Primary Immunodeficiency Diseases MedDRA version: 16.1 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: KIOVIG
Product Name: KIOVIG
Product Code: IGIV, 10%
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
CAS Number: 0
Other descriptive name: HUMAN
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects who meet ALL of the following criteria are eligible for this study: 1. Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS Scientific Committee 2009 and by diagnostic criteria according to Conley et al. (1999). The diagnosis must be confirmed by the Medical Director prior to first treatment with IP in the study. 2. Subject is 2 years or older at the time of screening. 3. Written informed consent is obtained from either the subject or the subject’s legally authorized representative prior to any study-related procedures and study product administration. 4. Subject has been receiving a consistent monthly equivalent dose of IgG over a period of at least 3 months prior to first treatment with IP at an average minimum dose over that interval equivalent to 300 mg/kg bodyweight (BW)/4 weeks and a maximum dose equivalent to 1.0 gram/kg BW/4 weeks. Examples of pre-study dosing frequency: a) IV at mean intervals of approximately 3 or 4 weeks or b) SC at mean intervals of approximately 1 or 2 weeks c) SC alternative treatment schedule (e.g. 2x/week) 5. Subject has a serum trough level of IgG >5 g/L at screening 6. Subject has not had a serious bacterial infection within the 3 months prior to screening. 7. Subject is willing and able to comply with the requirements of the protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 22 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: Subjects who meet ANY of the following criteria are not eligible for this study: 1. Subject has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2. 2. Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent): a) Persistent alanine aminotransferase (ALT) and aspartate amino transferase (AST) >2.5 times the upper limit of normal for the testing laboratory b) Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] =500/mm3) 3. Subject has creatinine clearance (CLcr) value that is 9 g/dL and subjects or myeloma, or macroglobulinemia (IgM) or paraproteinemia. 14. Women of childbearing potential meeting any one of the following criteria a) subject presents with a positive pregnancy test b) subject is breast feeding c) subject intends to begin nursing during the course of the study d) subject does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom [for male partner] with spermicidal jelly or foam, or birth control pills/patches) throughout the course of the study. 15. Subject has participated in another clinical study and has been exposed to an investigational product (IP) or device within 30 days prior to study enrollment (exception: treatment with immunoglobulin pre-study). 16. Subject is scheduled to participate in another (non-Baxter) non-observational (interventional) clinical study involving an IP or device during the course of the study. 17. Severe dermatitis that would
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to evaluate the efficacy of Immune Globulin Subcutaneous (Human) (IGSC), 20% in subjects with PID.;Timepoint(s) of evaluation of this end point: Infections will be reported as adverse events throughout the study and the number and types of infections will be determined.;Secondary Objective: The secondary objectives of this study, in addition to further efficacy assessments, are to evaluate the safety, tolerability, and pharmacokinetic (PK) characteristics of Immune Globulin Subcutaneous (Human) (IGSC), 20% in subjects with PID. Exploratory Objective(s) Exploratory objectives are to assess dose adjustments, quality of life aspects, treatment satisfaction and treatment preference.;Primary end point(s): The primary endpoint is the acute serious bacterial infection rate defined as the mean number of acute serious bacterial infections per subject per year in the intent-to-treat population. Acute serious bacterial infections will include bacteremia / sepsis, bacterial meningitis, osteomyelitis / septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Serious Acute Bacterial Infections of the US Food and Drug Administration, Center for Biologics Evaluation and Research; Guidance for Industry - Safety, Efficacy, and Pharmacokinetic Studies to Support Marketing of Immune Globulin Intravenous (Human) as Replacement Therapy for Primary Humoral Immunodeficiency, June 2008. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): EFFICACY 1. Pharmacokinetics a. Trough levels i. Immunoglobulin G (IgG) trough levels during approximately 3 months treatment with SUBCUVIA (subcutaneously) or KIOVIG (intravenously) (Study Epoch 1) and IgG trough levels after SC administration of IGSC, 20% (Study Epoch 2). ii. Trough levels of specific antibodies to clinically relevant pathogens (Clostridium tetani toxoid, Haemophilus influenzae and Hepatitis B Virus) during approximately 3 months treatment with SUBCUVIA (subcutaneously) or KIOVIG (intravenously) (Study Epoch 1) and trough levels of these specific antibodies after SC administration of IGSC, 20% (Study Epoch 2) b. Other PK parameters The following PK parameters will also be assessed for IgG: area under the curve (AUC), clearance (CL) for IV and apparent clearance (Cl/F) for SC administration, maximum concentration (Cmax), minimum concentration (Cmin), and time to maximum concentration (Tmax). 2. Infections a. The annual rate of all infections per subject b. The annual rate of sinus infections per subject c. The annual rate of fever episodes per subject d. Days not able to attend school/work or to perform normal daily activities due to illness/infection e. Days on antibiotics f. Number of hospitalizations and length of stay (in days) g. Acute (urgent or unscheduled) physician visits due to illness/infection Safety: 1. Related AEs a. Number of AEs (including and excluding infections) determined by the investigator to be related to the study drug that occur at any time during the study ("related") divided by the number of subjects b. Number of AEs (including and excluding infections) determined by the investigator to be related to the study drug that occur at any time during the study ("related") divided by the number of infusions 2. All AEs a. Annual rate of serious adverse events (SAEs), related and not related b. Rates of AEs (including and excluding infections) defined as number of AEs categorized by | — |
Countries
Austria, Belgium, Germany, Hungary, Netherlands, Sweden, United Kingdom
Contacts
Public ContactSusanne Schmiedl
Baxter Innovations GmbH
Outcome results
None listed