Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency

An Open-Label, Single-Arm, Historically Controlled, Prospective, Multi-Center Phase III Study to Evaluate the Pharmacokinetics and Safety of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency

The purpose of this study is to evaluate the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects with Primary Humoral Immunodeficiency (PHID).

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Deficiency
• Intervention / Treatment: Biological: GC5107

Detailed Description

This is a prospective, open-label, single-arm, historically controlled, multi-center Phase III study to assess the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects aged ≥ 2 years and < 17 years with PHID.

Subjects will receive intravenous infusions of the investigational product at the same dose and interval as used for their previous Immunoglobulin intravenous (IGIV) maintenance therapy. GC5107 will be infused every 21 or 28 days for a period of 12 months.

• Study Type: Interventional
• Enrollment (Anticipated): 24
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Hyejoo Kim; Phone Number: +82-31-260-9192; Email: hyejoo.kim@gccorp.com

Study Locations

• United States
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73131
      • Withdrawn
      • Oklahoma Institute of Allergy & Asthma Clinical Research, LLC
  • Texas
    • Dallas, Texas, United States, 75230
      • Recruiting
      • Allergy Partners of North Texas Research
  • Virginia
    • Fairfax, Virginia, United States, 22030
      • Recruiting
      • Lysosomal and Rare Disorders Research and Treatment Center, Inc.
    • Richmond, Virginia, United States, 23219
      • Recruiting
      • Children's Hospital of Richmond at VCU
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Withdrawn
      • University of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 16 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subject must be ≥ 2 to < 17 years of age, at the time of signing the informed consent
• Pediatric subject has a confirmed and documented clinical diagnosis of Primary Humoral Immunodeficiency, including hypogammaglobulinemia or agammaglobulinemia
• Subject who has received 300 - 900 mg/kg of IGIV therapy at 21 or 28 day intervals for at least 3 months prior to this study
• Subject who has at least 2 documented plasma IgG trough level of ≥ 500 mg/dL at two infusion cycles (21 or 28 days) within 12 months prior to enrollment
• Subject who is willing to comply with all requirements of the protocol

Exclusion Criteria:

• Subject who has a history of clinically significant reactions or hypersensitivity to IGIV or other injectable forms of IgG
• Subject who has IgA deficiency and is known to have antibodies to IgA
• Subject who has secondary immunodeficiency
• Subject who has participated in another clinical study (other than an IGIV study) within 3 weeks prior to screening
• Subject who has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency or isolated IgA deficiency, or who has clinically significant impairment of cellular or innate immunity at the discretion of the Investigator
• Subject who has received blood products other than human albumin or human immune globulin within 6 months prior to enrollment

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GC5107; Immune Globulin Intravenous (Human), 10% Liquid	Biological: GC5107; Intravenously infused at a dose of 300 - 900 mg per kg (of body weight) every 21 or 28 days for 12 months; Other Names: GCC 10% IGIV; Immune Globulin Intravenous (Human), 10% Liquid

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Pharmacokinetic (PK) Plasma concentration-time curve of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Half-life of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Area under the curve of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Volume of distribution of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Maximum concentration of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Minimum concentration of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Time of maximum concentration of total IgG		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Clearance of total IgG		before and after 5th infusion (12 or 16 weeks)
Trough serum total IgG levels before each infusion of GC5107 in all subjects and the interval between infusions		12 months
The proportion of infusions with temporally associated adverse events (AEs) that occur during or within 1 hour, 24 hours, and 72 hours following an infusion of investigational product	AEs that occur during or within 1 hour, 24 hours, and 72 hours following each infusion during 12 months of the study period	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Pharmacokinetic (PK) Maximum concentration of IgG subclasses		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Minimum concentration of IgG subclasses		before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Half-life of IgG subclasses		before and after 5th infusion (12 or 16 weeks)
Trough serum level of IgG subclasses and specific IgG antibodies before Infusion 1 and 13 (for subjects on 28-day infusion schedule) or Infusion 1 and 17 (for subjects on 21-day infusion schedule)		12 months
Number and proportion of subjects who failed to meet the target IgG trough level (500 mg/dL) at any time point equal to or subsequent to 5th infusion (estimated 5 half-lives)		12 months
The overall incidence of all AEs that occur during or within 1 hour, 24 hours, and 72 hours following an infusion of investigational product	AEs that occur during or within 1 hour, 24 hours, and 72 hours following each infusion during 12 months of the study period	12 months
The frequency of all AEs that occur during the study regardless of the investigator's assessment of their relationship to investigational product		13 months (12 months of treatment + 1 month of follow-up)
The frequency of suspected adverse reactions as defined by all AEs either classified as at least possibly related to GC5107		13 months (12 months of treatment + 1 month of follow-up)
The number and proportion of GC5107 infusions for which the infusion rate was decreased due to AEs		12 months
The proportion of AEs considered by the investigator to be investigational product related		13 months (12 months of treatment + 1 month of follow-up)
Viral safety (freedom from transmission of blood-borne viral diseases): the human immunodeficiency virus (HIV) type 1 & 2, hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), and parvovirus B19		13 months (12 months of treatment + 1 month of follow-up)

Other Outcome Measures

Outcome Measure	Time Frame
The incidence of acute serious bacterial infections (aSBIs) defined at United States Food and Drug Administration (FDA) guidance criteria (bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis)	13 months (12 months of treatment + 1 month of follow-up)
The incidence of infections other than acute serious bacterial infections	13 months (12 months of treatment + 1 month of follow-up)
The number of days missed from work, school, kindergarten, day care or days unable to perform normal daily activities due to infections	13 months (12 months of treatment + 1 month of follow-up)
The number of days that the care provider of the pediatric subject had to miss work in order to care for the child due to infections	13 months (12 months of treatment + 1 month of follow-up)
The number of days of unscheduled physician visits due to infection	13 months (12 months of treatment + 1 month of follow-up)
The number of days of hospitalizations due to infection	13 months (12 months of treatment + 1 month of follow-up)
The number of days of intravenous (IV) therapeutic antibiotics	13 months (12 months of treatment + 1 month of follow-up)
The number of days of oral (PO) therapeutic antibiotics	13 months (12 months of treatment + 1 month of follow-up)
Time to resolution of infections	13 months (12 months of treatment + 1 month of follow-up)
The incidence of infections by trough IgG levels	13 months (12 months of treatment + 1 month of follow-up)
Episodes of fever (annual rate of fever episodes per subject)	13 months (12 months of treatment + 1 month of follow-up)

Collaborators and Investigators

• Sponsor: Green Cross Corporation
• Collaborators: Atlantic Research Group

Study record dates

Study Major Dates

• Study Start (Actual): December 21, 2020
• Primary Completion (Anticipated): May 1, 2023
• Study Completion (Anticipated): November 1, 2023

Study Registration Dates

• First Submitted: September 7, 2020
• First Submitted That Met QC Criteria: September 21, 2020
• First Posted (Actual): September 25, 2020

Study Record Updates

• Last Update Posted (Actual): May 17, 2023
• Last Update Submitted That Met QC Criteria: May 15, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: GC5107D

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No