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

NCT04565015 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: GC5107D
• Study Type: interventional
• Target: 24
• Locations: 3 countries
• Sites: 8

Brief Summary

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).

Conditions

Primary Immune Deficiency

Outcome Measures

Primary Outcomes (10)

• 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 Outcomes (11)

• 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

Other Outcomes (11)

• 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)

Study Arms (1)

GC5107

EXPERIMENTAL

Immune Globulin Intravenous (Human), 10% Liquid

Biological: GC5107

Interventions

GC5107 BIOLOGICAL

Intravenously infused at a dose of 300 - 900 mg per kg (of body weight) every 21 or 28 days for 12 months

Also known as: GCC 10% IGIV, Immune Globulin Intravenous (Human), 10% Liquid

GC5107

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• GC Biopharma Corp: lead
• Atlantic Research Group: collaborator

Study Sites (8)

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Immunoe Health & Research Centers

Centennial, Colorado, 80112, United States

Location

Allergy Partners of North Texas Research

Dallas, Texas, 75230, United States

Location

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, 22030, United States

Location

Children's Hospital of Richmond at VCU

Richmond, Virginia, 23219, United States

Location

University Clinical Center Sarajevo

Sarajevo, Sarajevo, 71000, Bosnia and Herzegovina

Location

University clinical center Tuzla

Tuzla, Tuzla, 75000, Bosnia and Herzegovina

Location

Institute for Child and Youth Health Care of Vojvodina

Novi Sad, Novi Sad, 21000, Serbia

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Interventions

gamma-Globulins; Fluid Therapy

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Drug Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2020
• First Posted: September 25, 2020
• Study Start: December 21, 2020
• Primary Completion: May 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: July 25, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (5); BO (2); SE (1)