An Early Phase 1 interventional study of Granulocyte Colony-Stimulating Factor in Biliary Atresia, sponsored by Holterman, Ai-Xuan, M.D.. Completed at 2 sites in 2 countries. Open to participants aged 2 Weeks to 180 Days. Per ClinicalTrials.gov, last updated 2020-02-17.
Sponsored by Holterman, Ai-Xuan, M.D. · Early Phase 1, Interventional, and Supportive care
Among 22 studies in Biliary Atresia with a site in United States — 8 open to participants now, 10 under way, 2 of them phase 3. Biliary Atresia · United States.
Among the 22 studies in Biliary Atresia with a site in United States, this one is the 14th largest by enrollment of the 14 interventional ones that give a count, is one of 2 that started in 2018, is one of 3 in phase 1, and was the 12th registered.
77 studies on the registry are indexed under Biliary Atresia; 25 are open to participants now.
Browse Biliary Atresia studies →8 of the 22 studies in Biliary Atresia with a site in United States are open now. Recruiting first, the newest first:
University of Alabama at Birmingham · Sites in United States
Ages 2 wk–4 mo · Healthy volunteers
Boston Children's Hospital · Sites in United States
Ages 0 days–3 mo
Holterman, Ai-Xuan, M.D. · Sites in United States, Pakistan, Vietnam
Ages 14 days–180 days
University of Pittsburgh · Sites in United States
Sanford Health · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 21 Biliary Atresia studies recruiting now, 3 (14%) set no upper age limit and 18 (86%) stop at 75 or younger; 21 take children under 18; 6 take healthy volunteers.
This study: 2 Weeks to 180 Days · all sexes · no healthy volunteers.
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22 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 5 · Phase 2 6 · Phase 3 3
First in 2000; this study’s year is marked. 2 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 6 is below the median of 45 across 43 interventional studies indexed under Biliary Atresia.
Median enrollment 53 across the 14 interventional studies, 776 across the 8 observational. Of the 11 that completed or were terminated, 5 (45%) have results posted.
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4 of 22 have an industry lead sponsor. The 10 most frequent:
Holterman, Ai-Xuan, M.D. is the lead sponsor of 2 studies on the registry; 1 is open to participants now.
Holterman, Ai-Xuan, M.D. has 2 studies filed under Biliary Atresia — fewer than ten, so there is no comparison to draw.
1 of the studies in Biliary Atresia with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07250854University of Alabama at BirminghamThe Use of Near-Infrared Fluorescence Cholangiography With Indocyanine Green (ICG) in the Work Up of Neonatal Cholestasis | 2027-03 |
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Among the 22 studies in Biliary Atresia with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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The Investigators propose to test the hypothesis that GCSF therapy enhances the clinical outcome of Kasai operated Biliary Atresia (BA) patients. In this study, Investigators will conduct a dose determination for GCSF use in post Kasai subjects to support a future phase 2 efficacy study. The first 3 post Kasai BA subjects with liver biopsy-confirmed BA will be given 5 ug/kg/d of GCSF in 3 daily subcutaneous doses starting on post Kasai day 3. A second group of 3 subjects will be assigned to the 10 ug/Kg/d dose after the 5ug/kg/d dose has been proven to be safe. The levels of circulating hematopoietic stem cells and a 1-month safety profile will be analyzed.
In BA, neonatal fibrous obliteration of the biliary tract obstructs biliary drainage and promotes biliary fibrosis. BA is the leading cause of pediatric chronic end-stage liver disease and pediatric liver transplantation. Relief of cholestasis by the Kasai portoenterostomy is only partly successful with continued progression of fibrosis to hepatic insufficiency and, for long term survival, with eventual need for liver transplantation in the majority of the patients. In animal models of liver injury, GCSF enhances hematopoietic stem cell HSC mobilization and engraftment in the liver with associated improved liver repair response and attenuated hepatic necrosis and fibrosis. Randomized controlled trials of GCSF intervention for chronic liver failure in adult patients with acute hepatic decompensation showed improved short-term survival and hepatic indices such the model for end-stage liver disease (MELD) scores.
The Investigators propose that post Kasai GCSF therapy attenuates biliary fibrosis and progression to cirrhosis. The objectives are meant to demonstrate that Kasai-GCSF sequential therapy improves biliary drainage, and delays the progression of hepatic insufficiency. Toward this goal, Investigators will first evaluate in post Kasai subjects the maximum tolerated dose of GCSF in mobilizing circulating CD34+ hematopoietic stem cells, with the limiting dose based on GCSF-related severe adverse effects. A one-month safety of GCSF will be tested with the 2 standard doses of 5 ug/kg/d and 10 ug/kg/d.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
G-CSF is a glycoprotein produced by monocytes, fibroblasts, and endothelial cells. Filgrastim is a human granulocyte colony stimulating factor (G-CSF) produced by recombinant DNA technology with NEUPOGEN® as the Amgen Inc. trademark for filgrastim. G-CSF regulates the production, proliferation and differentiation of neutrophils and hematopoietic stem cell precursors within the bone marrow leading to dose-dependent increase in circulating neutrophils and hematopoietic stem cells in the blood. It is indicated to reduce the incidence of infection in patients with severe neutropenia, for neutrophil recovery in neutropenic patients with bone marrow depletion, to mobilize hematopoietic progenitor stem cell for collection by leukapheresis in hematopoietic stem cell transplantation.
Also known as: Filgrastim
Dose determination GCSF
To determine the maximum tolerated dose of GCSF based on GCSF dose limiting toxicity and the extent of peripheral blood stem cell mobilization as measured by increases in CD34+cells with upper levels limited by white blood cells (WBCs) less than 50,000 per microliter (mcL) of blood.
Time frame: 13 months
Plan to share: No
Ran from Jan 2018 to primary completion in Jan 2020, 24 months.
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This study is completed, as verified in Feb 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Granulocyte Colony-Stimulating Factor
Holterman, Ai-Xuan, M.D.