Clinical Trial: Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)

Brief Summary: The objective of this study is to assess the efficacy and safety of Vivaglobin in previously untreated patients (PUPs) with primary immunodeficiency (PID) over a 25-week observation period. The purpose is to investigate whether PUPs will respond to subcutaneous immunoglobulin (SCIG) treatment with adequate trough levels without first receiving immunoglobulins by the intravenous route by demonstrating that 100 mg immunoglobulin G/kg body weight (IgG/kg bw) administered on 5 consecutive days (i.e. resulting in a total dose of 500 mg IgG/kg bw) results in an IgG increase to ≥ 5 g/L on Day 12 after initiation of SCIG therapy.

Detailed Summary:
Sponsor: CSL Behring

Current Primary Outcome: Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12 [ Time Frame: On Day 12 ]

Original Primary Outcome: The primary efficacy endpoint consists of the proportion of patients achieving IgG levels ≥500 mg/dL on Day 12

Current Secondary Outcome:

  • Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19 [ Time Frame: On Day 19 ]
  • Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26 [ Time Frame: On Day 26 ]
  • IgG Increase (Change From Baseline) on Day 12 [ Time Frame: Baseline to Day 12 ]
  • Overall Rate of Infections [ Time Frame: For the duration of the study, up to approximately 25 weeks ]

    Annualized rate of any infection. The annualized rate was based on the total number of infections and the total number of patient study days for all patients in the specified analysis population and adjusted to 365 days.

    Infections were classified as all AEs with the system organ class "infections and infestations".

  • Total Serum IgG Trough Levels on Day 12 [ Time Frame: On Day 12 ]
  • Total Serum IgG Trough Levels at Week 25 [ Time Frame: At Week 25 ]
  • Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12 [ Time Frame: On Day 12 ]
  • Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25 [ Time Frame: At Week 25 ]
  • Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12 [ Time Frame: On Day 12 ]
  • Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25 [ Time Frame: At Week 25 ]
  • Use of Antibiotics for Infection Prophylaxis and Treatment [ Time Frame: For the duration of the study, up to approximately 25 weeks ]
    Number of patients. Medications were classified as antibiotics according to the anatomic therapeutic chemical code.
  • Quality of Life as Measured by the Adapted Short Form-36 Health Survey (SF-36; Age ≥ 14 Years) [ Time Frame: At study completion, approximately Week 25 ]
    The SF-36 is a 36-item questionnaire that measures generic health concepts that are relevant across age, disease, and treatment groups. The questions are grouped into eight domains: physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health. Scores range from 0 to 100, with higher scores indicating a better health state.
  • Quality of Life as Measured by the Child Health Questionnaire Parent Form-50 (CHQ-PF50; Age ≤ 13 Years) [ Time Frame: At study completion, approximately Week 25 ]
    The CHQ-PF50 is a 50-item questionnaire that measures generic health concepts and is suitable for patients younger than 14 years of age. The questions are grouped into 15 domains: global health, physical functioning, role/social limitations - emotional/behavioral, role/social limitations - physical, bodily pain, behavior, global behavior, mental health, self esteem, general health perceptions, change in health, parental impact - emotional, parental impact - time, family activities, and family cohesion. Scores range from 0 to 100, with higher scores indicating a better health state.
  • Number of Patients With Adverse Events (AEs) by Severity and Relatedness [ Time Frame: For the duration of the study, up to approximately 25 weeks ]

    Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.

    Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.

  • Rate of AEs by Severity and Relatedness [ Time Frame: For the duration of the study, up to approximately 25 weeks ]

    The rate was the number of AEs over the number of infusions administered.

    Mild AE: Did not interfere with activities; Moderate AE: Interfered somewhat with routine activities; Severe AE: Impossible to perform routine activities.

    Not related: Explained by factors not involving the drug, no temporal relationship; Possibly related: Occurred within a reasonable time of administration, could also be explained by concurrent disease or other drugs; Probably related: Compelling temporal relationship, could not be explained concurrent disease/other drugs; Related AE: Compelling temporal relationship, known/suspected response to the drug confirmed by improvement on stopping.

  • Number of Patients With Local Reactions by S

    Original Secondary Outcome:

    • Overall rate of infections
    • Serum concentrations of specific IgGs
    • Total serum IgG trough levels
    • Quality of life as measured by an adapted SF-36 (age ≥14 years) or CHQ-PF50 (age ≤13 years)
    • Use of Antibiotics for Infection Prophylaxis and Treatment
    • Overall rate, severity and relatedness of any adverse event (AE) per infusion and patient
    • Changes in routine laboratory parameters (blood chemistry, hematology, urinalysis) as compared to baseline assessments


    Information By: CSL Behring

    Dates:
    Date Received: August 23, 2007
    Date Started: March 2007
    Date Completion:
    Last Updated: June 12, 2013
    Last Verified: June 2013