A Phase III, Open-label, Prospective, Multicenter Study to Assess Efficacy, Safety, and Pharmacokinetics of Kedrion Intravenous Human Normal Immunoglobulin (IVIg) 10% in Pediatric Patients Affected by Primary Immunodeficiency Disease (PID)
The purpose of this study is to assess efficacy, safety and pharmacokinetics of Kedrion Immunoglobulin 10% (KIg10) in pediatric patients with Primary Immunodeficiency Disease (PID).
• Written informed consent/assent obtained from the patient and his/her parent(s) or legally acceptable representative indicating that they understand the purpose of and procedures required for the study and are willing to participate in it.
• Confirmed clinical diagnosis of a PID as defined by 2017 International Union of Immunological Societies (IUIS) Phenotypic Classification for Primary Immunodeficiencies (Bousfiha A, 2018 - and subsequent revisions) and The European Society for Immunodeficiencies (ESID) Registry Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity (Seidel MG et al., 2019 - and subsequent revisions) and requiring treatment with IVIg. Documented agammaglobulinemia (defined as the total absence of one or more classes of antibodies) or hypogammaglobulinemia (defined as low levels of one or more classes \[i.e., at least 2 standard deviations under the mean level per age\]).
• (NOTE: IVIg treatment is generally requested in the absence of IgG independently from whether other antibodies are absent).
• Male or female, age from 2 up to \< 16 years, at the time of screening.
• Received 200 to 800 mg/kg of a commercially available IVIg therapy in the range of 21- or 28-day intervals (±3 or ±4 days, respectively) for at least 3 infusions prior to screening.
• (NOTE: Other IVIgs will be prohibited after ICF signature and until study end, week 51/52).
• At least 2 documented IgG trough levels while receiving an IVIg, of ≥ 6 g/L obtained at 2 infusions within 12 months (1 must be within 6 months) prior to ICF signature.
• Patient and his/her parent(s)/legal guardian(s) are willing to comply with all requirements of the protocol.
• Females of child-bearing potential with a negative pregnancy test (serum or urine) and who agree to employ adequate birth control measures during the study, such as:
∙ sexual abstinence, to be evaluated in relation to the preferred and usual lifestyle of the subject;
‣ male or female condom with or without spermicide;
‣ cap, diaphragm or sponge with spermicide;
‣ progestogen-only oral hormonal contraception, if already used in the past on medical prescription.
• Adequate birth control measures should be maintained throughout the study under parental control.
• Authorization to access personal health information.
• Patients previously participating in a clinical trial with another experimental IVIg may be enrolled if they have received stable commercially available IVIg therapy for at least 3 infusions (21 or 28 days) prior to screening.
⁃ Patients currently on treatment with any subcutaneous immunoglobulin (SCIG) can be enrolled if they are switched to stable commercially available IVIg therapy for at least 3 infusions (21 or 28 days) prior to screening.
⁃ Males or females with a body weight greater than or equal to 15 kg (≥ 15 kg).