Trial · NCT01589523
GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis
NCT01589523, Phase 3, Completed, Children's Hospital Medical Center, Cincinnati; results first posted 2022-05-12
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis. Trials, record NCT01589523. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01589523
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis
- NCT number
- NCT01589523
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 5 participants, actual
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Conditions
- Bile Acid Synthesis Defect, Inborn Error of Bile Acid Metabolism, Inborn Error of Bile Acid Conjugation
- Interventions
- Glycocholic Acid
- First posted
- 2012-05-02
- Last updated
- 2022-06-08
- Results first posted
- 2022-05-12
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. | Up to 10 years | This is the number of participants with bile acid amidation defects treated with oral glycocholic acid (15 milligrams/kilograms (mg/kg) of body weight/day (bw/day)) |
| Primary | Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared | Average of 6 months, average 12 months, and average of after year 1 to 10 years | Semi-quantitative descriptive evaluation of the levels of atypical bile acids in urine measured by mass spectrometry (FAB MS) based on a scale of 0 = absent or traces levels, 1 = low levels, 2 = moderate levels, 3 = high levels using the signal/noise ratio and intensity of ions. Atypical bile acids evaluated included m/z 407 (unconjugated cholic acid), m/z 471 (dihydroxy-choleanoic-sulfate) and m/z 583 (trihydroxy-choleanoic glucuronide). |
| Secondary | Changes in Liver Function Tests of ALT From Baseline to Post-treatment | Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10) | Liver function tests Alanine Aminotransferease (ALT) |
| Secondary | Change in Liver Function Test: AST From Baseline to Post-treatment | Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10) | Measure of Aspartate Aminotransferase (AST) |
| Secondary | Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment | Pre-treatment and post treatment (average of available timepoints after year 1 through year 10) | Measure Vitamin D levels nanograms per milliliter (ng/mL) |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. | Up to 10 years | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared | Average of 6 months, average 12 months, and average of after year 1 to 10 years | score on a scale | MEDIAN | |
| SECONDARY | Changes in Liver Function Tests of ALT From Baseline to Post-treatment | Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10) | units per liter (U/L) | MEAN | |
| SECONDARY | Change in Liver Function Test: AST From Baseline to Post-treatment | Comparison between baseline and post-treatment (average of available timepoints after year 1 through year 10) | units per liter (U/L) | MEDIAN | |
| SECONDARY | Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment | Pre-treatment and post treatment (average of available timepoints after year 1 through year 10) | Missing data on Vitamin D from 2 participants. | nanograms per milliliter (ng/mL) | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Conjugated Cholic Acid (GCA) for the Treatment of Inborn Errors in Bile Acid Synthesis Involving Side-chain Conjugation. | 15 mg/Kg Body Weight/Day | 5 | Participants | ||
| Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared | 10-15 mg/Kg Body Weight/Day | 6 months average | 1.4 | Full Range 0 to 2.0 | score on a scale |
| Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared | 10-15 mg/Kg Body Weight/Day | 12 months average | 1 | Full Range 1 to 1 | score on a scale |
| Evaluation of Levels of Atypical Bile Acid Metabolites After GCA Treatment Compared | 10-15 mg/Kg Body Weight/Day | average of year 1 to 10 | 1 | Full Range 0 to 3 | score on a scale |
| Changes in Liver Function Tests of ALT From Baseline to Post-treatment | Glycocholic Acid | Baseline | 43.8 | Standard Error 10.5 | units per liter (U/L) |
| Changes in Liver Function Tests of ALT From Baseline to Post-treatment | Glycocholic Acid | average of available timepoints after year 1 through year 10 | 31.7 | Standard Error 2.2 | units per liter (U/L) |
| Change in Liver Function Test: AST From Baseline to Post-treatment | Glycocholic Acid | Baseline | 65 | Standard Error 14.2 | units per liter (U/L) |
| Change in Liver Function Test: AST From Baseline to Post-treatment | Glycocholic Acid | average of available timepoints after year 1 through year 10 | 39 | Standard Error 3.5 | units per liter (U/L) |
| Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment | Glycocholic Acid | Baseline | 1.0 | Standard Error 0.8 | nanograms per milliliter (ng/mL) |
| Change in Vitamin D, 25-OH Measure From Baseline to Post-treatment | Glycocholic Acid | average of available timepoints after year 1 through year 10 | 25.7 | Standard Error 5.9 | nanograms per milliliter (ng/mL) |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Glycocholic Acid | 5 | |
| Overall Study | COMPLETED | Glycocholic Acid | 4 | |
| Overall Study | NOT COMPLETED | Glycocholic Acid | 1 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Glycocholic Acid | 0 | 5 |
| Total, other adverse events | Glycocholic Acid | 0 | 5 |
| Total, serious adverse events | Glycocholic Acid | 0 | 5 |
Source ClinicalTrials.gov
Papers2
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/NCT01589523
GET https://conductscience.org/api/trials/v1/NCT01589523/registered