Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia

Phase 2

Recruiting

• Conditions: Biliary Atresia
• Interventions: Drug: Filgrastim

• Registration Number: NCT04373941
• Lead Sponsor: Holterman, Ai-Xuan, M.D.

Brief 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 ...

Detailed Description

This is a prospective, randomized, multi-institutional trial in KBA and NoK subjects to be conducted under a Food and Drug Administration approved Investigational New Drug application.
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Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2019-08-13
• Study First Submitted QC: 2020-05-01
• Study First Posted: 2020-05-05
• Status Verified: 2023-09
• Study Start: 2023-09-01
• Last Update Submitted: 2023-09-07
• Last Update Posted: 2023-09-08
• Primary Completion: 2024-08-31
• Study Completion: 2025-10-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 400

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Kasai GCSF	Filgrastim	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 Kasai GCSF	Filgrastim	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

Primary Outcome Measures

Name	Time	Method
GCSF Response on transplant-free survival (NoK)	24 months	For NoK subjects: Changes at 6, 12, 18 and 24 months-transplant free survival
GCSF Response on Bile flow (KBA)	3 months	For KBA subjects: Bile flow as measured by the percentage of subjects with total bilirubin\< 2 mg/dL at 3 months post-Kasai.

Secondary Outcome Measures

Name	Time	Method
GCSF response on liver function (NoK)	24 months	NoK subjects: Changes in Pediatric end-stage liver disease (PELD) score at 6, 12, 18, and 24 months after GCSF treatment.
GCSF response on liver function and outcome (KBA)	24 months	KBA subjects: Percentage of patients with cholangitis-free transplant-free survival at 6, 12, 18 and 24 months

Trial Locations

Locations (4)

Nation Children's Hospital; 🇻🇳 Hanoi, Dong Da District, Vietnam

Children Hospital 1; 🇻🇳 Ho Chi Minh City, Vietnam

Aga Khan University; 🇵🇰 Karachi, Pakistan

Oregon Health & Science University (OHSU); 🇺🇸 Portland, Oregon, United States