Using GCSF to improve outcomes in biliary atresia patients
Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia: Part II of a Prospective, Randomized Controlled, Multi-Institutional Trial
This study is testing if a medication called GCSF can help improve health outcomes for babies with biliary atresia after their surgery or diagnosis.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 400 (estimated) |
| Ages | 14 Days to 180 Days |
| Sex | All |
| Sponsor | Holterman, Ai-Xuan, M.D. Indiv |
| Locations | 4 sites (Portland, Oregon and 3 other locations) |
| Trial ID | NCT04373941 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and efficacy of Granulocyte-Colony Stimulating Factor (GCSF) in enhancing clinical outcomes for patients with biliary atresia. It involves a prospective, randomized, multi-institutional approach, where newly diagnosed biliary atresia patients are divided into two groups: those who have undergone the Kasai procedure and those who have not. Participants will receive GCSF or a placebo for three consecutive days following their diagnosis or surgery, and will be monitored for two years to assess the treatment's impact. The study is conducted under an FDA-approved Investigational New Drug application.
Who should consider this trial
Good fit: Ideal candidates include infants aged 14 to 180 days with a confirmed diagnosis of biliary atresia, either post-Kasai surgery or those who are not surgical candidates.
Not a fit: Patients with immediate access to liver transplantation or those with severe comorbidities may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the clinical outcomes for infants diagnosed with biliary atresia.
How similar studies have performed: While this approach is novel in the context of biliary atresia, previous studies have shown promise in using GCSF for other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion criteria 1. preliminary work up for cholestasis suspected or inconclusive diagnosis of BA. 2. Serum Direct bilirubin \> 2 mg/dl,GGT\> 100 U/L 3. Male or female infants with a gestational age\> 36 weeks 4. Admission weight \> 2 kg 5. Age \> 14 days - 180 days at diagnosis 6. For Kasai operated subjects, Type 3 or 4 anatomy of BA 7. For Kasai operated subjects, cholangiogram (if performed) diagnostic of BA 8. Liver biopsy supporting BA diagnosis Exclusion criteria 1. Patients having access to liver transplantation for immediate liver failure 2. Prior Kasai patients 3. Major cardiac, renal, central nervous system (CNS) malformations 4. Intracranial hemorrhage 5. History of recent total parenteral nutrition (TPN) use within the last 2 weeks 6. Gl tract obstruction For Kasai-operated subjects: Type 1 or 2 biliary atresia anatomy 7. Current systemic infection 8. WBC \> 20,000 cells/uL 9. Platelet count \< 20,000 cells/uL or \>1 million cells/uL 10. Concurrent respiratory, metabolic, neurological, cardiovascular, metabolic, and renal illness 11. Elevated serum creatinine \> 1 mg/dL 12. Purpura fulminans or unexplained vascular thrombosis
Where this trial is running
Portland, Oregon and 3 other locations
- Oregon Health & Science University (OHSU) — Portland, Oregon, United States (Not_yet_recruiting)
- Aga Khan University — Karachi, Pakistan (Not_yet_recruiting)
- Nation Children's Hospital — Hanoi, Dong Da District, Vietnam (Recruiting)
- Children Hospital 1 — Ho Chi Minh City, Vietnam (Enrolling_by_invitation)
Study contacts
- Study coordinator: AiXuan Holterman, MD
- Email: Aithanh@uic.edu
- Phone: 8473340230
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.