Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
Octanorm (cutaquig®) is a 16.5% human normal immunoglobulin solution developed by Octapharma for subcutaneous administration (SCIG). It is supplied as a liquid formulation ready to use. One important therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections. These 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 SCIGs have been shown to decrease the total number of severe infections and the duration of hospitalization. This study evaluated the efficacy, safety and tolerability of octanorm in adult PID patients in an open-label, multi center, phase 3 study with an 8-week wash-in/wash-out period followed by a 6-month efficacy period
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
25
Octanorm
The State Research Center, Institute of Immunology of the Federal Medical-Biological Agency
Moscow, Russia
Federal Research Center of Pediatric Hematology, Oncology and Immunology of the Ministry of Health and Social Development of the Russian Federation
Moscow, Russia
State Medical University
Rostov, Russia
Pasteur Institute
Saint Petersburg, Russia
Number of Serious Bacterial Infections Per Person-Year on Treatment
Serious Bacterial Infections defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess
Time frame: Primary Treatment Period (24 Weeks)
Number of Patients With Other Infections
The number of patients with all infections of any kind or seriousness.
Time frame: Primary Treatment Period (24 Weeks)
Number of 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: Primary Treatment Period (24 Weeks)
Time to Resolution of 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: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)
Number of Participants Using Antibiotics From 0 to > 20 Days
Number of patients using antibiotics during the whole treatment period (36 weeks) grouped per number of days with antibiotic usage.
Time frame: Primary Treatment Period (24 Weeks)
Annual Rate of Antibiotic Use
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Institute of Immunology and Physiology of the Ural Branch of the Russian Academy of sciences
Yekaterinburg, Russia
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 Octanorm treatment
Time frame: Primary Treatment Period (24 Weeks)
Hospitalizations Due to Infection
Number of days spent in hospital due to infection
Time frame: Primary Treatment Period (24 Weeks)
Rate of Hospitalizations Due to Infection
Annual Rate of Hospitalizations due to Infection
Time frame: Primary Treatment Period (24 Weeks)
Episodes of Fever
Number of episodes of fever
Time frame: Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks)
Rate of Episodes of Fever
The number of episodes of fever per person-year of treatment was calculated by the following formula: Total number of episodes of fever / patient-years of Octanorm treatment
Time frame: Primary Treatment Period (24 Weeks)
Patients With Days Missed From Work/Study Due to Infections and Treatment
Total number of patients who missed days from work or study due to infections or treatment thereof.
Time frame: Primary Treatment Period (24 Weeks)
Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study
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 the end of study (up to 36 weeks)
Trough Levels of Serum Total IgG
Total IgG trough concentrations were measured in serum samples taken before each infusion given at the study site.
Time frame: At baseline and at last infusion (week 33)
Number of Participants Experiencing Treatment-Emergent AEs
TEAEs were classified as temporally associated if the onset was during the infusion or within 72 hours after the end of the infusion.
Time frame: Up to 36 weeks
Proportion of Infusions With at Least 1 Temporally Associated AE
The proportion of infusions with at least 1 temporally associated AE (TAAE) was calculated by dividing the total number of TAAE by the total number of infusions.
Time frame: Up to 36 weeks
Total Number of Adverse Events Regardless of Causality
An AE is any untoward medical occurrence in a study patient receiving an IMP and which does not necessarily have a causal relationship with this treatment.
Time frame: Up to 36 weeks
Number of Related Adverse Events
A related adverse event is an AE for which a causal relationship between the IMP and the AE cannot be ruled out.
Time frame: Up to 36 weeks
Number of Infusions With Infusion Site Reaction
Total number of infusions that triggered an infusion site reaction and number of infusions that triggered mild, moderate, severe or no infusion site reactions.
Time frame: Up to 36 weeks
Annual Rate of Infections
The annual rate of all infections of any kind of seriousness
Time frame: Up to 36 weeks