Clinical trial NCT00520494: Efficacy and Safety of Vivaglobin® in Previously Untreated Patients With Primary Immunodeficiency
Official title: A Multicenter Study on the Efficacy and Safety of Vivaglobin® in Previously Untreated Patients (PUPs) With Primary Immunodeficiency (PID)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Common Variable Immunodeficiency
Agammaglobulinemia
Keywords: 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)
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2007-08-24
Enrollment: 18
Sex: ALL
Minimum age (years): 1
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Proportion of Patients Achieving Immunoglobulin G (IgG) Levels ≥ 5 g/L on Day 12
timeFrame: On Day 12
Secondary outcomes:
measure: Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 19
timeFrame: On Day 19
measure: Proportion of Patients Achieving IgG Levels ≥ 5 g/L on Day 26
timeFrame: On Day 26
measure: IgG Increase (Change From Baseline) on Day 12
timeFrame: Baseline to Day 12
measure: Overall Rate of Infections
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Total Serum IgG Trough Levels on Day 12
timeFrame: On Day 12
measure: Total Serum IgG Trough Levels at Week 25
timeFrame: At Week 25
measure: Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12
timeFrame: On Day 12
measure: Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25
timeFrame: At Week 25
measure: Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12
timeFrame: On Day 12
measure: Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25
timeFrame: At Week 25
measure: Use of Antibiotics for Infection Prophylaxis and Treatment
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Quality of Life as Measured by the Adapted Short Form-36 Health Survey (SF-36; Age ≥ 14 Years)
timeFrame: At study completion, approximately Week 25
measure: Quality of Life as Measured by the Child Health Questionnaire Parent Form-50 (CHQ-PF50; Age ≤ 13 Years)
timeFrame: At study completion, approximately Week 25
measure: Number of Patients With Adverse Events (AEs) by Severity and Relatedness
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Rate of AEs by Severity and Relatedness
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Number of Patients With Local Reactions by Severity and Relatedness
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Rate of Local Reactions by Severity and Relatedness
timeFrame: For the duration of the study, up to approximately 25 weeks
measure: Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters
timeFrame: At Weeks 12 and 25
measure: Number of Patients With Clinically Relevant Changes in Vital Signs
timeFrame: At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: Contact CSL Behring for facility details
state: Alberta
city: Montreal
country: Canada
facility: Contact CSL Behring for facility details
state: Quebec
city: Leipzig
country: Germany
facility: Contact CSL Behring for facility details
city: Brescia
country: Italy
facility: Contact CSL Behring for facility details
city: Roma
country: Italy
facility: Contact CSL Behring for facility details
city: Madrid
country: Spain
facility: Contact CSL Behring for facility details
Study officials:
affiliation: Klinik für Kinder-und Jugendmedizin am Städtischen Klinikum St. Georg, Leipzig, Germany
name: Michael Borte, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Borte M, Quinti I, Soresina A, Fernandez-Cruz E, Ritchie B, Schmidt DS, McCusker C. Efficacy and safety of subcutaneous vivaglobin(R) replacement therapy in previously untreated patients with primary immunodeficiency: a prospective, multicenter study. J Clin Immunol. 2011 Dec;31(6):952-61. doi: 10.1007/s10875-011-9588-5. Epub 2011 Sep 20.