- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01814800
Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
August 10, 2016 updated by: ADMA Biologics, Inc.
An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)
This is a Phase III, multicenter, open-label study of RI-002 administered as an intravenous infusion of RI-002 (IGIV) every 21 or 28 days in approximately 60 subjects with Primary Immunodeficiency Diseases (PIDD).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
Primary immunodeficiency diseases (PIDDs) are genetically determined disorders of the immune system resulting in greatly enhanced susceptibility to infectious disease, autoimmunity and malignancy.
As most subjects with PIDDs present with infections, the differential diagnosis and initial investigations for an underlying immune defect are typically guided by the clinical presentation.
In subjects with PIDDs, individual infections are not necessarily more severe than those that occur in a normal host.
Rather, the clinical features suggestive of an immune defect may be the recurring and/or chronic nature of infections with common pathogens that may result in end organ damage, such as bronchiectasis.
Several immune globulin products have already been approved by the FDA.
Study Type
Interventional
Enrollment (Actual)
59
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Colorado
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Cenntennial, Colorado, United States, 80112
- Immunoe Health Centers
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Florida
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North Palm Beach, Florida, United States, 33408
- Allergy Associates of the Palm Beaches, P.A.
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Georgia
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Atlanta, Georgia, United States, 30342
- Family Allergy Center, PC
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Indiana
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South Bend, Indiana, United States, 46617
- The South Bend Clinic, LLP
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Nebraska
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Omaha, Nebraska, United States, 68124
- Asthma & Immunology Associates
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New York
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NY, New York, United States, 10029
- Mount Sinai School of Medicine
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Texas
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Dallas, Texas, United States, 75231
- AARA Research Center
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Dallas, Texas, United States, 75230
- Dallas Immunology Research
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Houston, Texas, United States, 77030
- Baylor Texas Children's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years to 75 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
To be eligible to participate in this study, the subjects must meet the following criteria:
- Signed a written informed consent or a specific assent form for minors.
- Have a diagnosis of primary immunodeficiency disease.
- Be ≥ 2 years and ≤ 75 years.
- Have body weight ≥ 12 kg at screening.
- Have been receiving IGIV at a dose that has not been changed by >50% of the mean dose on a mg/kg basis for at least 3 months prior to study entry and have maintained a trough serum Immunoglobulin G (IgG) level ≥ 500 mg/dL on the previous 2 assessments prior to receiving RI 002. The trough level must be at least 300 mg/dL above the pre-treatment serum IgG level.
- For female subjects, be of non-childbearing potential or have a negative pregnancy test prior to study start and be deemed not at risk of becoming pregnant by adherence to a reliable contraceptive method for the duration of the study.
Exclusion Criteria:
Subjects must be excluded if they meet any of the following criteria:
- Have a known hypersensitivity to immunoglobulin or any excipient in RI-002.
- Have a history of a severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
- Have a specific Immunoglobulin A (IgA) deficiency, history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
- Have uncompensated hemodynamically significant congenital or other heart disease.
- Have a medical condition that is known to cause secondary immune deficiency.
- Have a significant T-cell deficiency or deficiency of granulocyte number or function.
- Have significant renal impairment or have a history of acute renal failure.
- Have abnormal liver function.
- Be receiving chronic anti-coagulation therapy.
- Have a history of deep vein thrombosis (DVT), thrombotic or thrombo-embolic event, or are at increased risk for thrombotic events.
Current daily use of the following medications:
- corticosteroids (> 7.5 mg (or equivalent dose on a mg/kg basis) of prednisone equivalent per day for > 30 days)
- immunomodulatory drugs
- immunosuppressive drugs (excluding topical pimecrolimus (Elidel) and tacrolimus (Protopic))
- Administration of a hyperimmune or specialty high titer immunoglobulin product.
- Have uncontrollable arterial hypertension.
- Have a history of hemolysis or positive Coombs test while undergoing treatment with IGIV therapy.
- Be morbidly obese as indicated by a Body Mass Index (BMI) ≥ 40
- Have received any blood product (other than Immunoglobulin G) within 3 months prior to screening.
- Have received any Respiratory Syncytial Virus (RSV) specific products, including palivizumab (Synagis®) within 3 months prior to screening.
- Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
- Have any condition or abnormal laboratory assessment judged by the investigator to preclude participation in the study.
- Are currently pregnant or nursing.
- Have hepatitis A, B, or C.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: RI-002 Treatment
Drug: RI-002 Dose: 300-800 mg/kg infusion Frequency: Once every 3 to 4 Weeks
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Immune Globulin Intravenous (IGIV)
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008))
Time Frame: One year
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The primary objective of this study was to demonstrate that RI-002 (IGIV) reduces the frequency of serious bacterial infections (SBIs), as defined by the Diagnostic Criteria for Serious Infection Types guideline, in subjects with primary humoral immunodeficiency.
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One year
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Incidence of All Infections (Serious and Non-serious)
Time Frame: Up to 1 Year
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Up to 1 Year
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Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Combined Days Lost
Time Frame: Up to 1 year
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Up to 1 year
|
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Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Per Subject-Year
Time Frame: Up to 1 year
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Up to 1 year
|
|
Number of Unscheduled Visits to Physician/ER Due to Infections - Total Number of Visits
Time Frame: Up to 1 year
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Up to 1 year
|
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Number of Unscheduled Visits to Physician/ER Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
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Up to 1 year
|
|
Time to Resolution of Infections - Duration Per Infection
Time Frame: Up to 1 year
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Up to 1 year
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Time to Resolution of Infections - Infection Days Per Subject
Time Frame: Up to 1 year
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Up to 1 year
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Number of Hospitalizations Due to Infections
Time Frame: Up to 1 year
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Up to 1 year
|
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Number of Hospitalizations Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
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Up to 1 year
|
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Days of Hospitalization Due to Infections
Time Frame: Up to 1 year
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Up to 1 year
|
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Days of Hospitalization Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
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Up to 1 year
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Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection)
Time Frame: Up to 1 year
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Summary of the days of antibiotic therapy in the study for prophylaxis, as treatment for infections, and combined
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Up to 1 year
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Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) - Per Subject-Year
Time Frame: Up to 1 year
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Summary of the days of antibiotic therapy in the study for prophylaxis, as treatment for infections, and combined, per subject-year of treatment with RI-002
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Up to 1 year
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Correlation Between Trough Level of RI-002 and Serious and Non-serious Infections
Time Frame: Up to 1 year
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The relationship between trough IgG concentrations and the number of infections of any kind/seriousness was evaluated using Pearson linear correlation coefficients using forward analysis (outcomes after infusion) and backward analysis (outcomes prior to infusion; outcomes post last infusion were excluded)
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - IgG
Time Frame: Up to 1 year
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Summary of trough total IgG concentration prior to specified infusion
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Haemophilus Influenzae Type B
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Haemophilus influenzae type B
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Respiratory Syncytial Virus (RSV)
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Respiratory Syncytial Virus (RSV)
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Tetanus
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Tetanus
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 1
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 1
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 3
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 3
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 4
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 4
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 5
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 5
|
Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 6B
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 6B
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Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 7F
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 7F
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Up to 1 year
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Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 9V
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 9V
|
Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 14
Time Frame: Up to 1 year
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Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 14
|
Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 18C
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 18C
|
Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 19A
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 19A
|
Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 19F
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 19F
|
Up to 1 year
|
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 23F
Time Frame: Up to 1 year
|
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 23F
|
Up to 1 year
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: James Mond, M.D., Ph.D., ADMA Biologics, Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
February 1, 2014
Primary Completion (Actual)
December 1, 2014
Study Completion (Actual)
January 1, 2015
Study Registration Dates
First Submitted
March 6, 2013
First Submitted That Met QC Criteria
March 18, 2013
First Posted (Estimate)
March 20, 2013
Study Record Updates
Last Update Posted (Estimate)
October 5, 2016
Last Update Submitted That Met QC Criteria
August 10, 2016
Last Verified
August 1, 2016
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ADMA-003
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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