70
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | Baseline, no atypical bile acid excretion | 10 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | On treatment, no atypical bile acid excretion | 51 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | Baseline, slight atypical bile acid excretion | 11 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | On treatment, slight atypical bile acid excretion | 9 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | Baseline, significant atypical bile acid excretion | 16 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | OT, significant atypical bile acid excretion | 4 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | Baseline, marked atypical bile acid excretion | 33 | Participants | |
| Number of Participants With Excretion of Atypical Bile Acids in Urine by Category | Cholic Acid | On treatment, marked atypical bile acid excretion | 6 | Participants | |
| Change in Liver Function Tests (LFTs) Measured in Serum | Cholic Acid | Baseline, ALT <ULN | 14 | Participants | |
| Change in Liver Function Tests (LFTs) Measured in Serum | Cholic Acid | On treatment, ALT <ULN | 49 | Participants |
1 to 10 of 70Source ClinicalTrials.gov
See all 70 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 | 85 | |
| Overall Study | COMPLETED | Cholic Acid | 41 | |
| Overall Study | NOT COMPLETED | Cholic Acid | 44 |
Source ClinicalTrials.gov
Adverse events72
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 | Disease progression | General disorders | Cholic Acid | 1 | 79 | 1 |
| other | Injection site inflammation | General disorders | Cholic Acid | 1 | 79 | 1 |
| other | Malaise | General disorders | Cholic Acid | 1 | 79 | 1 |
| other | Pyrexia | General disorders | Cholic Acid | 7 | 79 | 7 |
| other | Hallucination | Psychiatric disorders | Cholic Acid | 1 | 79 | 1 |
| other | Ovarian cyst | Reproductive system and breast disorders | Cholic Acid | 1 | 79 | 1 |
| other | Ovarian failure | Reproductive system and breast disorders | Cholic Acid | 1 | 79 | 1 |
| other | Contusion | Injury, poisoning and procedural complications | Cholic Acid | 1 | 79 | 1 |
| other | Fracture | Injury, poisoning and procedural complications | Cholic Acid | 4 | 79 | 6 |
| other | Amino acid level increased | Investigations | Cholic Acid | 1 | 79 | 1 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cholic Acid | 13 | 79 |
| Total, other adverse events | Cholic Acid | 35 | 79 |
| Total, serious adverse events | Cholic Acid | 20 | 79 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28644367 | DERIVED |
Source PubMed
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/NCT00007020
GET https://conductscience.org/api/trials/v1/NCT00007020/results?page=2