Trial record 15 of 4490 for:    Immunodeficiency

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT00520494
First received: August 23, 2007
Last updated: June 12, 2013
Last verified: June 2013
  Purpose

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.


Condition Intervention Phase
Common Variable Immunodeficiency
Agammaglobulinemia
Drug: Vivaglobin
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12 [ Time Frame: On Day 12 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19 [ Time Frame: On Day 19 ] [ Designated as safety issue: No ]
  • Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26 [ Time Frame: On Day 26 ] [ Designated as safety issue: No ]
  • IgG Increase (Change From Baseline) on Day 12 [ Time Frame: Baseline to Day 12 ] [ Designated as safety issue: No ]
  • Overall Rate of Infections [ Time Frame: For the duration of the study, up to approximately 25 weeks ] [ Designated as safety issue: No ]

    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 ] [ Designated as safety issue: No ]
  • Total Serum IgG Trough Levels at Week 25 [ Time Frame: At Week 25 ] [ Designated as safety issue: No ]
  • Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12 [ Time Frame: On Day 12 ] [ Designated as safety issue: No ]
  • Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25 [ Time Frame: At Week 25 ] [ Designated as safety issue: No ]
  • Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12 [ Time Frame: On Day 12 ] [ Designated as safety issue: No ]
  • Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25 [ Time Frame: At Week 25 ] [ Designated as safety issue: No ]
  • Use of Antibiotics for Infection Prophylaxis and Treatment [ Time Frame: For the duration of the study, up to approximately 25 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]

    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 ] [ Designated as safety issue: Yes ]

    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 Severity and Relatedness [ Time Frame: For the duration of the study, up to approximately 25 weeks ] [ Designated as safety issue: Yes ]

    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.


  • Rate of Local Reactions by Severity and Relatedness [ Time Frame: For the duration of the study, up to approximately 25 weeks ] [ Designated as safety issue: Yes ]

    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.


  • Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters [ Time Frame: At Weeks 12 and 25 ] [ Designated as safety issue: Yes ]
    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).

  • 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) ] [ Designated as safety issue: Yes ]
    Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.


Enrollment: 18
Study Start Date: March 2007
Study Completion Date: October 2008
Primary Completion Date: October 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Vivaglobin
Vivaglobin: 16% (160 mg/mL) liquid formulation of human IgG for SC use. Loading dose: 100 mg/kg for 5 consecutive days; maintenance dose: 100 mg/kg 1 to 2 times a week for 24 weeks.
Drug: Vivaglobin
Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.

  Eligibility

Ages Eligible for Study:   1 Year to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00520494

Locations
Canada, Alberta
Contact CSL Behring for facility details
Edmonton, Alberta, Canada, T6G 2B7
Canada, Quebec
Contact CSL Behring for facility details
Montreal, Quebec, Canada, H3H 1P3
Germany
Contact CSL Behring for facility details
Leipzig, Germany, 04129
Italy
Contact CSL Behring for facility details
Brescia, Italy, 25123
Contact CSL Behring for facility details
Roma, Italy, 00186
Spain
Contact CSL Behring for facility details
Madrid, Spain, 28007
Sponsors and Collaborators
CSL Behring
Investigators
Principal Investigator: Michael Borte, MD Klinik für Kinder-und Jugendmedizin am Städtischen Klinikum St. Georg, Leipzig, Germany
  More Information

Additional Information:
Publications:
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00520494     History of Changes
Other Study ID Numbers: ZLB06_005CR, 2006-006522-25, 1461
Study First Received: August 23, 2007
Results First Received: March 18, 2013
Last Updated: June 12, 2013
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by CSL Behring:
Previously Untreated Patient (PUP)
Primary Immunodeficiency (PID)
CVID
XLA
Subcutaneous immunoglobulin (SCIG)
IgG trough level
Quality of life
Common variable immunodeficiency (CVID)
X-linked agammaglobulinemia (XLA)

Additional relevant MeSH terms:
Agammaglobulinemia
Common Variable Immunodeficiency
Immunologic Deficiency Syndromes
Blood Protein Disorders
Hematologic Diseases
Immune System Diseases
Lymphatic Diseases
Lymphoproliferative Disorders

ClinicalTrials.gov processed this record on October 23, 2014