Study of BP-SCIG 20% in Patients With Primary Immunodeficiency (PID)

Официальное название

Open-label, Multicentre, Tolerability, Safety and Pharmacokinetics Study of the Medicinal Product BP-SCIG 20% (Human Normal Immunoglobulin for Subcutaneous Administration) Manufactured by BIOPHARMA PLASMA LLC, Followed by an Efficacy Assessment of Long-term Use in Patients With Primary Immunodeficiency (PID)

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат BP-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.

Первичные конечные точки

• Trough Plasma Concentration of Total IgG at Steady State [Срок оценки: From baseline through 12 months of treatment]
• Number of Serious Bacterial Infections per Subject per Year [Срок оценки: From baseline through 12 months of treatment]

Вторичные конечные точки (5)

• Fraction of patients reaching IgG ≥5 g/L [Срок оценки: From baseline through 12 months of treatment]
• Incidence of non-serious infections [Срок оценки: From baseline through 12 months of treatment]
• Duration of non-serious infections [Срок оценки: From baseline through 12 months of treatment]
• Antibiotic use [Срок оценки: From baseline through 12 months of treatment]
• Hospital admissions due to infection [Срок оценки: From baseline through 12 months of treatment]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Украина · 12 центров

• Municipal Non-Profit Enterprise "Cherkasy Regional Children's Hospital" of the Cherkasy Re — Cherkasy
• Regional Municipal Non-Profit Enterprise "Chernivtsi Regional Teaching Hospital" — Chernivtsi
• Municipal Non-Profit Enterprise "Ivano-Frankivsk Regional Teaching Hospital" of the Ivano- — Ivano-Frankivsk
• Municipal Non-Profit Enterprise "Ivano-Frankivsk Regional Children's Teaching Hospital" of — Ivano-Frankivsk
• Municipal Non-Profit Enterprise "Kyiv City Children's Teaching Hospital No. 1" — Kyiv
• LLC "Allergy and Cough Clinic" — Lutsk
• Municipal Enterprise "Volyn Regional Territorial Medical Association for the Protection of — Lutsk
• Municipal Non-Profit Enterprise of the Lviv Regional Council "Lviv Regional Teaching Diagn — Lviv
• … и ещё 4 центра

Идентификаторы

NCT: NCT07346859 · 2024-SCIg-BP

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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