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Filgrastim for Biliary Atresia (BA_GCSF2b Trial)

Phase 2

Recruiting

Research Sponsored by Holterman, Ai-Xuan, M.D.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Admission weight > 2 kg

For Kasai operated subjects, cholangiogram (if performed) diagnostic of BA

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

BA_GCSF2b Trial Summary

This trial will test if GCSF can improve outcomes in biliary atresia patients who have or haven't had Kasai surgery. Participants will be followed for 2 years.

Who is the study for?

This trial is for infants over 14 days old diagnosed with Biliary Atresia, a liver condition. They must have specific blood test results and weigh more than 2 kg. It's not for those with major organ malformations, recent nutrition through IV, immediate need for a liver transplant, or certain blood conditions.Check my eligibility

What is being tested?

The study tests if the drug Filgrastim can improve outcomes in babies with Biliary Atresia. Some had surgery (Kasai procedure), others didn't. Participants are monitored for two years to assess safety and effectiveness of this treatment.See study design

What are the potential side effects?

While not specified here, common side effects of Filgrastim include bone pain, muscle ache, fever-like symptoms and redness or discomfort at injection site.

BA_GCSF2b Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My weight at admission was over 2 kg.

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My cholangiogram after Kasai surgery shows I have biliary atresia.

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My bile ducts were operated on and are classified as Type 3 or 4.

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I was diagnosed between 2 weeks and 6 months of age.

BA_GCSF2b Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

GCSF Response on Bile flow (KBA)

GCSF Response on transplant-free survival (NoK)

Secondary outcome measures

GCSF response on liver function (NoK)

GCSF response on liver function and outcome (KBA)

Side effects data

From 2014 Phase 3 trial • 118 Patients • NCT00003138

31%

Fatigue

23%

Pulmonary

15%

Infection

Cardiac adverse event

Diarrhea

Esophagitis

Hot flashes

Other toxicities

Hemorrhage

Skin

Weight loss

Neuropathy-clinical

Edema

Anemia

Neuropathy-psych

Abdominal cramps

100%

80%

60%

40%

20%

Study treatment Arm

Erythropoietin (300 Units/kg) and Filgrastim (Step 4)

Supportive Care (Step 1)

Erythropoietin (Step 1)

Erythropoietin (Cross-over; Step 2)

Erythropoietin (150 Units/kg) and Filgrastim (Step 3)

BA_GCSF2b Trial Design

4Treatment groups

Experimental Treatment

Active Control

Group I: No Kasai GCSFExperimental Treatment1 Intervention

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

Group II: Kasai GCSFExperimental Treatment1 Intervention

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

Group III: Kasai no GCSFActive Control1 Intervention

The no GCSF group will not receive GCSF and receives the standard of care

Group IV: No Kasai No GCSFActive Control1 Intervention

The No Kasai No GCSF group will receive the standard of care and will not receive GCSF

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Filgrastim

FDA approved

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Prometheus USAUNKNOWN

Big Leap ResearchUNKNOWN

Holterman, Ai-Xuan, M.D.Lead Sponsor

1 Previous Clinical Trials

6 Total Patients Enrolled

1 Trials studying Biliary Atresia

6 Patients Enrolled for Biliary Atresia

Media Library

No Kasai GCSF Clinical Trial Eligibility Overview. Trial Name: NCT04373941 — Phase 2

Biliary Atresia Research Study Groups: Kasai no GCSF, No Kasai No GCSF, No Kasai GCSF, Kasai GCSF

Biliary Atresia Clinical Trial 2023: No Kasai GCSF Highlights & Side Effects. Trial Name: NCT04373941 — Phase 2

No Kasai GCSF 2023 Treatment Timeline for Medical Study. Trial Name: NCT04373941 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment open to participants age 30 and over?

"This clinical trial requires patients to be aged between 14 days and 180 days old, in order for them to meet the eligibility requirements."

Answered by AI

Are there any enrolment opportunities available for this trial at present?

"Clinicaltrials.gov confirms that this medical experiment is actively enlisting participants, with the initial posting taking place on September 1st 2023 and the last edit occurring on August 11th 23."

Answered by AI

What safety measures have been taken to ensure No Kasai GCSF is suitable for human consumption?

"We have assigned No Kasai GCSF a score of 2 based on the limited safety data available from Phase 2 trials, which does not yet include evidence regarding efficacy."

Answered by AI

What clinical applications is No Kasai GCSF typically employed for?

"No, Kasai GCSF is not an uncommonly utilized medication for treating febrile neutropenia. It can also be administered to address numerous additional conditions including infection and myeloablative chemotherapy."

Answered by AI

Could you provide an overview of the experiments that have been conducted using No Kasai GCSF?

"Currently, 140 clinical trials are studying No Kasai GCSF. Of these investigations, 30 are in the advanced Phase 3 status. Most research is located in Saint Louis, Missouri; however a total of 4657 sites across the globe have launched studies for this medication."

Answered by AI

How many sites are administering this research project?

"This trial is presently enrolling from 4 distinct medical centres, including those in Ho Chi Minh City, Hanoi and Karachi. To minimise travel time for participants, it's essential to find the closest clinic available."

Answered by AI

How many participants are being sought for this research program?

"Affirmative. The latest information on clinicaltrials.gov implies that this medical research is currently looking for participants, with the initial posting occurring on September 1st 2023 and its most recent update being made on August 11th 2023. It hopes to find 400 patients between 4 separate sites."

Answered by AI

Do I qualify for enrollment in this experiment?

"This clinical trial is seeking 400 neonates aged between 14 days and 180 days with biliary atresia. In addition to these criteria, the following must be met: preliminary workup for cholestasis or inconclusive diagnosis of BA., serum direct bilirubin higher than 2 mg/dl; GGT levels exceeding 100 U/L, gestational age greater than 36 weeks; admission weight above 2 kg; Kasai operated subjects displaying type 3 or 4 anatomy of BA as seen on cholangiogram (if performed); liver biopsy confirming a diagnosis of BA."

Answered by AI

Recent research and studies

The Journal of PediatricsJournal

Total Serum Bilirubin Within 3 Months of Hepatoportoenterostomy Predicts Short-term Outcomes in Biliary Atresia

Shneider, Benjamin L., John C. Magee, Saul J. Karpen, Elizabeth B. Rand, Michael R. Narkewicz, Lee M. Bass, Kathleen Schwarz, et al.. 2016. “Total Serum Bilirubin Within 3 Months of Hepatoportoenterostomy Predicts Short-term Outcomes in Biliary Atresia”. The Journal of Pediatrics. Elsevier BV. doi:10.1016/j.jpeds.2015.11.058.

The Journal of PediatricsJournal

Total Serum Bilirubin Within 3 Months of Hepatoportoenterostomy Predicts Short-term Outcomes in Biliary Atresia

clinicaltrials.govStudy

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

2023. "Part II: Granulocyte-Colony Stimulating Factor Adjunct Therapy for Biliary Atresia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04373941.