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GCSF Adjunct Therapy for Biliary Atresia

NCT03395028 · Status: COMPLETED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2020-02-17

No results posted yet for this study

Summary

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.

Conditions

  • Biliary Atresia

Interventions

DRUG

Granulocyte Colony-Stimulating Factor

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.

Sponsors & Collaborators

  • T. Rose Clinical, Inc., United States

    collaborator UNKNOWN

  • Children's National Research Institute

    collaborator OTHER

  • Big Leap Research

    collaborator OTHER

  • Holterman, Ai-Xuan, M.D.

    lead INDIV

Principal Investigators

  • Evan P Nadler, MD · Children's National Research Institute

Study Design

Allocation
NA
Purpose
SUPPORTIVE_CARE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Weeks
Max Age
180 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-15
Primary Completion
2020-01-31
Completion
2020-01-31
FDA Drug
Yes

Countries

  • United States
  • Vietnam

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03395028 on ClinicalTrials.gov