Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia: Part II of a Prospective, Randomized Controlled, Multi-Institutional Trial
Who is this study for? Infant patients with Biliary Atresia
What treatments are being studied? Filgrastim
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The Investigators propose to test the hypothesis that GCSF enhances the clinical outcome of biliary atresia in a multi-institutional Phase 2 trial to prospectively evaluate the safety and efficacy of GCSF in each of the 2 groups of newly diagnosed BA patients: KBA (i.e., Kasai-operated) or NoK (i.e., patients who did not undergo Kasai surgery). Subjects who participate in the trial will be followed for 2 years.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 14 days
Maximum Age: 6 months
Healthy Volunteers: f
View:
• preliminary work up for cholestasis suspected or inconclusive diagnosis of BA.
• Serum Direct bilirubin \> 2 mg/dl,GGT\> 100 U/L
• Male or female infants with a gestational age\> 36 weeks
• Admission weight \> 2 kg
• Age \> 14 days - 180 days at diagnosis
• For Kasai operated subjects, Type 3 or 4 anatomy of BA
• For Kasai operated subjects, cholangiogram (if performed) diagnostic of BA
• Liver biopsy supporting BA diagnosis
Locations
United States
Oregon
Oregon Health & Science University (OHSU)
NOT_YET_RECRUITING
Portland
Other Locations
Pakistan
Aga Khan University
NOT_YET_RECRUITING
Karachi
Viet Nam
Nation Children's Hospital
RECRUITING
Hanoi
Children Hospital 1
ENROLLING_BY_INVITATION
Ho Chi Minh City
Contact Information
Primary
AiXuan Holterman, MD
Aithanh@uic.edu
8473340230
Backup
Sherri J Boykin
Sboykin@trclinical.com
9195596061
Time Frame
Start Date:2023-09-01
Estimated Completion Date:2025-10-31
Participants
Target number of participants:400
Treatments
Experimental: Kasai GCSF
The Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously by day 3 post Kasai surgery
No_intervention: Kasai no GCSF
The no GCSF group will not receive GCSF and receives the standard of care
Experimental: No Kasai GCSF
The No Kasai GCSF group will receive the standard of care PLUS 3 consecutive daily doses of 10 ug/kg of GCSF to be administered subcutaneously once the diagnosis of BA is established
No_intervention: No Kasai No GCSF
The No Kasai No GCSF group will receive the standard of care and will not receive GCSF