Primary Immunodeficiency Diseases MedDRA version: 14.1 Level: HLT Classification code 10036700 Term: Primary immunodeficiency syndromes System Organ Class: 10021428 - Immune system disorders
Conditions
Interventions
Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: Human normal immunoglobulin
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: HUMAN NORMAL IMMUNOGLOBULIN
Other descripti
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS Scientific Committee 2009 and by diagnostic criteria according to Conley et al. prior to enrollment. 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. Subject has been receiving a consistent dose of IgG with a non-Baxter product (Hizentra SC or a non-Baxter product IV), or Subcuvia SC, administered in compliance with the respective product information for a period of at least 3 months prior to screening. The average minimum pre-study dose over that interval was an equivalent of 300 mg/kg BW every 4 weeks at a dosing frequency as follows: a) For IV treatment prior to the study: at mean intervals of 3 or 4 weeks (± 3 days) or b) For SC treatment prior to the study: at mean intervals of approximately 1 or 2 weeks (± 2 days) 4. Subject has a serum trough level of IgG >5 g/L at screening 5. Subject has not had a serious bacterial infection within the 3 months prior to screening. Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range ;Inclusion criteria: 1. Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS Scientific Committee 2009 and by diagnostic criteria according to Conley et al. prior to enrollment. 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. Subject has been receiving a consistent dose of IgG with a non-Baxter product (Hizentra SC or a non-Baxter product IV), or Subcuvia SC, administered in compliance with the respective product information for a period of at least 3 months prior to screening. The average minimum pre-study dose over that interval was an equivalent of 300 mg/kg BW every 4 weeks at a dosing frequency as follows: a) For IV treatment prior to the study: at mean intervals of 3 or 4 weeks (± 3 days) or b) For SC treatment prior to the study: at mean intervals of approximately 1 or 2 weeks (± 2 days) 4. Subject has a serum trough level of IgG >5 g/L at screening 5. Subject has not had a serious bacterial infection within the 3 months prior to screening. Are the trial subjects under 18? yes Number of subjects for this age range: 30 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has a known history of or is positive at screening for one or more of the following: 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 or myeloma, or macroglobulinemia (IgM) or paraproteinemia 15. Subject has severe dermatitis that would preclude adequate sites for safe product administration ;Exclusion criteria: 1. Subject has a known history of or is positive at screening for one or more of the following: 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 or myeloma, or macroglobulinemia (IgM) or paraproteinemia 15. Subject has severe dermatitis that would preclude adequate sites for safe product administration
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Tolerability of rHuPH20 facilitated SC treatment of IG, 10% treatment in subjects with PIDD who were on intravenous (IV) or subcutaneous (SC) treatment before the study. ;Secondary Objective: Safety, product administration, IgG trough levels, and further tolerability assessments.;Primary end point(s): Primary outcome measure: Percent of infusions tolerated.;Timepoint(s) of evaluation of this end point: 2-, 3- and 4-week intervals.;Main Objective: Tolerability of rHuPH20 facilitated SC treatment of IG, 10% treatment in subjects with PIDD who were on intravenous (IV) or subcutaneous (SC) treatment before the study. ;Secondary Objective: Safety, product administration, IgG trough levels, and further tolerability assessments.;Primary end point(s): Primary outcome measure: Percent of infusions tolerated.;Timepoint(s) of evaluation of this end point: 2-, 3- and 4-week intervals. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary outcome measures: TOLERABILITY AND SAFETY 1. Proportion of subjects who achieve a treatment interval of 2 weeks in Epoch 2 and of 3 or 4 weeks in Epoch 3 (Study Arms 2 or 3 only) 2. Proportion of subjects who maintain a treatment interval of 2 weeks in Epoch 2 and of 2, 3 or 4 weeks in Epoch 3 for a minimum of 8 weeks 3. Number and rate per subject and per infusion (excluding infections) of related systemic adverse events (AEs) 4. Number and rate per subject and per infusion (excluding infections) of related local AEs 5. Number and rate per subject and per infusion (excluding infections) of all AEs 6. Number of subjects who develop neutralizing antibodies to rHuPH20 EFFICACY Trough levels of IgG PRODUCT ADMINISTRATION 1. Infusions a) Duration of infusion and mean rate of infusion b) Maximum infusion rate achieved c) Percent of subjects who achieve maximum allowable infusion rate per protocol for any/all infusions d) Number of infusions (as training) prior to independent self-infusion (subject/caregiver) e) Number of subjects/caregivers approved by investigator for independent self-infusion 2. Proportion of subjects who prefer IG, 10% and rHuPH20 to previous IgG treatment (to be measured at the End-of-Study visit) FURTHER KEY VARIABLES - Efficacy - Quality of Life - Treatment Satisfaction Questionnaire - Treatment Preference Questionnaire - Product Administration;Timepoint(s) of evaluation of this end point: 2-, 3- and 4-week intervals.;Secondary end point(s): Secondary outcome measures: TOLERABILITY AND SAFETY 1. Proportion of subjects who achieve a treatment interval of 2 weeks in Epoch 2 and of 3 or 4 weeks in Epoch 3 (Study Arms 2 or 3 only) 2. Proportion of subjects who maintain a treatment interval of 2 weeks in Epoch 2 and of 2, 3 or 4 weeks in Epoch 3 for a minimum of 8 weeks 3. Number and rate per subject and per infusion (excluding infections) of related systemic adverse events (AEs) 4. Nu | — |
Countries
Belgium, Czech Republic, Germany, Italy, Netherlands, Sweden, Switzerland, United Kingdom
Contacts
Public ContactMedical Director;Medical Director ;
Baxter Healthcare Corporation ;Baxter Healthcare Corporation
Outcome results
None listed