Skip to main content
OpenTrials
Completed

NCT Number: NCT00520494

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Common Variable Immunodeficiency Agammaglobulinemia Blood Protein Disorders Bruton type agammaglobulinemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Immunologic Deficiency Syndromes Lymphatic Diseases Lymphoproliferative Disorders Primary Immunodeficiency Diseases

Age range

1 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Contact CSL Behring for facility details, Edmonton, Alberta, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key Inclusion Criteria:

  • Written informed consent, age-adapted
  • Male or female aged 1 to 70 years
  • Diagnosis of primary humoral immunodeficiency
  • No prior immunoglobulin substitution therapy
  • IgG level of <5 g/L at screening
  • Women of childbearing potential must use medically approved contraception and must have a negative urine pregnancy test at screening

Key Exclusion Criteria:

  • Evidence of serious infection between screening and first treatment
  • Bleeding disorders that require medical treatments
  • Any medical disorder causing secondary immune disorders, autoimmune neutropenia, or a clinically significant defect in cell mediated immunity
  • Any condition likely to interfere with evaluation of the study drug or satisfactory conduct of the trial

Treatment and study plan

Vivaglobin

Drug

Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.

Primary outcomes

  1. Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12

    Time frame: On Day 12

Secondary outcomes

  1. Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19

    Time frame: On Day 19

  2. Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26

    Time frame: On Day 26

  3. IgG Increase (Change From Baseline) on Day 12

    Time frame: Baseline to Day 12

  4. 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".

  5. Total Serum IgG Trough Levels on Day 12

    Time frame: On Day 12

  6. Total Serum IgG Trough Levels at Week 25

    Time frame: At Week 25

  7. Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12

    Time frame: On Day 12

  8. Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25

    Time frame: At Week 25

  9. Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12

    Time frame: On Day 12

  10. Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25

    Time frame: At Week 25

  11. 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.

  12. 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.

  13. 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.

  14. 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.

  15. 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.

  16. Number of Patients With Local Reactions by Severity and Relatedness

    Time frame: For the duration of the study, up to approximately 25 weeks

    Local reactions included: infusion site erythema, infusion site pain, infusion site pruritus, infusion site rash, infusion site reaction, infusion site swelling, injection site bruising, injection site erythema, injection site irritation, injection site pruritus, injection site swelling, edema peripheral, tenderness, erythema, pruritus, and skin swelling.

    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.

  17. Rate of Local Reactions by Severity and Relatedness

    Time frame: For the duration of the study, up to approximately 25 weeks

    The rate was the number of local reactions over the number of infusions administered.

    Local reactions included:

    • infusion site: erythema, pain, pruritus, rash, reaction, swelling;
    • injection site: bruising, erythema, irritation, pruritus, swelling;
    • edema peripheral;
    • tenderness;
    • erythema;
    • pruritus; and
    • skin swelling.

    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.

  18. Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters

    Time frame: At Weeks 12 and 25

    Laboratory parameters included hematology, serum chemistry, and urinalysis parameters, and were assessed at screening, Week 12 (hematology and serum chemistry) and at the completion visit (approximately Week 25).

  19. Number of Patients With Clinically Relevant Changes in Vital Signs

    Time frame: At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)

    Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2007
Primary completion
2008
Study completion
2008
First posted
Aug 24, 2007
Registry last updated
Jun 20, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.