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)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 56
- 试验地点
- 12
- 主要终点
- Trough Plasma Concentration of Total IgG at Steady State
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
Single group receiving BP-SCIG20%
Patients receive BP-SCIG 20% (human normal immunoglobulin, 200 mg/mL, IgG ≥95%) administered subcutaneously. Dosage: 0.4-0.8 g/kg per month, divided into weekly infusions. Duration: Long-term treatment as per study protoco
干预措施: BP-SCIG 20% (Drug)
结局指标
主要结局
Trough Plasma Concentration of Total IgG at Steady State
时间窗: 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
时间窗: 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.
次要结局
- Fraction of patients reaching IgG ≥5 g/L(From baseline through 12 months of treatment)
- Incidence of non-serious infections(From baseline through 12 months of treatment)
- Duration of non-serious infections(From baseline through 12 months of treatment)
- Antibiotic use(From baseline through 12 months of treatment)
- Hospital admissions due to infection(From baseline through 12 months of treatment)