Primary Immunodeficiency Diseases
Conditions
Brief summary
All patients will receive the investigational medicinal product BP-SCIG 20%, solution for subcutaneous administration, manufactured by BIOPHARMA PLASMA LLC. A loading dose of IMP may be required (at the Investigator's decision): at least 0.2-0.5 g/kg (1.0-2.5 mL/kg) of body weight. This dose is distributed across several days, with a maximum daily dose of 0.1 to 0.15 g/kg. After achieving a steady-state (minimal) level of IgG, maintenance doses are administered at repeated intervals (approximately once a week) to achieve a cumulative monthly dose of 0.4-0.8 g/kg.
Detailed description
The clinical trial will consist of three periods, namely: screening period: lasting up to 2 months, and involving three screening visits; treatment period (from Cycle 1 visit \[the first 4 administrations of the IMP are to be performed in a hospital setting\] during the 52 weeks to the last visit of Cycle 13). follow-up period after completion of the last cycle of the treatment period, with a duration of 1 week.
Interventions
DRUGBP-SCIG 20%
Human normal immunoglobulin 200 mg/mL (including immunoglobulin G (IgG) no less than 95 %) administered subcutaneously. Distribution of immunoglobulin G subclasses in the product: IgG1: 65.6 %, IgG2: 22.1 %, IgG3:10.8 %, IgG4:1.5 % The maximum content of immunoglobulin A is 100 µg/mL.
Sponsors
Biopharma Plasma LLC
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This study does not involve the distribution of patients into groups. Of patients in all age categories who meet inclusion criteria and passed the screening, and who do not meet any of the non-inclusion (exclusion) criteria, one group is formed, which will receive the investigational product.
Eligibility
Sex/Gender
ALL
Age
0 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* a signed informed written consent of the patient/patient's parents or legally authorised representative to take part in the study; * children from 0 to 18 years of age, of both sexes; or adult patients: males and females older than 18 years; * a diagnosis of primary immunodeficiency with impaired antibody production document-supported according to the ESID criteria, requiring replacement therapy with immunoglobulins; * patients with a body weight of no less than 9.1 kg; * patients who received replacement therapy with intravenous immunoglobulin (IVIG) products at intervals from 21 to 28 days at the dose of 0.4-0.8 g/kg, for at least 4 months prior to the start of the study and have total serum IgG levels ≥5 g/L\*, which is confirmed by the results of no less than 2 measurements performed immediately before the scheduled IVIG administration after signing the ICF, or patients who received replacement therapy with subcutaneous immunoglobulin (SCIG) products at the dose of 0.4-0.8 g/kg per month, for at least 4 months prior to the start of the study, and have total serum IgG levels ≥5 g/L\*, based on the results of no less than 2 measurements performed immediately before the scheduled SCIG administration after signing the ICF; or patients with a newly established diagnosis of PID who have not yet received replacement therapy with IgG products (treatment-naïve patients) and have a serum IgG level ≤ 4 g/L; * absence of episodes of serious bacterial infections (bacteraemia or sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, visceral abscess) while taking the previous medicinal product and absence of episodes of hospitalisation for at least 3 months prior to inclusion in the study and during the screening period (prior to the first administration of the IMP BP-SCIG 20%); * a negative pregnancy test (in female patients with childbearing potential); readiness to use reliable methods of contraception throughout the study period; * patient's ability, in the Investigator's judgement, to comply with all the requirements of the study protocol.
Exclusion criteria
* at the request of the patient/patient's parents/patient's legally authorised representative at any time and for any reason; * patient developing serious and/or unexpected AEs/ARs during the study, which require discontinuation of the product; * blood transfusions or transfusions of blood components and products, with the exception of the IMP; * the need to use medicinal products disallowed as part of this study; * systematic lack of patient compliance with the treatment regimen prescribed by the Investigator; * systematic lack of patient compliance with the procedures specified in this protocol; * exclusion based on screening results, including emergence of episodes of serious bacterial infections while receiving a previous IV immunoglobulin product and episodes of hospitalisation during the screening period (before the first administration of the IMP BP-SCIG 20%). * Receiving the IMP BP-SCIG 20% for less than 4 months; * Presence of episodes of serious bacterial infections (bacteraemia or sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, visceral abscess) at the time of investigation of PK parameters.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Trough Plasma Concentration of Total IgG at Steady State | From baseline through 12 months of treatment | Mean monthly trough plasma concentration of total IgG prior to the next BP-SCIG 20% infusion, measured after 4 months of treatment and assessed over 6 months, compared with previous therapy (BIOVEN). |
| Number of Serious Bacterial Infections per Subject per Year | From baseline through 12 months of treatment | Occurrence of less than 1 serious bacterial infection per patient per year during 52 weeks of BP-SCIG 20% treatment. Serious bacterial infection includes sepsis, bacterial pneumonia, bacterial visceral infections, bacterial meningitis, osteomyelitis/septic arthritis. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of non-serious infections | From baseline through 12 months of treatment | Total duration of non-serious infections per patient per year. |
| Fraction of patients reaching IgG ≥5 g/L | From baseline through 12 months of treatment | Percentage of patients achieving serum IgG ≥5 g/L prior to scheduled infusion. |
| Hospital admissions due to infection | From baseline through 12 months of treatment | Number and duration of hospital admissions due to infection per patient per year. |
| Antibiotic use | From baseline through 12 months of treatment | Fraction of patients receiving antibiotics and mean number of days of antibiotic therapy per year. |
| Incidence of non-serious infections | From baseline through 12 months of treatment | Number of non-serious infectious disease episodes per patient per year |
Countries
Ukraine
Outcome results
None listed