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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 belowSelect...
My weight at admission was over 2 kg.
Select...
My cholangiogram after Kasai surgery shows I have biliary atresia.
Select...
My bile ducts were operated on and are classified as Type 3 or 4.
Select...
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:Select...
I have had sudden severe bruising or unexplained blood clots.
Select...
I do not have major heart, kidney, or brain malformations.
Select...
I have had a brain bleed.
Select...
I have a blockage in my digestive tract.
Select...
I am currently fighting an infection in my body.
Select...
I am eligible for a liver transplant in case of immediate liver failure.
Select...
I had a Kasai procedure for type 1 or 2 biliary atresia.
Select...
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
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 • NCT0000313831%
Fatigue
23%
Pulmonary
15%
Infection
8%
Cardiac adverse event
8%
Diarrhea
8%
Esophagitis
8%
Hot flashes
8%
Other toxicities
8%
Hemorrhage
8%
Skin
8%
Weight loss
8%
Neuropathy-clinical
8%
Edema
8%
Anemia
8%
Neuropathy-psych
8%
Abdominal cramps
100%
80%
60%
40%
20%
0%
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
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.
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.
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had sudden severe bruising or unexplained blood clots.I do not have major heart, kidney, or brain malformations.I have had a brain bleed.I have a blockage in my digestive tract.My weight at admission was over 2 kg.I have been on IV nutrition in the last 2 weeks.I am currently fighting an infection in my body.My cholangiogram after Kasai surgery shows I have biliary atresia.My baby was born after more than 36 weeks of pregnancy.I am eligible for a liver transplant in case of immediate liver failure.I had a Kasai procedure for type 1 or 2 biliary atresia.My bile ducts were operated on and are classified as Type 3 or 4.I was diagnosed between 2 weeks and 6 months of age.I do not have serious heart, lung, kidney, nerve, or metabolic diseases.
Research Study Groups:
This trial has the following groups:- Group 1: Kasai no GCSF
- Group 2: No Kasai No GCSF
- Group 3: No Kasai GCSF
- Group 4: Kasai GCSF
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.