N/A
N=140
A Randomized, Double-Blinded, Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
Biliary Atresia
Bottom Line
View on ClinicalTrials.gov: NCT00294684 ↗Enrolled (actual)
140
Serious AEs
80.7%
Results posted
May 2017
Primary outcome: Primary: The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy — 58.6; 48.6 percentage of participants — p=0.43
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Corticosteroids (Drug); Placebo (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Primary completion
- Jan 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Percentage of Patients With Serum Total Bilirubin <1.5 mg/dL and With Native Liver at 6 Months After Portoenterostomy |
58.6; 48.6 | 0.43 |
| SECONDARY Survival With Native Liver at 24 Months of Age |
58.7; 59.4 | 0.99 |
| SECONDARY Serum Total Bilirubin Concentration |
3.5; 5.1 | 0.0973 |
| SECONDARY Total Bilirubin Concentration at 12 Months |
1.7; 3.7 | 0.0552 |
| SECONDARY Total Bilirubin Concentration at 24 Months of Age |
1.3; 1.6 | 0.6607 |
| SECONDARY Weight Z-Score |
-0.8; -0.8 | 0.1603 |
| SECONDARY Height Z-Score |
-0.7; -0.6 | 0.2801 |
| SECONDARY Presence of Ascites at 12 Months |
5; 3 | 0.41 |
| SECONDARY Presence of Ascites at 24 Months |
1; 3 | 0.29 |
Summary
The Children Liver Disease Research and Education Network (ChiLDREN) is conducting a clinical trial to evaluate whether long-term treatment with corticosteroids improves the outcome of the Kasai or gall-bladder Kasai in infants with biliary atresia. In this clinical trial, ChiLDREN is testing whether corticosteroid therapy following the Kasai will improve bile drainage and long term outcome in infants with biliary atresia. Subjects in this trial must start treatment within 72 hours of the Kasai procedure and be part of a prospective study of the natural history of biliary atresia also being conducted by ChiLDREN (http://www.clinicaltrials.gov/ct/show/NCT00061828?order=3).
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00294684). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.