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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
59

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

  • United States
    • Colorado
      • Cenntennial, Colorado, United States, 80112
        • Immunoe Health Centers
    • Florida
      • North Palm Beach, Florida, United States, 33408
        • Allergy Associates of the Palm Beaches, P.A.
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Family Allergy Center, PC
    • Indiana
      • South Bend, Indiana, United States, 46617
        • The South Bend Clinic, LLP
    • Nebraska
      • Omaha, Nebraska, United States, 68124
        • Asthma & Immunology Associates
    • New York
      • NY, New York, United States, 10029
        • Mount Sinai School of Medicine
    • Texas
      • Dallas, Texas, United States, 75231
        • AARA Research Center
      • Dallas, Texas, United States, 75230
        • Dallas Immunology Research
      • Houston, Texas, United States, 77030
        • Baylor Texas Children's Hospital

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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:

  1. Signed a written informed consent or a specific assent form for minors.
  2. Have a diagnosis of primary immunodeficiency disease.
  3. Be ≥ 2 years and ≤ 75 years.
  4. Have body weight ≥ 12 kg at screening.
  5. 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.
  6. 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:

  1. Have a known hypersensitivity to immunoglobulin or any excipient in RI-002.
  2. Have a history of a severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
  3. Have a specific Immunoglobulin A (IgA) deficiency, history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
  4. Have uncompensated hemodynamically significant congenital or other heart disease.
  5. Have a medical condition that is known to cause secondary immune deficiency.
  6. Have a significant T-cell deficiency or deficiency of granulocyte number or function.
  7. Have significant renal impairment or have a history of acute renal failure.
  8. Have abnormal liver function.
  9. Be receiving chronic anti-coagulation therapy.
  10. Have a history of deep vein thrombosis (DVT), thrombotic or thrombo-embolic event, or are at increased risk for thrombotic events.

  11. 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))
  12. Administration of a hyperimmune or specialty high titer immunoglobulin product.
  13. Have uncontrollable arterial hypertension.
  14. Have a history of hemolysis or positive Coombs test while undergoing treatment with IGIV therapy.
  15. Be morbidly obese as indicated by a Body Mass Index (BMI) ≥ 40
  16. Have received any blood product (other than Immunoglobulin G) within 3 months prior to screening.
  17. Have received any Respiratory Syncytial Virus (RSV) specific products, including palivizumab (Synagis®) within 3 months prior to screening.
  18. Have abused alcohol, opiates, psychotropic agents, or other chemicals or drugs within the past 12 months.
  19. Have any condition or abnormal laboratory assessment judged by the investigator to preclude participation in the study.
  20. Are currently pregnant or nursing.
  21. 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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: RI-002 Treatment
Drug: RI-002 Dose: 300-800 mg/kg infusion Frequency: Once every 3 to 4 Weeks
Biological: RI-002
Immune Globulin Intravenous (IGIV)
Other Names:
  • Immune Globulin (Human)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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
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.
One year

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Incidence of All Infections (Serious and Non-serious)
Time Frame: Up to 1 Year
Up to 1 Year
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
Up to 1 year
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
Up to 1 year
Number of Unscheduled Visits to Physician/ER Due to Infections - Total Number of Visits
Time Frame: Up to 1 year
Up to 1 year
Number of Unscheduled Visits to Physician/ER Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
Up to 1 year
Time to Resolution of Infections - Duration Per Infection
Time Frame: Up to 1 year
Up to 1 year
Time to Resolution of Infections - Infection Days Per Subject
Time Frame: Up to 1 year
Up to 1 year
Number of Hospitalizations Due to Infections
Time Frame: Up to 1 year
Up to 1 year
Number of Hospitalizations Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
Up to 1 year
Days of Hospitalization Due to Infections
Time Frame: Up to 1 year
Up to 1 year
Days of Hospitalization Due to Infections - Per Subject-Year
Time Frame: Up to 1 year
Up to 1 year
Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection)
Time Frame: Up to 1 year
Summary of the days of antibiotic therapy in the study for prophylaxis, as treatment for infections, and combined
Up to 1 year
Number of Days of Antibiotic Therapy (Prophylaxis and Treatment of Infection) - Per Subject-Year
Time Frame: Up to 1 year
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
Up to 1 year
Correlation Between Trough Level of RI-002 and Serious and Non-serious Infections
Time Frame: Up to 1 year
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)
Up to 1 year
Trough Total IgG and Specific Antibody Levels - IgG
Time Frame: Up to 1 year
Summary of trough total IgG concentration prior to specified infusion
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Haemophilus Influenzae Type B
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Haemophilus influenzae type B
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Respiratory Syncytial Virus (RSV)
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Respiratory Syncytial Virus (RSV)
Up to 1 year
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
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 1
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 1
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 3
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 3
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 4
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 4
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 5
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 5
Up to 1 year
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
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 7F
Time Frame: Up to 1 year
Summary of trough antibody concentrations prior to specified infusion for Streptococcus Pneumoniae, Serotype 7F
Up to 1 year
Trough Total IgG and Specific Antibody Levels - Streptococcus Pneumoniae, Serotype 9V
Time Frame: Up to 1 year
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
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
ADMA Biologics, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 1, 2014

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2014

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 6, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 18, 2013

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 20, 2013

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 5, 2016

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 10, 2016

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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