The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.
NewGam is a new 10% human normal immunoglobulin (IVIG) solution developed by Octapharma for intravenous administration. It is supplied as a liquid formulation ready to use. The primary therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins; IVIG exhibits an immunomodulatory effect. Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). Theses diseases can be severe and can lead to substantial morbidity. Responses to antibacterial therapy are often poor. At present, most primary immune deficiencies are not curable, but IVIGs have been shown to decrease the total number of severe infections and the duration of hospitalization.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
51
The dose of NewGam, solvent/detergent treated human normal immunoglobulin 10%, remained the same throughout the study, as long as minimum trough levels of serum immunoglobulin G (IgG) was above 5 g/L. If serum IgG trough levels dropped to 5 g/L or less, the dose was to be adjusted at the investigator's discretion. NewGam was supplied as a solution for infusion.
Sudir Gupta, MD
Irvine, California, United States
Isaac Melamed, MD
Centennial, Colorado, United States
James Moy, MD
Chicago, Illinois, United States
William Smits, MD
Fort Wayne, Indiana, United States
Number of Serious Bacterial Infections Per Person-year of Treatment
The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.
Time frame: Baseline to end of the study (up to 12 months)
IgG Trough Level Concentration
Total IgG trough concentrations were measured in serum samples taken before each infusion.
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Haemophilus Influenzae
Trough level concentrations of antibodies against Haemophilus influenzae were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Measles
Trough level concentrations of antibodies against measles were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae
Trough level concentrations of antibodies against Streptococcus pneumoniae (serotypes types 6B, 14, 9V, 18C, 19F, 4, and 23F) were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Dr. Alan Knutsen
St Louis, Missouri, United States
Ai Lan Kobayashi, MD
Papillion, Nebraska, United States
Hans Ochs, MD
Seattle, Washington, United States
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Cytomegalovirus
Trough level concentrations of antibodies against cytomegalovirus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Tetanus
Trough level concentrations of antibodies against tetanus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Time frame: Baseline to end of the study (up to 12 months)
Trough Level Concentration of Antibodies Against Varicella-zoster Virus
Trough level concentrations of antibodies against varicella-zoster virus were measured in serum blood samples collected before the 1st infusion in all participants, before the 9th and 10th infusions in participants receiving NewGam every 3 weeks, before the 7th and 8th infusions in participants receiving NewGam every 4 weeks, and at the termination visit for all participants.
Time frame: Baseline to end of the study (up to 12 months)
Total Number of Infections
The number of infections included serious bacterial infections (bacterial pneumonia, bacteraemia/sepsis, osteomyelitis/septic arthritis, visceral abscess, bacterial meningitis) and other infections. For other infections, the Medical Dictionary for Regulatory Activities (MedDRA) preferred term was used to determine the type of infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified.
Time frame: Baseline to end of the study (up to 12 months)
Number of Non-serious Infections
The MedDRA preferred term was used to determine the type of non-serious infection. They were grouped into the following categories as determined by a medical expert: Ear infections, eye infections, infections of the gastrointestinal tract, infections of the genitourinary tract, upper respiratory tract infections, lower respiratory tract infections, infections of the skin, and infections not elsewhere classified. The total number of infections and the number in each category are reported.
Time frame: Baseline to end of the study (up to 12 months)
Time to Resolution of Serious and Other Infections
Since infections were reported as adverse events, the time to resolution of an infection was the time from the start date of the infection adverse event to the end date of the infection adverse event.
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants Treated With Antibiotics
The total percentage of participants treated with antibiotics, as well as, the percentage of participants treated with antibiotics therapeutically and prophylactically are reported.
Time frame: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Episodes Per Person-year of Treatment
The number of antibiotic treatment episodes per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment episodes / patient-years of NewGam treatment.
Time frame: Baseline to end of the study (up to 12 months)
Number of Antibiotic Treatment Days Per Person-year of Treatment
The number of antibiotic treatment days per person-year of treatment was calculated by the following formula: Total number of antibiotic treatment days / patient-years of NewGam treatment.
Time frame: Baseline to end of the study (up to 12 months)
Number of Participants Hospitalized Due to an Infection
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants With at Least 1 Episode of Fever
Time frame: Baseline to end of the study (up to 12 months)
Percentage of Participants That Missed School or Work Due to an Infection
Time frame: Baseline to end of the study (up to 12 months)
Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study
The Quality of Life (QoL) questionnaire Child Health Questionnaire-Parent Form (CHQ-PF50) was completed by a parent or guardian in study participants \< 14 years of age. The CHQ-PF50 consists of 50 items organized into 15 subscales.The 15 subscales could be combined into 2 summary scores, physical and psychosocial. The calculated summary scores were transformed to a range of 0-100, where a higher score indicates more positive functioning or better health status. A positive change score indicates improvement.
Time frame: Baseline to end of the study (up to 12 months)
Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study
The Quality of Life (QoL) questionnaire Short Form-36 Health Survey (SF-36-HS) was completed by participants ≥ 14 years of age. The SF-36-HS consists of 36 items organized into 8 subscales. The 8 subscales could be combined into 2 summary scores, physical and mental. The calculated summary scores were transformed to a range of 0-100, where a higher score indicates better health. A positive change score indicates improvement.
Time frame: Baseline to end of the study (up to 12 months)