Primary ImmunodeficiencyPhase 3NCT02269163
Study of ProMetic BioTherapeutics Immune Globulin Intravenous (Human) 10%
A Phase 3 study testing Immune Globulin Intravenous and Prometic's Immune Globulin Intravenous 10% in people with Primary Immunodeficiency, run by Prometic Biotherapeutics, Inc. at 13 sites in United States.
Trial summary
Phase 3 multicenter, open-label study of safety, tolerability, efficacy, and pharmacokinetics (PK) of ProMetic's Immune Globulin Intravenous (Human) 10%, the investigational medicinal product [IMP]), in Adults and Children with Primary Immunodeficiency Diseases (PIDD). Read more about this study
- Who is it for?
- Ages 2 to 80 years
- Conditions the study is for
- Primary Immunodeficiency
- What is being tested?
- Immune Globulin Intravenous and Prometic's Immune Globulin Intravenous 10%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 82 participants
- Where
- 13 sitesUnited States
- Study dates
- 26 Jan 2016to 11 Jan 2019
- 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 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is male or female between the ages of 2 and 80 years at Screening.
- Female subjects of childbearing potential must agree to employ adequate birth control measures, as determined by their IRB/IEC, for the duration of the study.
- The subject must have one of the following three diagnoses (isolated PIDD of other types will be excluded):
- Common variable immunodeficiency
- X-linked agammaglobulinemia
- Hyper-IgM syndrome and documented low IgG levels (<4.5 mg/mL [450 mg/dL]).
- Subjects must have been treated with a stable dose of immune globulin administered intravenously (IGIV) or subcutaneously (IGSC) and has documented trough or steady state IgG levels of ≥ 5 mg/mL.
Who cannot join
- Subject has secondary immunodeficiency or has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency; has known hypoalbuminemia (<3 gm/dL), protein-losing enteropathy, or nephrotic syndrome.
- Subject has ever had a history of severe anaphylactic or anaphylactoid reaction to immunoglobulins or other blood products.
- Subject has a known history of immunoglobulin A (IgA) deficiency and known anti-IgA antibodies, thrombotic event, such as deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism, at any time.
- Subject has received blood products except IGIV, IGSC, or albumin within the previous 12 months or has participated in another study (except for IGIV, IGSC studies) within the previous 4 weeks.
- Subject has had cancer in the past 5 years, except for basal cell or squamous cell cancers of the skin.
- Subject has had a documented active infection within 7 days prior to Screening, or subject is on continuous prophylactic antibiotics.
Show all 17Show fewer
- Subject is positive for human immunodeficiency virus (HIV)-1 or HIV-2, a positive hepatitis C virus (HCV) or hepatitis B virus (HBV).
- Subject has levels of alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2.5 times the upper limit of normal (ULN).
- Subject has serum creatinine >1.5 times the ULN or a severe chronic condition such as renal failure with proteinuria.
- Subject has anemia with a hemoglobin level ≤8 g/dL.
- Subject has severe neutropenia with neutrophil count ≤1000 per mmᴧ3 or has lymphopenia with <500 per/ mmᴧ3.
- Subject is taking prednisone at a dose ≥0.15 mg/kg/day and receiving other immunosuppressive drugs or chemotherapy.
- Subject has known atrial fibrillation requiring anticoagulant therapy; congestive heart failure (New York Heart Association Class III/IV); cardiomyopathy; or cardiac arrhythmia associated with thromboembolic events, unstable or advanced ischemic heart disease, or hyperviscosity.
- Subject has known decreased Protein C and/or Protein S levels.
- Subject is positive for antibodies to β2GPI and/or β2GPI DI at Screening.
- Female subject who is pregnant, breast-feeding, or planning a pregnancy during the course of the study.
- A history of epilepsy or multiple episodes of migraine (defined as at least one episode within 6 months of enrolment) not completely controlled by medication, or any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study in the Investigator's opinion.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Bellingham Asthma, Allergy and Immunology Clinic
Bellingham, Washington, 98225
Immunoe International Research
Centennial, Colorado, 80112
Rush University Medical Center
Chicago, Illinois, 60612
Optimed Research
Columbus, Ohio, 43236
Dallas Allergy Immunology
Dallas, Texas, 75230
National Jewish Hospital
Denver, Colorado, 80206
Duke University Medical Center
Durham, North Carolina, 27710
Fort Wayne Medical Institute
Fort Wayne, Indiana, 46815
University of California, Irvine
Irvine, California, 92697
Children's Hospital Los Angeles
Los Angeles, California, 90027
Allergy Associates of the Palm Beaches
North Palm Beach, Florida, 33408
St. Louis University
St Louis, Missouri, 63104
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
About this study
Official title: A Phase 3, Multicenter, Open-Label Study of the Safety, Tolerability, Efficacy, and PK of ProMetic BioTherapeutics IGIV (Human) 10% in Adults and Children With Primary Immunodeficiency Diseases
Phase 3 multicenter, open-label study of safety, tolerability, efficacy, and pharmacokinetics (PK) of ProMetic's Immune Globulin Intravenous (Human) 10%, the investigational medicinal product [IMP]), in Adults and Children with Primary Immunodeficiency Diseases (PIDD).
Read the detailed description
This is a pivotal Phase 3, open-label, single-arm, multicenter study to assess the tolerability, safety, efficacy, and Pharmacokinetics of the Investigational Medicinal Product in adults and children with Primary Immunodeficiency Diseases (PIDD). A total of approximately 75 subjects aged 2-80 years will be enrolled in the study. Subjects who switch from an investigational immune globulin or subcutaneous immune globulin (IGSC) are required to receive a stable dose of commercial product (CP), which is a licensed commercially available immune globulin intravenous (IGIV) product for at least 3 cycles before they can be given the Investigational Medicinal Product . This study schema will result in the Commercial Product Treatment Period and Investigational Medicinal Product Treatment Period. All subjects will be treated on an outpatient basis with the Investigational Medicinal Product for approximately 1 year, with the dose and schedule based on their previous IGIV treatment regimen (21-day or 28-day dosing interval). A subset of subjects will participate in a Pharmacokinetics sub-study.
The primary objective of the study is to examine the rate of clinically documented serious bacterial infections (SBIs) in subjects treated with the Investigational Medicinal Product to achieve a rate of less than one SBI per year.
- Conditions
- Primary Immunodeficiency
- Being tested
- Immune Globulin Intravenous, Prometic's Immune Globulin Intravenous 10%
- Medical subject headings
- Primary Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Immune Globulin IntravenousBiological
Gammargard, Gammaplex,Gamunex, or Octogam IGIV Product
Prometic's Immune Globulin Intravenous 10%Biological
Liquid formulation of Prometic Immune Globulin Intravenous 10% (human) in 50 mL vials containing 100 mg/mL of immunoglobulin G (IgG)
| Study group | Role | What they receive |
|---|---|---|
| Gammargard, Gammaplex, Gamunex, or Octogam Treatment Period | Active comparator | Subjects who enroll in the study while on Gammargard, Gammaplex, Gamunex, or Octogam IGIV Product and need to wait for the scheduled start of Prometic IGIV (10%) treatment will continue on their usual dose and treatment cycle with Gammargard, Gammaplex, Gamunex, or Octogam IVIG Product during this period. Receives: Biological: Immune Globulin Intravenous |
| Prometic IGIV 10% Treatment Period | Experimental | Subjects will receive Prometic Immune Globulin Intravenous 10% Receives: Biological: Prometic's Immune Globulin Intravenous 10% |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Rate of Occurrence of Serious Bacterial Infections (SBI)
SBIs were calculated for each subject as 52n/w, where n is the number of reported SBIs and w is the number of weeks on study. For the combined cohorts only, a 99% one-sided (upper) confidence limit for the incidence rate of SBIs (scaled to represent 12 months exposure if necessary) was derived, and the objective of demonstrating that the true infection rate was below 1 per subject per year was considered established if this upper limit was less than 1. To calculate the confidence limit, a negative binomial regression model will be used. This model includes an overdispersion parameter to account for possible intra-subject correlation as well as the actual time period each subject is on the study as an offset variable.
Measured: One year
Secondary outcomes (1)
Trough Levels of IgG (Total) Prior to Each Prometic IGIV 10% Infusion
Subject's total IgG levels will be assessed prior to each Prometic IGIV 10% infusion
Measured: One year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2014 (estimated)
- Study start26 Jan 2016
- Primary completion11 Jan 2019
- Study completion11 Jan 2019
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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 82 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prometic Biotherapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Atlantic Research Group
- Responsible party
- Sponsor
- Investigators
- James Moy, MD, Study chair, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02269163
- Sponsor's protocol ID
- 2004C009G
- Status last verified
- Nov 2021
- First submitted
- 16 Oct 2014
- Last updated
- 5 Nov 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Open-Label Study of the Safety, Tolerability, Efficacy, and PK of ProMetic BioTherapeutics IGIV (Human) 10% in Adults and Children With Primary Immunodeficiency Diseases
- ClinicalTrials.gov ID
- NCT02269163
- Acronym / sponsor ID
- 2004C009G
- Conditions
- Primary Immunodeficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 82 participants (actual)
- Lead sponsor
- Prometic Biotherapeutics, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Atlantic Research Group
- Study start
- 26 Jan 2016 (actual)
- Primary completion
- 11 Jan 2019 (actual)
- Study completion
- 11 Jan 2019 (actual)
- First posted
- 20 Oct 2014 (estimated)
- Results
- Posted 20 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Phase 3 multicenter, open-label study of safety, tolerability, efficacy, and pharmacokinetics (PK) of ProMetic's Immune Globulin Intravenous (Human) 10%, the investigational medicinal product [IMP]), in Adults and Children with Primary Immunodeficiency Diseases (PIDD).
Detailed description
This is a pivotal Phase 3, open-label, single-arm, multicenter study to assess the tolerability, safety, efficacy, and Pharmacokinetics of the Investigational Medicinal Product in adults and children with Primary Immunodeficiency Diseases (PIDD). A total of approximately 75 subjects aged 2-80 years will be enrolled in the study. Subjects who switch from an investigational immune globulin or subcutaneous immune globulin (IGSC) are required to receive a stable dose of commercial product (CP), which is a licensed commercially available immune globulin intravenous (IGIV) product for at least 3 cycles before they can be given the Investigational Medicinal Product . This study schema will result in the Commercial Product Treatment Period and Investigational Medicinal Product Treatment Period. All subjects will be treated on an outpatient basis with the Investigational Medicinal Product for approximately 1 year, with the dose and schedule based on their previous IGIV treatment regimen (21-day or 28-day dosing interval). A subset of subjects will participate in a Pharmacokinetics sub-study.
The primary objective of the study is to examine the rate of clinically documented serious bacterial infections (SBIs) in subjects treated with the Investigational Medicinal Product to achieve a rate of less than one SBI per year.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Gammargard, Gammaplex, Gamunex, or Octogam Treatment Period | Active comparator | Biological: Immune Globulin IntravenousSubjects who enroll in the study while on Gammargard, Gammaplex, Gamunex, or Octogam IGIV Product and need to wait for the scheduled start of Prometic IGIV (10%) treatment will continue on their usual dose and treatment cycle with Gammargard, Gammaplex, Gamunex, or Octogam IVIG Product during this period. |
| Prometic IGIV 10% Treatment Period | Experimental | Biological: Prometic's Immune Globulin Intravenous 10%Subjects will receive Prometic Immune Globulin Intravenous 10% |
Who may qualify
- Ages
- 2 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Subject is male or female between the ages of 2 and 80 years at Screening.
- Female subjects of childbearing potential must agree to employ adequate birth control measures, as determined by their IRB/IEC, for the duration of the study.
- The subject must have one of the following three diagnoses (isolated PIDD of other types will be excluded):
- Subjects must have been treated with a stable dose of immune globulin administered intravenously (IGIV) or subcutaneously (IGSC) and has documented trough or steady state IgG levels of ≥ 5 mg/mL.
Key exclusion criteria (17 listed)
- Subject has secondary immunodeficiency or has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency; has known hypoalbuminemia (<3 gm/dL), protein-losing enteropathy, or nephrotic syndrome.
- Subject has ever had a history of severe anaphylactic or anaphylactoid reaction to immunoglobulins or other blood products.
- Subject has a known history of immunoglobulin A (IgA) deficiency and known anti-IgA antibodies, thrombotic event, such as deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism, at any time.
- Subject has received blood products except IGIV, IGSC, or albumin within the previous 12 months or has participated in another study (except for IGIV, IGSC studies) within the previous 4 weeks.
- Subject has had cancer in the past 5 years, except for basal cell or squamous cell cancers of the skin.
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 |
|---|---|---|
| Immune Globulin IntravenousGammargard, Gammaplex,Gamunex, or Octogam IGIV Product | Biological | Gammargard, Gammaplex, Gamunex, or Octogam Treatment Period |
| Prometic's Immune Globulin Intravenous 10%Liquid formulation of Prometic Immune Globulin Intravenous 10% (human) in 50 mL vials containing 100 mg/mL of immunoglobulin G (IgG) | Biological | Prometic IGIV 10% Treatment Period |
Primary outcome
Annual Rate of Occurrence of Serious Bacterial Infections (SBI)
Time frame: One year
SBIs were calculated for each subject as 52n/w, where n is the number of reported SBIs and w is the number of weeks on study. For the combined cohorts only, a 99% one-sided (upper) confidence limit for the incidence rate of SBIs (scaled to represent 12 months exposure if necessary) was derived, and the objective of demonstrating that the true infection rate was below 1 per subject per year was considered established if this upper limit was less than 1. To calculate the confidence limit, a negative binomial regression model will be used. This model includes an overdispersion parameter to account for possible intra-subject correlation as well as the actual time period each subject is on the study as an offset variable.
1 secondary outcome
Trough Levels of IgG (Total) Prior to Each Prometic IGIV 10% Infusion
Time frame: One year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
James Moy, MD study chair
Rush University Medical Center
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Immune Globulin Intravenous and Prometic's Immune Globulin Intravenous 10% in people with Primary Immunodeficiency, run by Prometic Biotherapeutics, Inc. at 13 sites in United States.
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 7 inclusion and 17 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 5 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States. 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 20 Aug 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?
Annual Rate of Occurrence of Serious Bacterial Infections (SBI) (time frame: One year). 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 5 Nov 2021.