What is the mechanism of action of this treatment?

Granulocyte Colony-Stimulating Factor (G-CSF) works by stimulating the bone marrow to produce more white blood cells, particularly neutrophils. This is important for Biliary Atresia patients because they are prone to infections and immune deficiencies. By increasing the white blood cell count, G-CSF helps enhance the immune response, reduce infection rates, and potentially improve overall clinical outcomes. This mechanism is vital in managing Biliary Atresia, where supporting the immune system and preventing infections are key aspects of treatment.

What side effects are associated with this type of intervention?

The most common treatment for Biliary Atresia is the Kasai procedure, which can be followed by liver transplantation if necessary. Granulocyte Colony-Stimulating Factor (GCSF) is being studied as an adjunct therapy to enhance clinical outcomes. Known side effects of GCSF include bone pain, fatigue, fever, and injection site reactions. In some cases, it can cause more severe effects such as spleen enlargement or rupture, acute respiratory distress syndrome, and allergic reactions. These side effects are important to consider when evaluating the overall treatment plan for Biliary Atresia.

What prior FDA approvals exist?

There is limited information on FDA-approved treatments specifically for Biliary Atresia that are similar to Granulocyte Colony-Stimulating Factor (GCSF). Biliary Atresia is primarily managed through surgical intervention, such as the Kasai procedure, and liver transplantation. GCSF is being studied in clinical trials for its potential to enhance clinical outcomes in Biliary Atresia by stimulating the bone marrow to produce more white blood cells, but it is not yet an FDA-approved treatment for this condition.

What other types of research exist?

Currently, there are no specific novel alternatives to GCSF mentioned for Biliary Atresia patients in the provided context. However, ongoing research in immunomodulatory therapies and advanced pharmacological interventions may offer potential alternatives in the future. Patients should consult with their healthcare providers to explore the most up-to-date treatment options available in 2024.

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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

Must not have

Purpura fulminans or unexplained vascular thrombosis

Major cardiac, renal, central nervous system (CNS) malformations

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 months

Awards & highlights

All Individual Drugs Already Approved

No Placebo-Only Group

Summary

This trial tests if a treatment can improve outcomes for newly diagnosed biliary atresia patients by helping their bodies produce more white blood cells. It includes patients who have had surgery and those who have not. The goal is to see if this treatment can help these patients fight infections and heal better.

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.

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had sudden severe bruising or unexplained blood clots.

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I do not have major heart, kidney, or brain malformations.

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I have had a brain bleed.

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I have a blockage in my digestive tract.

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I am currently fighting an infection in my body.

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I am eligible for a liver transplant in case of immediate liver failure.

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I had a Kasai procedure for type 1 or 2 biliary atresia.

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I do not have serious heart, lung, kidney, nerve, or metabolic diseases.

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

GCSF Response on Bile flow (KBA)

GCSF Response on transplant-free survival (NoK)

Secondary study objectives

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)

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

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 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Granulocyte Colony-Stimulating Factor (G-CSF) works by stimulating the bone marrow to produce more white blood cells, particularly neutrophils. This is important for Biliary Atresia patients because they are prone to infections and immune deficiencies. By increasing the white blood cell count, G-CSF helps enhance the immune response, reduce infection rates, and potentially improve overall clinical outcomes. This mechanism is vital in managing Biliary Atresia, where supporting the immune system and preventing infections are key aspects of treatment.

Granulocyte colony-stimulating factor attenuates liver damage by M2 macrophage polarization and hepatocyte proliferation in alcoholic hepatitis in mice.Use of granulocyte macrophage colony stimulating factor in children after orthotopic liver transplantation.