Trial Outcomes & Findings for Study of Immune Globulin Intravenous (Human) GC5107 in Subjects With Primary Humoral Immunodeficiency (NCT NCT02783482)

Last Updated: 2022-12-09

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

49 participants

Primary outcome timeframe

One year

Results posted on

2022-12-09

Participant Flow

Participant milestones

Participant milestones
Measure	28-day Schedule Infusion every 28 days	21-day Schedule Infusion every 21 days
Overall Study STARTED	29	20
Overall Study COMPLETED	24	19
Overall Study NOT COMPLETED	5	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	28-day Infusion n=29 Participants Infusion every 28 days	21-day Infusion n=20 Participants Infusion every 21 days	Total n=49 Participants Total of all reporting groups
Age, Continuous	41.5 years STANDARD_DEVIATION 21.7 • n=5 Participants	30.7 years STANDARD_DEVIATION 23.2 • n=7 Participants	37.1 years STANDARD_DEVIATION 22.7 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	8 Participants n=7 Participants	21 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants	12 Participants n=7 Participants	28 Participants n=5 Participants
Race/Ethnicity, Customized White	28 Participants n=5 Participants	19 Participants n=7 Participants	47 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment Canada	11 Participants n=5 Participants	2 Participants n=7 Participants	13 Participants n=5 Participants
Region of Enrollment United States	18 Participants n=5 Participants	18 Participants n=7 Participants	36 Participants n=5 Participants
Duration since first IVIG infusion	8.8 years STANDARD_DEVIATION 8.3 • n=5 Participants	8.8 years STANDARD_DEVIATION 6.7 • n=7 Participants	8.8 years STANDARD_DEVIATION 7.6 • n=5 Participants
IVIG dose prior to enrollment	501.2 mg/kg STANDARD_DEVIATION 94.0 • n=5 Participants	592.7 mg/kg STANDARD_DEVIATION 135.9 • n=7 Participants	538.5 mg/kg STANDARD_DEVIATION 120.6 • n=5 Participants

PRIMARY outcome

Timeframe: One year

Population: Intent-to-Treat (ITT) Population: Defined as of all subjects who were enrolled into the study and received any amount of the IMP. This population was used for display of demographics, disposition, and for the primary safety and efficacy analyses

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Incidence of Acute Serious Bacterial Infections (SBI)	0.04 Number of events per patient per year Interval to 0.35 Pre-specified to only calculate the upper limit of the CI	0 Number of events per patient per year Here NA signifies that the upper limit of 99% CI was not estimable due to zero number of events	0.02 Number of events per patient per year Interval to 0.21 Pre-specified to only calculate the upper limit of the CI

PRIMARY outcome

Timeframe: Within 72 hours after an infusion of GC5107

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

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=667 Total No of infusions 28-Day schedule and 21-Day schedule
The Proportion of Infusions With Temporally Associated Adverse Events (TAAEs) That Occur Within 72 Hours Following an Infusion of Test Product	0.31 Proportion of infusion with TAAEs Interval to 0.4 Pre-specified to only calculate the upper limit of the CI, meeting FDA guidance	0.16 Proportion of infusion with TAAEs Interval to 0.22 Pre-specified to only calculate the upper limit of the CI, meeting FDA guidance	0.24 Proportion of infusion with TAAEs Interval to 0.31 Pre-specified to only calculate the upper limit of the CI, meeting FDA guidance

SECONDARY outcome

Timeframe: One year

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Incidence of Infections Other Than Acute Serious Bacterial Infections	2.4 Numberof infections per patient per year	3.6 Numberof infections per patient per year	2.9 Numberof infections per patient per year

SECONDARY outcome

Timeframe: One year

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.

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Number of Days Missed From Work/School/Kindergarten/Daycare, or Days Unable to Perform Normal Daily Activities Due to Infections	2.9 Days Standard Deviation 4.20	12.7 Days Standard Deviation 26.11	7.1 Days Standard Deviation 18.04

SECONDARY outcome

Timeframe: One year

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.

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Number of Days of Unscheduled Physician Visits Due to Infections	2.3 Days Standard Deviation 4.56	2.4 Days Standard Deviation 2.25	2.3 Days Standard Deviation 3.75

SECONDARY outcome

Timeframe: One year

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.

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Number of Days of Hospitalizations Due to Infections	0.1 Days Standard Deviation 0.57	0.1 Days Standard Deviation 0.46	0.1 Days Standard Deviation 0.53

SECONDARY outcome

Timeframe: One year

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.

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Number of Days of Intravenous (IV) Therapeutic Antibiotics	0.1 Days Standard Deviation 0.57	0.0 Days Standard Deviation 0.00	0.1 Days Standard Deviation 0.44

SECONDARY outcome

Timeframe: One year

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.

Outcome measures

Outcome measures
Measure	28-day Schedule n=29 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 28 days for 12 months of study infusions.	21-day Schedule n=20 Participants GC5107 was administered at a dose of 300-900 mg/kg (of body weight) every 21 days for 12 months of study infusions.	Total n=49 Participants 28-Day schedule and 21-Day schedule
The Number of Days of Oral Therapeutic Antibiotics	13.3 Days Standard Deviation 24.50	13.1 Days Standard Deviation 18.38	13.2 Days Standard Deviation 22.09

Adverse Events

GC5107 IVIG 10%

Serious events: 4 serious events

Other events: 49 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	GC5107 IVIG 10% n=49 participants at risk GC5107 IVIG 10% 28-day and 21-day infusion schedule
Infections and infestations Influenza	2.0% 1/49 • Number of events 1 • One year
Skin and subcutaneous tissue disorders Acute urticaria	2.0% 1/49 • Number of events 1 • One year
Cardiac disorders ST elevation myocardial infarction	2.0% 1/49 • Number of events 1 • One year
Infections and infestations Bacterial pneumonia	2.0% 1/49 • Number of events 1 • One year
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma of base of tongue	2.0% 1/49 • Number of events 1 • One year

Other adverse events

Other adverse events
Measure	GC5107 IVIG 10% n=49 participants at risk GC5107 IVIG 10% 28-day and 21-day infusion schedule
Nervous system disorders Headache	57.1% 28/49 • One year
Gastrointestinal disorders Nausea	40.8% 20/49 • One year
Respiratory, thoracic and mediastinal disorders Cough	30.6% 15/49 • One year
General disorders Fatigue	26.5% 13/49 • One year
General disorders Pyrexia	26.5% 13/49 • One year
Infections and infestations Sinusitis	26.5% 13/49 • One year
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	22.4% 11/49 • One year
Gastrointestinal disorders Diarrhoea	18.4% 9/49 • One year
Respiratory, thoracic and mediastinal disorders Nasal congestion	18.4% 9/49 • One year
Gastrointestinal disorders Abdominal pain upper	16.3% 8/49 • One year
Gastrointestinal disorders Vomiting	16.3% 8/49 • One year
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	14.3% 7/49 • One year
Musculoskeletal and connective tissue disorders Arthralgia	12.2% 6/49 • One year
Infections and infestations Bronchitis	12.2% 6/49 • One year
Infections and infestations Influenza	12.2% 6/49 • One year
Infections and infestations Nasopharyngitis	12.2% 6/49 • One year
Infections and infestations Upper respiratory tract infection	12.2% 6/49 • One year
Infections and infestations Acute sinusitis	10.2% 5/49 • One year
Respiratory, thoracic and mediastinal disorders Epistaxis	10.2% 5/49 • One year
Respiratory, thoracic and mediastinal disorders Sinus congestion	10.2% 5/49 • One year
Infections and infestations Urinary tract infection	10.2% 5/49 • One year
Skin and subcutaneous tissue disorders Urticaria	10.2% 5/49 • One year
Gastrointestinal disorders Abdominal pain	8.2% 4/49 • One year
Respiratory, thoracic and mediastinal disorders Asthma	8.2% 4/49 • One year
Skin and subcutaneous tissue disorders Rash	8.2% 4/49 • One year
Infections and infestations Viral infection	8.2% 4/49 • One year
General disorders Asthenia	6.1% 3/49 • One year
General disorders Chest discomfort	6.1% 3/49 • One year
Injury, poisoning and procedural complications Contusion	6.1% 3/49 • One year
Nervous system disorders Dizziness	6.1% 3/49 • One year
Eye disorders Ear pain	6.1% 3/49 • One year
Infections and infestations Gastroenteritis viral	6.1% 3/49 • One year
General disorders Infusion site extravasation	6.1% 3/49 • One year
Musculoskeletal and connective tissue disorders Musculoskeletal chest pain	6.1% 3/49 • One year
General disorders Non-cardiac chest pain	6.1% 3/49 • One year
Infections and infestations Otitis media	6.1% 3/49 • One year
General disorders Pain	6.1% 3/49 • One year
Musculoskeletal and connective tissue disorders Pain in extremity	6.1% 3/49 • One year
Respiratory, thoracic and mediastinal disorders Productive cough	6.1% 3/49 • One year
Nervous system disorders Sinus headache	6.1% 3/49 • One year
Investigations Urine analysis abnormal	6.1% 3/49 • One year
Infections and infestations Viral upper respiratory tract infection	6.1% 3/49 • One year

Additional Information

Clinical Project Manager

Green Cross Corporation

Phone: 82 31 260 9472

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place