A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
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ClinicalTrials.gov Identifier: NCT00294684 |
Recruitment Status :
Completed
First Posted : February 22, 2006
Results First Posted : May 5, 2017
Last Update Posted : October 22, 2019
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Condition or disease | Intervention/treatment | Phase |
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Biliary Atresia | Drug: Corticosteroids Drug: Placebo | Not Applicable |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 141 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy in Infants With Biliary Atresia |
Study Start Date : | November 2005 |
Actual Primary Completion Date : | January 2013 |
Actual Study Completion Date : | January 2013 |
Arm | Intervention/treatment |
---|---|
Experimental: Corticosteroids |
Drug: Corticosteroids
Schedule and dosing of corticosteroids following portoenterostomy in infants with biliary atresia are listed below. Days 1-3: Methylprednisolone, IV-4mg/kg/day, divided BID Days 4-7: Prednisolone, PO-4mg/kg/day, divided BID Week 2: 4 mg/kg/day, divided BID Week 3: 2 mg/kg/day, divided BID Week 4: 2 mg/kg/day, divided BID Week 5: 1 mg/kg/day, once a day Week 6: 1 mg/kg/day, once a day Week 7: 0.8 mg/kg/day, once a day Week 8: 0.6 mg/kg/day, once a day Week 9: 0.4 mg/kg/day, once a day Week 10: 0.2 mg/kg/day, once a day Week 11: 0.1 mg/kg/day, once a day Week 12-13: 0.1 mg/kg/day, every other day Week 14: Stop Other Names:
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Placebo Comparator: Placebo |
Drug: Placebo
Schedule and dosing of placebo following portoenterostomy in infants with biliary atresia: Days 1-3: IV - normal saline 4 mg/kg/day, divided BID Days 4-7: PO placebo 4 mg/kg/day, divided BID Week 2: PO placebo 4 mg/kg/day, divided BID Week 3: PO placebo 2 mg/kg/day, divided BID Week 4: PO placebo 2 mg/kg/day, divided BID Week 5: PO placebo 1 mg/kg/day, once a day Week 6: PO placebo 1 mg/kg/day, once a day Week 7: PO placebo 0.8 mg/kg/day, once a day Week 8: PO placebo 0.6 mg/kg/day, once a day Week 9: PO placebo 0.4 mg/kg/day, once a day Week 10: PO placebo 0.2 mg/kg/day, once a day Week 11: PO placebo 0.1 mg/kg/day, once a day Week 12-13: PO placebo 0.1 mg/kg/day, once a day every other day Week 14: Stop |
- The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy [ Time Frame: Measurements will be made at 6 months after portoenterostomy ]
- Survival With Native Liver at 24 Months of Age [ Time Frame: Measurements will be made at 24 months of age ]
- Serum Total Bilirubin Concentration [ Time Frame: Measurements will be made at 3 months after portoenterostomy ]
- Total Bilirubin Concentration at 12 Months [ Time Frame: 12 Months post HPE ]
- Total Bilirubin Concentration at 24 Months of Age [ Time Frame: At 24 Months of Age ]
- Weight Z-Score [ Time Frame: HPE until 24 months of age ]weight for age Z-score (in subjects without ascites) over the course of the study
- Height Z-Score [ Time Frame: HPE to age 24 Months ]Height by Age Z-score over the course of the study
- Presence of Ascites at 12 Months [ Time Frame: 12 Months ]
- Presence of Ascites at 24 Months [ Time Frame: 24 Months ]
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin E [ Time Frame: 24 Months ]Vitamin E sufficiency is measured as the ratio of serum vitamin E/total lipids
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin K [ Time Frame: 24 Months ]Vitamin K sufficiency is measured by INR (international normalized ratio)
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin D [ Time Frame: 24 Months ]Vitamin D sufficiency is measured by the serum level of 25-hydroxy vitamin D
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin A [ Time Frame: 24 months ]Vitamin A sufficiency is defined as the molar ratio of serum retinol/retinol binding protein
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin D [ Time Frame: 12 Months ]Vitamin D sufficiency is measured by the serum level of 25-hydroxy vitamin D
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin K [ Time Frame: 12 Months ]Vitamin K sufficiency is measured by INR (international normalized ratio)
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin E [ Time Frame: 12 Months ]Vitamin E sufficiency is measured as the ratio of serum vitamin E/total lipids
- Serum Biomarkers of Sufficiency of Fat-soluble Vitamins - Vitamin A [ Time Frame: 12 months ]Vitamin A sufficiency is measured by the molar ratio of serum retinol/retinol binding protein

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Ages Eligible for Study: | up to 6 Months (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Portoenterostomy or gall bladder Kasai operation for biliary atresia within the previous 72 hours
- Post-conception age ≥ 36 weeks
- Weight at enrolment ≥ 2000 gm
- Written informed consent to participate in the study obtained prior to or within 72 hours of completion of portoenterostomy. (Note: Families of potential subjects may be approached prior to the portoenterostomy.)
Exclusion Criteria:
- Known immunodeficiency
- Diabetes mellitus
- Presence of significant systemic hypertension for age (persistent systolic blood pressure ≥112 mmHg)
- A serum indirect (unconjugated) bilirubin ≥ 5 mg/dL for infants under 4 weeks of age or ≥ 7 mg/dL for infants between 4 and 8 weeks of age
- Known sensitivity to corticosteroids
- Documented bacteremia or other tissue infection which is felt to be clinically relevant
- Known congenital infection or disease with herpes simplex virus, toxoplasmosis, or cytomegalovirus inclusion disease of the liver
- Infants whose mother is known to have human immunodeficiency virus infection
- Infants whose mother is known to be HBsAg or hepatitis C virus positive
- Infants with other severe concurrent illnesses such as neurological, cardiovascular, pulmonary, metabolic, endocrine, and renal disorders that would interfere with the conduct and results of the study
- Any other clinical condition that is a contraindication to the use of corticosteroid (e.g., bowel perforation)
- Infants who have received the live attenuated rotavirus vaccine (e.g., Rotateq) within 5 days prior to proposed administration of study drug

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00294684
United States, California | |
Children's Hospital Los Angeles | |
Los Angeles, California, United States, 90027 | |
University of California at San Francisco | |
San Francisco, California, United States, 94143 | |
United States, Colorado | |
The Children's Hospital | |
Aurora, Colorado, United States, 80045 | |
United States, Georgia | |
Children's Hospital of Atlanta - Emory University | |
Atlanta, Georgia, United States, 30322 | |
United States, Illinois | |
Children's Memorial Hospital | |
Chicago, Illinois, United States, 60614 | |
United States, Indiana | |
Riley Children's Hospital | |
Indianapolis, Indiana, United States, 46202 | |
United States, Maryland | |
Johns Hopkins School of Medicine | |
Baltimore, Maryland, United States, 21287 | |
United States, Missouri | |
Washington University School of Medicine | |
Saint Louis, Missouri, United States, 63110 | |
United States, New York | |
Mount Sinai Medical Center | |
New York, New York, United States, 10029 | |
United States, Ohio | |
Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
Children's Hospital at Pittsburgh | |
Pittsburgh, Pennsylvania, United States, 15213 | |
United States, Texas | |
Texas Children's Hospital/Baylor College of Medicine | |
Houston, Texas, United States, 77030 | |
United States, Washington | |
Children's Hospital and Regional Medical Center | |
Seattle, Washington, United States, 98105 |
Study Chair: | Ronald Sokol, MD | Children's Hospital Colorado | |
Study Director: | Ed Doo, MD | National Institute of Diabetes and Digestive and Kidney Disease (NIDDK) | |
Principal Investigator: | John Magee, MD | University of Michigan Medical Center, Ann Arbor | |
Study Director: | Averell Sherker, MD | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Publications of Results:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
ClinicalTrials.gov Identifier: | NCT00294684 |
Other Study ID Numbers: |
CHILDREN (START) (IND) U01DK062456 ( U.S. NIH Grant/Contract ) |
First Posted: | February 22, 2006 Key Record Dates |
Results First Posted: | May 5, 2017 |
Last Update Posted: | October 22, 2019 |
Last Verified: | October 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | Archived dataset will be entered into NIDDK Repository |
Supporting Materials: |
Study Protocol Analytic Code |
Time Frame: | Within six months of the publication date for the primary outcome publication or within two years of the date that the database is locked for analysis, whichever occurs first. |
URL: | https://repository.niddk.nih.gov/studies/start/?query=start |
biliary atresia hepatoportoenterostomy corticosteroids |
Biliary Atresia Bile Duct Diseases Biliary Tract Diseases Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities Methylprednisolone Prednisolone Anti-Inflammatory Agents Antiemetics Autonomic Agents |
Peripheral Nervous System Agents Physiological Effects of Drugs Gastrointestinal Agents Glucocorticoids Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Neuroprotective Agents Protective Agents Antineoplastic Agents, Hormonal Antineoplastic Agents |