Immunologic Deficiency Syndromes
Conditions
Brief summary
Octagam is a human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in more than 60 countries. This study will evaluate the efficacy, safety and the kinetics of Octagam 10% for replacement therapy in primary immunodeficiency diseases.
Detailed description
The primary objective of the study is to investigate the safety of Octagam 10% in replacement therapy in PID and to compare the pharmacokinetic profile of Octagam 10% with that of the previously used Octagam 5%. The secondary objective is to investigate the efficacy of Octagam 10% in replacement therapy in PID by monitoring the rate of occurence of serious bacterial infections, the rate of other infections, the trough (pre-next-dose) levels of total serum IgG and IgG subclasses (IgG1, IgG2, IgG3 and IgG4), the trough (pre-next-dose) levels of selected antigen specific antibodies, the use of antibiotics, the rate of absence from school/ work, and the number of days in hospital.
Interventions
DRUGOctagam 10%
300-600 mg/kg every 21 (+/- 3 days) to 28 days (+/- 3 days)
Sponsors
Octapharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of primary immunodeficiency (acc. WHO) * Previous treatment with commercial Octagam 5% every 3-4 weeks for at least 6 infusions intervals * Documented IgG trough levels of the two previous infusions before enrollment with a value of at least 5.5 g/L for both
Exclusion criteria
* Acute infection requiring intravenous antibiotic treatment within two weeks before screening * Exposure to blood or any blood product or derivative other than commercially available Octagam 5%, within the past 3 months * History of hypersensitivity to blood or plasma derived products * Requirement of any routine premedication for IGIV treatment * History of congenital impairment of pulmonary function * Severe liver function impairment * Severe renal function impairment or predisposition for acute renal failure * History of autoimmune haemolytic anaemia * History of diabetes mellitus * Congestive heart failure NYHA III or IV * Non-controlled arterial hypertension * History of DVT or thrombotic complications with IGIV treatment * Known infection with HIV, HCV or HBV * Treatment with steroids, immunosuppressive or immunomodulatory drugs * Planned vaccination during study period * Pregnant or nursing woman
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse Events | During infusion or within 72 hours after end of infusion | Occurrence of Adverse Events |
| Pharmacokinetics of Serum Total IgG and IgG Subclasses (IgG1, IgG2, IgG3 and IgG4) and Pharmacokinetics of Specific Antibodies Against Defined Infectious Agents Comparing Octagam 5% Treatment With Octagam 10% Treatment | after 6 months of treatment | Pharmacokinetics of serum total IgG and IgG subclasses (IgG1, IgG2, IgG3 and IgG4) and pharmacokinetics of specific antibodies against defined infectious agents comparing Octagam 5% treatment with Octagam 10% treatment |
Secondary
| Measure | Time frame |
|---|---|
| Assessment of Viral Safety | Every three months |
| Pre-next-dose Levels of Serum Total IgG | before each treatment |
| Vital Signs | during each treatment |
| Therapeutic Efficacy (Number of Infections, Number of Missed Days at School/ Work, Number of Hospitalisation Days, and Use of Antibiotics) | 12 months |
| Pre-next-dose Levels of IgG Subclasses (IgG1, IgG2, IgG3, IgG4) and Pre-next-dose Levels of Specific Antibodies Against Defined Infectious Agents | before treatement 10 and 13 (of 13 or 17 treatments) and at the end |
| Laboratory Parameters (Hematology, Clinical Chemistry, Direct Coombs Test and Urin Analysis) | at each treatment date (every three to four weeks) |
Countries
Austria
Participant flow
Recruitment details
Patients previously treated with commercial Octagam 5% were to be enrolled in the study on the basis of the in- and exclusion criteria defined in the study protocol. Patients were to be enrolled only after written informed consent by the patient had been obtained.
Participants by arm
| Arm | Count |
|---|---|
| Octagam 10% Octagam 10% : 300-600 mg/kg every 21 (+/- 3 days) to 28 days (+/- 3 days) | 5 |
| Total | 5 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 2 |
| Overall Study | Sponsors Decision | 1 |
Baseline characteristics
| Characteristic | Octagam 10% |
|---|---|
| Age, Categorical <=18 years | 2 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 5 / 5 |
| serious Total, serious adverse events | 1 / 5 |
Outcome results
Primary
Adverse Events
Occurrence of Adverse Events
Time frame: During infusion or within 72 hours after end of infusion
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Octagam 10% | Adverse Events | 4 participants |
Primary
Pharmacokinetics of Serum Total IgG and IgG Subclasses (IgG1, IgG2, IgG3 and IgG4) and Pharmacokinetics of Specific Antibodies Against Defined Infectious Agents Comparing Octagam 5% Treatment With Octagam 10% Treatment
Pharmacokinetics of serum total IgG and IgG subclasses (IgG1, IgG2, IgG3 and IgG4) and pharmacokinetics of specific antibodies against defined infectious agents comparing Octagam 5% treatment with Octagam 10% treatment
Time frame: after 6 months of treatment
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Assessment of Viral Safety
Time frame: Every three months
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Laboratory Parameters (Hematology, Clinical Chemistry, Direct Coombs Test and Urin Analysis)
Time frame: at each treatment date (every three to four weeks)
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Pre-next-dose Levels of IgG Subclasses (IgG1, IgG2, IgG3, IgG4) and Pre-next-dose Levels of Specific Antibodies Against Defined Infectious Agents
Time frame: before treatement 10 and 13 (of 13 or 17 treatments) and at the end
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Pre-next-dose Levels of Serum Total IgG
Time frame: before each treatment
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Therapeutic Efficacy (Number of Infections, Number of Missed Days at School/ Work, Number of Hospitalisation Days, and Use of Antibiotics)
Time frame: 12 months
Population: Due to premature termination of the study, data were unavailable and no analysis was done.
Secondary
Vital Signs
Time frame: during each treatment
Population: Due to premature termination of the study, data were unavailable and no analysis was done.