Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)

Primary Immunodeficiency Clinical Trial

Official title:

Multicenter, Open-label, Historically Controlled, Phase III Study to Assess the Efficacy, Tolerability, Safety and Pharmacokinetics of Kedrion IVIG 10% in Adult and Pediatric Subjects With Primary Immunodeficiency (PID).

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01581593
• Other study ID #: KB052
• Status: Completed
• Phase: Phase 3
• Start date: November 12, 2012
• Est. completion date: August 27, 2014

Study information

• Verified date: January 2021
• Source: Kedrion S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).

Description:

People with primary immunodeficiency diseases (PID) have a defective immune system and experience recurrent protozoal, bacterial, fungal and viral infections. Antibody deficiencies make up the largest group of PIDs.

The standard care for patients with PID is replacement immunoglobulin (a class of antibodies) solution. Prophylactic treatment with intravenous immunoglobulin (IVIG) solution has been shown to increase the time free from serious infection.

Kedrion IVIG 10% is a new preparation of an immunoglobulin G (IgG) solution. Kedrion IVIG 10% will be given by IV infusion to all study participants. The data collected will help determine whether Kedrion IVIG 10% is suitable for treating PID subjects.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: August 27, 2014
• Est. primary completion date: August 27, 2014
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 70 Years

Eligibility

Inclusion Criteria:

• Confirmed clinical diagnosis of a Primary Immunodeficiency Disease

• Male or female, ages 2 to 70 years

• Received 300-900 mg/kg of a licensed IVIG therapy at 21 or 28 day intervals for at least 3 months prior to this study

• 2 documented IgG trough levels of = 5 g/L are obtained at two infusion cycles (21 or 28 days) within 12 months (one must be within 6 months) prior to study enrolment

• Non-pregnant females of child-bearing potential who agree to use adequate birth control during the study

• Subject is willing to comply with the protocol

• Authorization to access personal health information.

• Signed the informed consent form and a child assent form, if appropriate.

• If currently participating in a clinical trial with another experimental IVIG may be enrolled if they have received stable IVIG therapy for at least 3 infusion cycles prior to receiving Kedrion IVIG 10% and all inclusion and exclusion criteria are satisfied

• If currently participating in a trial of SCIG can be enrolled if they are switched to IVIG for three infusion cycles (21 or 28 days) prior to enrolment in this study

Exclusion Criteria:

• Has secondary immunodeficiency.

• Newly diagnosed and has not been treated with immunoglobulin or has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency.

• Has a history of repeated reactions or hypersensitivity to IVIG or other injectable forms of IgG.

• Has a history of thrombotic events defined by at least 1 event in subject's lifetime.

• Has IgA deficiency and is known to have antibodies to IgA.

• Has received blood products other than human albumin or human immunoglobulin within 12 months prior to enrolment.

• Has significant protein losing enteropathy, nephrotic syndrome or lymphangiectasia.

• Has an acute infection as documented by culture or diagnostic imaging and/or a body temperature exceeding 38.5 °C (101.3 °F) within 7 days prior to screening

• Has a known history or is positive at enrolment for human immunodeficiency virus (HIV) type 1 by NAT, hepatitis B virus (HBsAg and NAT), hepatitis C virus (by NAT), or hepatitis A virus (by NAT).

• Has levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2.5 times of the upper limit of normal for the laboratory designated for the study.

• Has an implanted venous access device

• Has profound anemia or persistent severe neutropenia (= 1000 neutrophils per mm3)or lymphopenia of less than 500 cells per microliter.

• Has a severe chronic condition such as renal failure (creatinine concentration > 2.0 times the upper limit of normal) with proteinuria, congestive heart failure (New York Heart Association III/IV), cardiomyopathy, cardiac arrhythmia associated with thromboembolic events (e.g. atrial fibrillation), unstable or advanced ischemic heart disease, hyperviscosity, or any other condition that the investigator believes is likely to interfere with evaluation of the study drug or with satisfactory conduct of the trial.

• Has a history of a malignant disease other than properly treated carcinoma in situ of the cervix or basal cell or squamous cell carcinoma of the skin within 24 months prior to enrolment.

• Has history of epilepsy or multiple episodes of migraine (defined as at least one episode within 6 months of enrolment) not completely controlled by medication.

• Is receiving steroids (oral or parenteral daily dose of = 0.15 mg/kg/day of prednisone or equivalent) OR other immunosuppressive drugs or chemotherapy.

• Females who are pregnant, breast feeding or planning a pregnancy during the course of the study. Women who become pregnant during the study will be withdrawn from the study.

• Has participated in another clinical study within 3 weeks prior to study enrolment.

Related Conditions & MeSH terms

• Agammaglobulinemia
• Antibody Deficiency
• Hypogammaglobulinemia
• Immunologic Deficiency Syndromes
• Primary Immunodeficiency

Intervention

Biological:
• Kedrion IVIG 10%
Dosage form - Intravenous (IV) infusion of Kedrion IVIG 10%; Dosage - 300 to 900 mg/kg body weight (bw); Frequency - every 21 to 28 days; Treatment duration - 12 months

Locations

Country	Name	City	State
Canada	Gordon Sussman Clinical Research Inc.	Toronto	Ontario
Canada	Pediatric & Adult Allergy & Clinical Immunology	Toronto	Ontario
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	Family Allergy & Asthma Center, PC	Atlanta	Georgia
United States	Rush University	Chicago	Illinois
United States	Optimed Research, LTD	Columbus	Ohio
United States	AARA Research Center	Dallas	Texas
United States	Dallas Allergy Immunology Research	Dallas	Texas
United States	University of Iowa Hospital and Clinics	Iowa City	Iowa
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Allergy Associates of the Palm Beaches	North Palm Beach	Florida
United States	Midwest Immunology Clinic	Plymouth	Minnesota
United States	Virginia Commonwealth University Health Systems	Richmond	Virginia
United States	AAIR Research Center	Rochester	New York
United States	Marycliff Allergy Specialists	Spokane	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Kedrion S.p.A.

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Acute, Serious Bacterial Infections in the Total ITT Population.	The incidence of acute serious bacterial infections, e.g. bacterial pneumonia, bacteremia/sepsis, bacterial meningitis, visceral abscess, osteomyelitis/septic arthritis, meeting EMA and FDA criteria.	13 months
Secondary	Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population.		13 months
Secondary	Days Out of Work/School/Daycare Due to Infection in the Total ITT Population.		13 months
Secondary	Days Unable to Perform Normal Daily Activities Due to Infection in the Total ITT Population.		13 months
Secondary	Days on Therapeutic Antibiotics in the Total ITT Population.		13 months
Secondary	Days of Unscheduled Visits to Physicians in the Total ITT Population.		13 months
Secondary	Number of Hospitalizations Due to Infection in ITT Population.		13 months
Secondary	Days of Hospitalization Due to Infection in the Total ITT Population.		13 months
Secondary	Yearly Hospitalization Rate Due to Infection in the Total ITT Population.		13 months
Secondary	Yearly Hospitalization Duration Due to Infection in the Total ITT Population.		13 months
Secondary	Distribution of All-cause Hospitalizations in the Total ITT Population.		13 months
Secondary	Duration of All-cause Hospitalizations		13 months
Secondary	Distribution of Fever Episodes in the Total ITT Population.		13 months
Secondary	Duration of Fever Episodes		13 months
Secondary	IgG Trough Levels at Steady State in the Total ITT Population.		13 months