Trial · NCT01438411

Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis

NCT01438411, Phase 3, Completed, Mirum Pharmaceuticals, Inc.; results first posted 2020-08-21

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis. Trials, record NCT01438411. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01438411

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis
NCT number: NCT01438411
Status: Completed
Phases: Phase 3
Enrollment: 53 participants, actual
Lead sponsor: Mirum Pharmaceuticals, Inc.
Conditions: Bile Acid Synthesis Defect
Interventions: Cholic Acid
First posted: 2011-09-22
Last updated: 2023-10-03
Results first posted: 2020-08-21

Source ClinicalTrials.gov

Registered outcomes10

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	At baseline, then every 12 months for an average of 3.5 years	The level of atypical urinary bile acid secretion was scored using a scale of: 0, normal; 1, slight; 2, significant; or 3, marked. A Cochran-Mantel-Haenszel (CMH) test with modified ridit scoring was used to compare the difference between the score at baseline and the worst post-baseline score during treatment with cholic acid in this single-arm trial.
Secondary	Evaluation of Serum Transaminases: ALT	At baseline, then every 12 months for an average of 3.5 years	Changes in ALT were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: \<ULN; ≥1 ULN but \<2 ULN; ≥2 ULN but \<3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.
Secondary	Evaluation of Serum Transaminases: AST	At baseline, then every 12 months for an average of 3.5 years	Changes in AST were evaluated in terms of elevations above the upper limit of normal (ULN) and were categorized as: \<ULN; ≥1 ULN but \<2 ULN; ≥2 ULN but \<3 ULN; and ≥3x ULN. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented.
Secondary	Clinical Laboratory Results: Bilirubin	At baseline, then every 12 months for an average of 3.5 years	Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for bilirubin. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Secondary	Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)	At baseline, then every 12 months for an average of 3.5 years	Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for GGT. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Secondary	Clinical Laboratory Results: Alkaline Phosphatase	At baseline, then every 12 months for an average of 3.5 years	Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for alkaline phosphatase. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Secondary	Clinical Laboratory Results: Prothrombin Time	At baseline, then every 12 months for an average of 3.5 years	Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial for prothrombin time. Changes from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial were presented in terms of descriptive statistics.
Secondary	Physical Examinations: Height	At baseline, then every 12 months for an average of 3.5 years	Changes in height percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.
Secondary	Physical Examinations: Body Weight	At baseline, then every 12 months for an average of 3.5 years	Changes in body weight percentiles from baseline to the worst post-baseline value during treatment with cholic acid in this single-arm trial.
Secondary	Incidence of Adverse Events	At baseline, then every 12 months for an average of 3.5 years	Number (%) of patients with any AE

Source ClinicalTrials.gov

Outcomes10

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	At baseline, then every 12 months for an average of 3.5 years	Patients with values	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Evaluation of Serum Transaminases: ALT	At baseline, then every 12 months for an average of 3.5 years	Patients with values	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Evaluation of Serum Transaminases: AST	At baseline, then every 12 months for an average of 3.5 years	Patients with values	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Clinical Laboratory Results: Bilirubin	At baseline, then every 12 months for an average of 3.5 years	Patients with values	mg/dL	MEAN
SECONDARY	Clinical Laboratory Results: Gamma Glutamyl Transferase (GGT)	At baseline, then every 12 months for an average of 3.5 years	Patients with values	U/L	MEAN
SECONDARY	Clinical Laboratory Results: Alkaline Phosphatase	At baseline, then every 12 months for an average of 3.5 years	Patients with values	U/L	MEAN
SECONDARY	Clinical Laboratory Results: Prothrombin Time	At baseline, then every 12 months for an average of 3.5 years	Patients with values	sec	MEAN
SECONDARY	Physical Examinations: Height	At baseline, then every 12 months for an average of 3.5 years	Patients with values	Height percentiles	MEAN
SECONDARY	Physical Examinations: Body Weight	At baseline, then every 12 months for an average of 3.5 years	Patients with values	Body weight percentiles	MEAN
SECONDARY	Incidence of Adverse Events	At baseline, then every 12 months for an average of 3.5 years		Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group37

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Baseline score: normal	25	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Baseline score: slight	6	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Baseline score: significant	10	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Baseline score: marked	10	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Worst-post-BL score: normal	25	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Worst-post-BL score: slight	11	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Worst-post-BL score: significant	6	Participants
Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry)	Cholic Acid	Worst-post-BL score: marked	9	Participants
Evaluation of Serum Transaminases: ALT	Cholic Acid	Baseline: ALT<ULN	27	Participants
Evaluation of Serum Transaminases: ALT	Cholic Acid	Baseline: ULN<=ALT<2 ULN	9	Participants

1 to 10 of 37Source ClinicalTrials.gov

See all 37 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Cholic Acid	53
Overall Study	COMPLETED	Cholic Acid	40
Overall Study	NOT COMPLETED	Cholic Acid	13

Source ClinicalTrials.gov

Adverse events39

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	Abdominal pain	Gastrointestinal disorders	Cholic Acid	6	53	8
other	Diarrhoea	Gastrointestinal disorders	Cholic Acid	6	53	7
other	Nasopharyngitis	Infections and infestations	Cholic Acid	4	53	4
other	Upper respiratory tract infection	Infections and infestations	Cholic Acid	8	53	8
other	Hepatic enzyme increased	Investigations	Cholic Acid	4	53	6
other	Vitamin D decreased	Investigations	Cholic Acid	6	53	6
other	Arthralgia	Musculoskeletal and connective tissue disorders	Cholic Acid	3	53	3
serious	Coagulopathy	Blood and lymphatic system disorders	Cholic Acid	3	53	3
serious	Bradykardia	Cardiac disorders	Cholic Acid	1	53	1
serious	Cardiac arrest	Cardiac disorders	Cholic Acid	1	53	1

1 to 10 of 39Source ClinicalTrials.gov

See all 39 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Cholic Acid	6	53
Total, other adverse events	Cholic Acid	21	53
Total, serious adverse events	Cholic Acid	19	53

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
31899729	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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT01438411

GET https://conductscience.org/api/trials/v1/NCT01438411/results?page=2

API reference For AI assistants