Primary ImmunodeficiencyPhase 3NCT01581593
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
A Phase 3 study testing Kedrion IVIG 10% in people with Primary Immunodeficiency and Agammaglobulinemia, run by Kedrion S.p.A. at 15 sites in 2 countries.
Trial summary
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID). Read more about this study
- Who is it for?
- Ages 2 to 70 years
- Conditions the study is for
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia
- What is being tested?
- Kedrion IVIG 10%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 15 sitesin 2 countries
- Study dates
- 12 Nov 2012to 27 Aug 2014
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
15 sites in 2 countries.
15 sites in 2 countries
United States12 sites
Family Allergy & Asthma Center, PC
Atlanta, Georgia, 30342
Rush University
Chicago, Illinois, 60612
Optimed Research, LTD
Columbus, Ohio, 43235
Dallas Allergy Immunology Research
Dallas, Texas, 75230
AARA Research Center
Dallas, Texas, 75231
University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Allergy Associates of the Palm Beaches
North Palm Beach, Florida, 33408
Midwest Immunology Clinic
Plymouth, Minnesota, 55446
Virginia Commonwealth University Health Systems
Richmond, Virginia, 23298
AAIR Research Center
Rochester, New York, 14618
Marycliff Allergy Specialists
Spokane, Washington, 99204
Canada3 sites
Gordon Sussman Clinical Research Inc.
Toronto, Ontario, M4V1R2
Pediatric & Adult Allergy & Clinical Immunology
Toronto, Ontario, M5G1E2
The Hospital for Sick Children
Toronto, Ontario, M5G1X8
About this study
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).
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
Read the detailed 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.
- Being tested
- Kedrion IVIG 10%
- Keywords
- Primary Immunodeficiency, PID, Agammaglobulinemia, Hypogammaglobulinemia, Antibody deficiency
- Medical subject headings
- Primary Immunodeficiency Diseases, Agammaglobulinemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Kedrion IVIG 10%Biological
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
| Study group | Role | What they receive |
|---|---|---|
| Kedrion IVIG 10% | Experimental | Kedrion IVIG 10% treatment. Receives: Biological: Kedrion IVIG 10% |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 13 months
Secondary outcomes (14)
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population.
Measured: 13 months
Days Out of Work/School/Daycare Due to Infection in the Total ITT Population.
Measured: 13 months
Days Unable to Perform Normal Daily Activities Due to Infection in the Total ITT Population.
Measured: 13 months
Days on Therapeutic Antibiotics in the Total ITT Population.
Measured: 13 months
Days of Unscheduled Visits to Physicians in the Total ITT Population.
Measured: 13 months
Number of Hospitalizations Due to Infection in ITT Population.
Measured: 13 months
Days of Hospitalization Due to Infection in the Total ITT Population.
Measured: 13 months
Yearly Hospitalization Rate Due to Infection in the Total ITT Population.
Measured: 13 months
Yearly Hospitalization Duration Due to Infection in the Total ITT Population.
Measured: 13 months
Distribution of All-cause Hospitalizations in the Total ITT Population.
Measured: 13 months
Duration of All-cause Hospitalizations
Measured: 13 months
Distribution of Fever Episodes in the Total ITT Population.
Measured: 13 months
Duration of Fever Episodes
Measured: 13 months
IgG Trough Levels at Steady State in the Total ITT Population.
Measured: 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2012 (estimated)
- Study start12 Nov 2012
- Primary completion27 Aug 2014
- Study completion27 Aug 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary ImmunodeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kedrion S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mirella Calcinai, MD, Study director, Kedrion SpA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01581593
- Sponsor's protocol ID
- KB052
- Status last verified
- Jan 2021
- First submitted
- 16 Apr 2012
- Last updated
- 16 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT01581593
- Acronym / sponsor ID
- KB052
- Keywords
- Primary Immunodeficiency, PID, Agammaglobulinemia, Hypogammaglobulinemia, Antibody deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Kedrion S.p.A.Industry (pharma, biotech, devices)
- Study start
- 12 Nov 2012 (actual)
- Primary completion
- 27 Aug 2014 (actual)
- Study completion
- 27 Aug 2014 (actual)
- First posted
- 20 Apr 2012 (estimated)
- Results
- Posted 16 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Kedrion IVIG 10% | Experimental | Biological: Kedrion IVIG 10%Kedrion IVIG 10% treatment. |
Who may qualify
- Ages
- 2 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (18 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Biological | Kedrion IVIG 10% |
Primary outcome
Incidence of Acute, Serious Bacterial Infections in the Total ITT Population.
Time frame: 13 months
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.
14 secondary outcomes
Infections Other Than Acute, Serious Bacterial Infections (ASBIs) in the Total ITT Population.
Time frame: 13 months
Days Out of Work/School/Daycare Due to Infection in the Total ITT Population.
Time frame: 13 months
Days Unable to Perform Normal Daily Activities Due to Infection in the Total ITT Population.
Time frame: 13 months
Days on Therapeutic Antibiotics in the Total ITT Population.
Time frame: 13 months
Days of Unscheduled Visits to Physicians in the Total ITT Population.
Time frame: 13 months
Number of Hospitalizations Due to Infection in ITT Population.
Time frame: 13 months
Days of Hospitalization Due to Infection in the Total ITT Population.
Time frame: 13 months
Yearly Hospitalization Rate Due to Infection in the Total ITT Population.
Time frame: 13 months
Yearly Hospitalization Duration Due to Infection in the Total ITT Population.
Time frame: 13 months
Distribution of All-cause Hospitalizations in the Total ITT Population.
Time frame: 13 months
Duration of All-cause Hospitalizations
Time frame: 13 months
Distribution of Fever Episodes in the Total ITT Population.
Time frame: 13 months
Duration of Fever Episodes
Time frame: 13 months
IgG Trough Levels at Steady State in the Total ITT Population.
Time frame: 13 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mirella Calcinai, MD study director
Kedrion SpA
15 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Kedrion IVIG 10% in people with Primary Immunodeficiency and Agammaglobulinemia, run by Kedrion S.p.A. at 15 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Acute, Serious Bacterial Infections in the Total ITT Population. (time frame: 13 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Feb 2021.