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

Common Variable Immunodeficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00520494
• Other study ID #: ZLB06_005CR
• Secondary ID: 2006-006522-2514
• Status: Completed
• Phase: Phase 4
• First received: August 23, 2007
• Last updated: June 12, 2013
• Start date: March 2007
• Est. completion date: October 2008

Study information

• Verified date: June 2013
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: October 2008
• Est. primary completion date: October 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 70 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Agammaglobulinemia
• Common Variable Immunodeficiency
• Immunologic Deficiency Syndromes

Intervention

Drug:
• Vivaglobin
Human normal immunoglobulin G (IgG) for subcutaneous (SC) use.

Locations

Country	Name	City	State
Canada	Contact CSL Behring for facility details	Edmonton	Alberta
Canada	Contact CSL Behring for facility details	Montreal	Quebec
Germany	Contact CSL Behring for facility details	Leipzig
Italy	Contact CSL Behring for facility details	Brescia
Italy	Contact CSL Behring for facility details	Roma
Spain	Contact CSL Behring for facility details	Madrid

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Canada,  Germany,  Italy,  Spain, 

References & Publications (1)

Borte M, Quinti I, Soresina A, Fernández-Cruz E, Ritchie B, Schmidt DS, McCusker C. Efficacy and safety of subcutaneous vivaglobin® replacement therapy in previously untreated patients with primary immunodeficiency: a prospective, multicenter study. J Cli — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Patients Achieving Immunoglobulin G (IgG) Levels = 5 g/L on Day 12		On Day 12	No
Secondary	Proportion of Patients Achieving IgG Levels = 5 g/L on Day 19		On Day 19	No
Secondary	Proportion of Patients Achieving IgG Levels = 5 g/L on Day 26		On Day 26	No
Secondary	IgG Increase (Change From Baseline) on Day 12		Baseline to Day 12	No
Secondary	Overall Rate of Infections	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".	For the duration of the study, up to approximately 25 weeks	No
Secondary	Total Serum IgG Trough Levels on Day 12		On Day 12	No
Secondary	Total Serum IgG Trough Levels at Week 25		At Week 25	No
Secondary	Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles on Day 12		On Day 12	No
Secondary	Serum Concentrations of Specific IgGs Against Cytomegalovirus, Tetanus, and Measles at Week 25		At Week 25	No
Secondary	Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae On Day 12		On Day 12	No
Secondary	Serum Concentrations of Specific IgGs Against H. Influenzae Type B and S. Pneumoniae at Week 25		At Week 25	No
Secondary	Use of Antibiotics for Infection Prophylaxis and Treatment	Number of patients. Medications were classified as antibiotics according to the anatomic therapeutic chemical code.	For the duration of the study, up to approximately 25 weeks	No
Secondary	Quality of Life as Measured by the Adapted Short Form-36 Health Survey (SF-36; Age = 14 Years)	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.	At study completion, approximately Week 25	No
Secondary	Quality of Life as Measured by the Child Health Questionnaire Parent Form-50 (CHQ-PF50; Age = 13 Years)	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.	At study completion, approximately Week 25	No
Secondary	Number of Patients With Adverse Events (AEs) by Severity and Relatedness	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.	For the duration of the study, up to approximately 25 weeks	Yes
Secondary	Rate of AEs by Severity and Relatedness	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.	For the duration of the study, up to approximately 25 weeks	Yes
Secondary	Number of Patients With Local Reactions by Severity and Relatedness	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.	For the duration of the study, up to approximately 25 weeks	Yes
Secondary	Rate of Local Reactions by Severity and Relatedness	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.	For the duration of the study, up to approximately 25 weeks	Yes
Secondary	Number of Patients With Clinically Relevant Changes in Routine Laboratory Parameters	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).	At Weeks 12 and 25	Yes
Secondary	Number of Patients With Clinically Relevant Changes in Vital Signs	Vital signs included heart rate, systolic blood pressure, diastolic blood pressure, and body temperature.	At the screening visit, before and after infusions (Days 1 to 5), and at the completion visit (Week 25)	Yes

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