Ignite Creation Date:
2024-05-05 @ 4:42 PM
Last Modification Date:
2024-10-26 @ 9:23 AM
Study NCT ID:
NCT00294684
Status:
COMPLETED
Last Update Posted:
2019-10-22
First Post:
2006-02-21
Brief Title:
A Randomized Double-Blinded Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy
Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Organization:
National Institute of Diabetes and Digestive and Kidney Diseases NIDDK
Study Overview
Official Title:
A Randomized Double-Blinded Placebo-Controlled Trial of Corticosteroid Therapy Following Portoenterostomy in Infants With Biliary Atresia
Status:
COMPLETED
Status Verified Date:
2019-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 httpwwwclinicaltrialsgovctshowNCT00061828order3
Detailed Description:
This is a multi-center randomized double-blinded placebo-controlled trial to prospectively determine the efficacy of corticosteroids on the outcome of infants with biliary atresia The trial will be conducted by the NIDDK-funded network of 15 clinical centers comprising the Biliary Children Liver Disease Research and Education Network ChiLDREN whose goal is to study the etiology pathogenesis diagnosis and treatment of infants with biliary atresia For the trial our overall hypothesis is that therapy with corticosteroids following portoenterostomy including gall bladder Kasai procedure will improve bile drainage and long-term outcome in infants with biliary atresia This hypothesis will be tested through the following specific aims and hypotheses
Aim 1 To determine whether corticosteroid therapy decreases serum bilirubin concentration after portoenterostomy
Aim 2 To determine whether corticosteroid treatment after portoenterostomy will improve outcome as defined by survival without transplantation at 24 months of age
Aim 3 To determine whether corticosteroid treatment after portoenterostomy will improve growth of infants with biliary atresia
Aim 4 To determine whether corticosteroid treatment improves biochemical indicators of each of the fat-soluble vitamins after supplementation with standard doses
Aim 5 To determine whether corticosteroid treatment after portoenterostomy will decrease the incidence of persistent ascites or ascites that requires medical treatment
The significance of the proposed trial is that it will determine whether corticosteroids are an effective medical treatment to improve bile drainage and long-term outcome and whether its use reduces the need for liver transplantation in infants with biliary atresia
Subjects will be recruited from patients enrolled in the ChiLDREN prospective observational database study who undergo portoenterostomy or portochelecystostomy gall bladder Kasai for biliary atresia
The Primary outcome measure is the percentage of patients with serum total bilirubin 15 mgdL and with native liver at 6 months after portoenterostomy
Secondary outcome measures are
1 Serum total bilirubin concentration and also at 3 months after portoenterostomy
2 Survival with native liver at 24 months of age
3 Growth
1 Weight for age Z-score in patients without ascites
2 Height for age Z score
4 Serum biomarkers of sufficiency of fat-soluble vitamins
1 Vitamin A molar ratio of serum retinolretinol binding protein
2 Vitamin D serum level of 25-hydroxy vitamin D
3 Vitamin E ratio of serum vitamin Etotal lipids
4 Vitamin K International Normalized Ratio INR
5 Presence of ascites
All measurements will be made at 12 and 24 months of age unless noted otherwise
Aim 1 To determine whether corticosteroid therapy decreases serum bilirubin concentration after portoenterostomy
Aim 2 To determine whether corticosteroid treatment after portoenterostomy will improve outcome as defined by survival without transplantation at 24 months of age
Aim 3 To determine whether corticosteroid treatment after portoenterostomy will improve growth of infants with biliary atresia
Aim 4 To determine whether corticosteroid treatment improves biochemical indicators of each of the fat-soluble vitamins after supplementation with standard doses
Aim 5 To determine whether corticosteroid treatment after portoenterostomy will decrease the incidence of persistent ascites or ascites that requires medical treatment
The significance of the proposed trial is that it will determine whether corticosteroids are an effective medical treatment to improve bile drainage and long-term outcome and whether its use reduces the need for liver transplantation in infants with biliary atresia
Subjects will be recruited from patients enrolled in the ChiLDREN prospective observational database study who undergo portoenterostomy or portochelecystostomy gall bladder Kasai for biliary atresia
The Primary outcome measure is the percentage of patients with serum total bilirubin 15 mgdL and with native liver at 6 months after portoenterostomy
Secondary outcome measures are
1 Serum total bilirubin concentration and also at 3 months after portoenterostomy
2 Survival with native liver at 24 months of age
3 Growth
1 Weight for age Z-score in patients without ascites
2 Height for age Z score
4 Serum biomarkers of sufficiency of fat-soluble vitamins
1 Vitamin A molar ratio of serum retinolretinol binding protein
2 Vitamin D serum level of 25-hydroxy vitamin D
3 Vitamin E ratio of serum vitamin Etotal lipids
4 Vitamin K International Normalized Ratio INR
5 Presence of ascites
All measurements will be made at 12 and 24 months of age unless noted otherwise
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| U01DK062456 | NIH | None | httpsreporternihgovquickSearchU01DK062456 |