Trial · NCT00294684

A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy

NCT00294684, Not applicable, Completed, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); results first posted 2017-05-05

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy. Trials, record NCT00294684. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00294684

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Trial

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

Title: A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
NCT number: NCT00294684
Status: Completed
Phases: Not applicable
Enrollment: 141 participants, actual
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Conditions: Biliary Atresia
Interventions: Corticosteroids, Placebo
First posted: 2006-02-22
Last updated: 2019-10-22
Results first posted: 2017-05-05

Source ClinicalTrials.gov

Registered outcomes17

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

List	Outcome	Time frame	Description
Primary	The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy	Measurements will be made at 6 months after portoenterostomy
Secondary	Survival With Native Liver at 24 Months of Age	Measurements will be made at 24 months of age
Secondary	Serum Total Bilirubin Concentration	Measurements will be made at 3 months after portoenterostomy
Secondary	Total Bilirubin Concentration at 12 Months	12 Months post HPE
Secondary	Total Bilirubin Concentration at 24 Months of Age	At 24 Months of Age
Secondary	Weight Z-Score	HPE until 24 months of age	weight for age Z-score (in subjects without ascites) over the course of the study
Secondary	Height Z-Score	HPE to age 24 Months	Height by Age Z-score over the course of the study
Secondary	Presence of Ascites at 12 Months	12 Months
Secondary	Presence of Ascites at 24 Months	24 Months
Other	Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin E	24 Months	Vitamin E sufficiency is measured as the ratio of serum vitamin E/total lipids

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes17

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy	Measurements will be made at 6 months after portoenterostomy	Intent to Treat	percentage of participants	NUMBER
SECONDARY	Survival With Native Liver at 24 Months of Age	Measurements will be made at 24 months of age	Intent to Treat	percentage of participants	NUMBER
SECONDARY	Serum Total Bilirubin Concentration	Measurements will be made at 3 months after portoenterostomy	Intent to treat patients with 3 month bilirubin available are analyzed. There are no imputations for unobserved data. Patients with missing data are simply not included.	mg/dL	MEAN
SECONDARY	Total Bilirubin Concentration at 12 Months	12 Months post HPE	Intent to treat patients with 12 month bilirubin available are analyzed. There are no imputations for unobserved data. Patients with missing data are simply not included.	mg/dL	MEAN
SECONDARY	Total Bilirubin Concentration at 24 Months of Age	At 24 Months of Age	Intent to treat patients with 24 month bilirubin available are analyzed. There are no imputations for unobserved data. Patients with missing data are simply not included.	mg/dL	MEAN
SECONDARY	Weight Z-Score	HPE until 24 months of age		Z-score	MEAN
SECONDARY	Height Z-Score	HPE to age 24 Months		Z-score	MEAN
SECONDARY	Presence of Ascites at 12 Months	12 Months	Participants with their native liver at 12 months	participants	NUMBER
SECONDARY	Presence of Ascites at 24 Months	24 Months	Participants with their native liver at 24 months	participants	NUMBER
OTHER_PRE_SPECIFIED	Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin E	24 Months	Although the protocol specified analyses of serum biomarkers as secondary outcomes, the intent was to only perform these analyses if the primary endpoint showed sufficient efficacy. Given the lack of efficacy of steroids (vs placebo), the analyses of fat-soluble vitamins were not performed; thus, data are not summarized for this assessment.

1 to 10 of 17Source ClinicalTrials.gov

See all 17 outcomes

Results by group18

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

Outcome	Group	Value	Spread	Units
The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy	Corticosteroids	58.6		percentage of participants
The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy	Placebo	48.6		percentage of participants
Survival With Native Liver at 24 Months of Age	Corticosteroids	58.7		percentage of participants
Survival With Native Liver at 24 Months of Age	Placebo	59.4		percentage of participants
Serum Total Bilirubin Concentration	Corticosteroids	3.5	Standard Deviation 5.32	mg/dL
Serum Total Bilirubin Concentration	Placebo	5.1	Standard Deviation 6.15	mg/dL
Total Bilirubin Concentration at 12 Months	Corticosteroids	1.7	Standard Deviation 3.63	mg/dL
Total Bilirubin Concentration at 12 Months	Placebo	3.7	Standard Deviation 6.63	mg/dL
Total Bilirubin Concentration at 24 Months of Age	Corticosteroids	1.3	Standard Deviation 3.37	mg/dL
Total Bilirubin Concentration at 24 Months of Age	Placebo	1.6	Standard Deviation 3.73	mg/dL

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	Corticosteroids	70
Overall Study	STARTED	Placebo	70
Overall Study	COMPLETED	Corticosteroids	65
Overall Study	COMPLETED	Placebo	62
Overall Study	NOT COMPLETED	Corticosteroids	5
Overall Study	NOT COMPLETED	Placebo	8

Source ClinicalTrials.gov

Adverse events26

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
serious	Congenital	Congenital, familial and genetic disorders	Corticosteroids	1	70	1
serious	Congenital	Congenital, familial and genetic disorders	Placebo	1	70	1
serious	Gastrointestinal	Gastrointestinal disorders	Corticosteroids	5	70	6
serious	Gastrointestinal	Gastrointestinal disorders	Placebo	3	70	3
serious	Hepatic	Hepatobiliary disorders	Corticosteroids	22	70	41
serious	Hepatic	Hepatobiliary disorders	Placebo	16	70	30
serious	Immunological	Immune system disorders	Corticosteroids	1	70	1
serious	Immunological	Immune system disorders	Placebo	0	70	0
serious	Infectious	Infections and infestations	Corticosteroids	50	70	129
serious	Infectious	Infections and infestations	Placebo	46	70	109

1 to 10 of 26Source ClinicalTrials.gov

See all 26 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Corticosteroids
Total, all-cause mortality	Placebo
Total, other adverse events	Corticosteroids	0	70
Total, other adverse events	Placebo	0	70
Total, serious adverse events	Corticosteroids	57	70
Total, serious adverse events	Placebo	56	70

Source ClinicalTrials.gov

Papers6

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
22891232	DERIVED	PubMed
24794368	RESULT	PubMed
25419594	DERIVED	PubMed
26725209	DERIVED	PubMed
29519540	DERIVED	PubMed
30244988	DERIVED	PubMed

Grants1

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.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
U01DK062456	Childhood Liver Disease Research Network Data Coordinating Center	MAGEE, JOHN C; MERION, ROBERT M	UNIVERSITY OF MICHIGAN AT ANN ARBOR	NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES	2002 to 2019

Source NIH RePORTER

Trial comparisons3

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Corticosteroids vs Placebo: Presence of Ascites at 24 Months	Trial comparisons
Corticosteroids vs Placebo: Presence of Ascites at 12 Months	Trial comparisons
Corticosteroids vs Placebo: serious adverse events	Trial comparisons

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.

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Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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