Clinical trial NCT04565015: Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency
Official title: 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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Primary Immune Deficiency
Lead sponsor: GC Biopharma Corp
Sponsor class: INDUSTRY
Collaborators: name: Atlantic Research Group
First posted: 2020-09-25
Enrollment: 24
Sex: ALL
Minimum age (years): 2
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

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.

Eligibility criteria: 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

Primary outcomes:
measure: The Pharmacokinetic (PK) Plasma concentration-time curve of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Half-life of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Area under the curve of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Volume of distribution of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Maximum concentration of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Minimum concentration of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Time of maximum concentration of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Clearance of total IgG
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: Trough serum total IgG levels before each infusion of GC5107 in all subjects and the interval between infusions
timeFrame: 12 months
measure: 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
timeFrame: 12 months
Secondary outcomes:
measure: The Pharmacokinetic (PK) Maximum concentration of IgG subclasses
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Minimum concentration of IgG subclasses
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: The Pharmacokinetic (PK) Half-life of IgG subclasses
timeFrame: before and after 5th infusion (12 or 16 weeks)
measure: 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)
timeFrame: 12 months
measure: 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)
timeFrame: 12 months
measure: The overall incidence of all AEs that occur during or within 1 hour, 24 hours, and 72 hours following an infusion of investigational product
timeFrame: 12 months
measure: The frequency of all AEs that occur during the study regardless of the investigator's assessment of their relationship to investigational product
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The frequency of suspected adverse reactions as defined by all AEs either classified as at least possibly related to GC5107
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number and proportion of GC5107 infusions for which the infusion rate was decreased due to AEs
timeFrame: 12 months
measure: The proportion of AEs considered by the investigator to be investigational product related
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: 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
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
Other outcomes:
measure: 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)
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The incidence of infections other than acute serious bacterial infections
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number of days missed from work, school, kindergarten, day care or days unable to perform normal daily activities due to infections
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: 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
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number of days of unscheduled physician visits due to infection
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number of days of hospitalizations due to infection
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number of days of intravenous (IV) therapeutic antibiotics
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The number of days of oral (PO) therapeutic antibiotics
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: Time to resolution of infections
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: The incidence of infections by trough IgG levels
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
measure: Episodes of fever (annual rate of fever episodes per subject)
timeFrame: 13 months (12 months of treatment + 1 month of follow-up)
Locations:
city: Oklahoma City
country: United States
facility: Oklahoma Institute of Allergy & Asthma Clinical Research, LLC
state: Oklahoma
city: Dallas
country: United States
facility: Allergy Partners of North Texas Research
state: Texas
city: Fairfax
country: United States
facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc.
state: Virginia
city: Richmond
country: United States
facility: Children's Hospital of Richmond at VCU
state: Virginia
city: Milwaukee
country: United States
facility: University of Wisconsin
state: Wisconsin
Study officials:
References: