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

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

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

Study Overview

• Status: Completed
• Conditions: Immunologic Deficiency Syndromes
• Intervention / Treatment: Biological: GC5107

Detailed Description

This was a prospective, open-label, single-arm, historically controlled, multicenter phase 3 study measuring the safety, efficacy and pharmacokinetics and tolerability of GC5107 in subjects with Primary Humoral Immunodeficiency disease (PHID).

Subjects received intravenous infusions of the investigational product at the same dose and interval as used for their previous Immunoglobulin intravenous (IVIG) maintenance therapy. GC5107 was administered every 21 or 28 days for a period of 12 months.

• Study Type: Interventional
• Enrollment (Actual): 49
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2V2
      • University of Alberta Hospital
  • Ontario
    • Hamilton, Ontario, Canada, L8S4K1
      • Hamilton Health Sciences Corporation
    • Kingston, Ontario, Canada, K7L 2V7
      • Queen's University - Kingston General Hospital (KGH)
    • Ottawa, Ontario, Canada, K1H 8L6
      • The Ottawa Hospital
    • Toronto, Ontario, Canada, M4V 1R2
      • Gordon Sussman Clinical Research
    • Toronto, Ontario, Canada, M5B 1W8
      • Saint Michael's Hospital
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • CHU Ste-Justine - University of Montreal
    • Montreal, Quebec, Canada, H4A 3J1
      • McGill University Health Centre (MUHC) - The Montreal Children's Hospital
    • Montréal, Quebec, Canada, H2W 1T8
      • Hotel Dieu de Montreal
    • Québec City, Quebec, Canada, G1V 4W2
      • Clinique Specialisee en Allergie de la Capitale
• United States
  • Colorado
    • Centennial, Colorado, United States, 80112
      • IMMUNO International Research Centers
  • Florida
    • North Palm Beach, Florida, United States, 33408
      • Allergy Associates of Palm Beaches PA
  • Minnesota
    • Plymouth, Minnesota, United States, 55446
      • Midwest Immunology Clinic and Infusion Center
  • Ohio
    • Columbus, Ohio, United States, 43235
      • Optimed Infusions LLC
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73131
      • Oklahoma Institute of Allergy Ashma and Immunology
  • Texas
    • Dallas, Texas, United States, 75230
      • Allergy Partners of North Texas Research
    • Dallas, Texas, United States, 75231
      • Allergy and Asthma Specialists
    • Frisco, Texas, United States, 75034
      • Pediatric Pulmonary Associates of North Texas
    • Irving, Texas, United States, 75063
      • Allergy Asthma and Immunology Clinic PA
  • Virginia
    • Fairfax, Virginia, United States, 22030
      • Lysosomal Rare Disorder Research and Treatment Center, Inc.

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 70 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subject with a confirmed clinical diagnosis of a Primary Humoral Immunodeficiency Disease as defined by IUIS (International Union of Immunological Societies) and require treatment with IGIV. Documented agammaglobulinemia or hypogammaglobulinemia
• Male or Female, ages 2 to 70 years
• The subject has received 300-900 mg/kg of a licensed IGIV therapy at 21 or 28 day intervals for at least 3 months prior to this study
• At least 2 documented IgG trough levels of ≥ 500 mg/dL are obtained at two infusion cycles (21 or 28 days) within 12 months prior to study enrollment

Exclusion Criteria:

• Subject has secondary immunodeficiency
• Subject was newly diagnosed with PHID and has not yet been treated with immunoglobulin
• Subject has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency or isolated IgA deficiency with known anti-IgA antibodies
• History of severe reaction or hypersensitivity to IGIV or other injectable form of IgG
• Subject has a lifetime history of at least one thrombotic event including deep vein thrombosis, cerebrovascular accident, pulmonary embolism, transient ischemic attacks, or myocardial infarction
• Subject has received blood products other than human albumin or human immunoglobulin within 12 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; GC5107 Immune globulin intravenous (human) solution, 10% liquid	Biological: GC5107; GC5107 20g/200mL, intravenously, dose of 300 - 900 mg/kg (of body weight) every 21 or 28 days for 12 months, a follow-up (3 or 4 weeks); Other Names: IGIV; Immune globulin intravenous (human) solution, 10% liquid

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Incidence of Acute Serious Bacterial Infections (SBI)	The incidence of acute serious bacterial infections (aSBIs) meeting FDA guidance criteria, which includes bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscesses, and osteomyelitis/septic arthritis. Efficacy data is evaluated by comparing the frequency of acute serious bacterial infections per subject per year according to the FDA guideline of an upper one-sided 99% confidence limit < 1.0 per subject per year.	One year
The Proportion of Infusions With Temporally Associated Adverse Events (TAAEs) That Occur Within 72 Hours Following an Infusion of Test Product	The proportion of infusions with temporally associated adverse events occurring during or within 72 hours following infusion, whether or not they were thought to be related to GC5107	Within 72 hours after an infusion of GC5107

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The Incidence of Infections Other Than Acute Serious Bacterial Infections		One year
The Number of Days Missed From Work/School/Kindergarten/Daycare, or Days Unable to Perform Normal Daily Activities Due to Infections	Based on the total number of days missed from work/school/kindergarten/daycare or days unable to perform normal daily activities due to infections for each subject. Mean and SD are calculated based on weighting for the duration of data available for each subject, where duration is defined as (date of last visit - first infusion date + 1) for subjects who complete the study; and defined as (date of withdrawal - first infusion date + 1) for subjects who withdraw from the study.	One year
The Number of Days of Unscheduled Physician Visits Due to Infections	Based on the total number of days of unscheduled physician visits due to infections for each subject. Mean and SD are calculated based on weighting for the duration of data available for each subject, where duration is defined as (date of last visit - first infusion date + 1) for subjects who complete the study; and defined as (date of withdrawal - first infusion date + 1) for subjects who withdraw from the study.	One year
The Number of Days of Hospitalizations Due to Infections	Subject with no experience of specific event will be included in the analysis as zero incidence, zero day, or zero time duration. The mean and SD will be calculated weighting for the duration of data available for each subject.	One year
The Number of Days of Intravenous (IV) Therapeutic Antibiotics	Based on the total number of days of IV therapeutic antibiotics for each subject. Mean and SD are calculated based on weighting for the duration of data available for each subject, where duration is defined as (date of last visit -first infusion date + 1) for subjects who complete the study; and defined as (date of withdrawal - first infusion date + 1) for subjects who withdraw from the study.	One year
The Number of Days of Oral Therapeutic Antibiotics	Based on the total number of days of oral (PO) therapeutic antibiotics for each subject. Mean and SD are calculated based on weighting for the duration of data available for each subject, where duration is defined as (date of last visit - first infusion date + 1) for subjects who complete the study; and defined as (date of withdrawal - first infusion date + 1) for subjects who withdraw from the study.	One year

Collaborators and Investigators

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

Publications and helpful links

General Publications

• Perez EE, Hébert J, Ellis AK, Alpan O, Lumry WR, Shapiro R, Suez D, Mandujano JF, Wasserman RL. Efficacy, Safety and Tolerability of a New 10% Intravenous Immunoglobulin for the Treatment of Primary Immunodeficiencies. Front Immunol. 2021 Jul 8;12:707463. doi: 10.3389/fimmu.2021.707463. eCollection 2021.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2016
• Primary Completion (Actual): July 1, 2019
• Study Completion (Actual): July 1, 2019

Study Registration Dates

• First Submitted: April 24, 2016
• First Submitted That Met QC Criteria: May 25, 2016
• First Posted (Estimate): May 26, 2016

Study Record Updates

• Last Update Posted (Estimate): December 9, 2022
• Last Update Submitted That Met QC Criteria: November 11, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Immunologic Deficiency Syndromes; Physiological Effects of Drugs; Immunologic Factors; Immunoglobulins; Immunoglobulins, Intravenous; gamma-Globulins; Rho(D) Immune Globulin
• Other Study ID Numbers: GC5107B_P3

Plan for Individual participant data (IPD)

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