Pharmacokinetics, Efficacy, and Safety Study of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
ADMA Biologics, Inc.
ClinicalTrials.gov Identifier:
NCT01814800
First received: March 6, 2013
Last updated: November 25, 2013
Last verified: November 2013
  Purpose

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).


Condition Intervention Phase
Primary Immunodeficiency Disorders
Biological: RI-002
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label, Multicenter, Study to Evaluate the Pharmacokinetics, Efficacy and Safety of RI-002 (IGIV) in Subjects With Primary Immunodeficiency Diseases (PIDD)

Resource links provided by NLM:


Further study details as provided by ADMA Biologics, Inc.:

Primary Outcome Measures:
  • Demonstrate that RI-002 prevents the frequency of serious bacterial infections(FDA Guidance for Industry (2008)) [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Incidence of all infections (serious and non-serious) [ Time Frame: Up to 1 Year ] [ Designated as safety issue: No ]
  • Number of days lost from work/school/usual activities due to infections and their treatment [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Number of unscheduled visits to physician/ER due to infections [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Time to resolution of infections [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Number of hospitalizations and days of hospitalizations due to infections [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Number of days of antibiotic therapy [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Relationship among dose of RI-002 and serious and non-serious infections [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Evaluate trough total IgG and specific antibody levels(streptococcus pneumoniae, H. influenza type B, CMV, measles, tetanus, and RSV) [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]
  • Relationship among trough level of RI-002 and serious and non-serious infections [ Time Frame: Up to 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: February 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: September 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RI-002 Treatment
Drug: RI-002 Dose: 300-800 mg/kg infusion
Biological: RI-002
Other Name: Immune Globulin (Human)

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.

  Eligibility

Ages Eligible for Study:   2 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 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 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 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01814800

Locations
United States, Colorado
IMMUNOe Health Centers
Cenntennial, Colorado, United States, 80112
United States, Florida
Allergy Associates of the Palm Beaches, P.A.
North Palm Beach, Florida, United States, 33408
United States, Georgia
Family Allergy Center, PC
Atlanta, Georgia, United States, 30342
United States, Indiana
The South Bend Clinic, LLP
South Bend, Indiana, United States, 46617
United States, Nebraska
Asthma & Immunology Associates
Omaha, Nebraska, United States, 68124
United States, New York
Mount Sinai School of Medicine
NY, New York, United States, 10029
United States, Texas
Dallas Immunology Research
Dallas, Texas, United States, 75230
AARA Research Center
Dallas, Texas, United States, 75231
Baylor Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
ADMA Biologics, Inc.
Investigators
Study Director: James Mond, M.D., Ph.D. ADMA Biologics, Inc.
  More Information

No publications provided

Responsible Party: ADMA Biologics, Inc.
ClinicalTrials.gov Identifier: NCT01814800     History of Changes
Other Study ID Numbers: ADMA-003
Study First Received: March 6, 2013
Last Updated: November 25, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by ADMA Biologics, Inc.:
PIDD, PID, humoral immunity, antibody deficiency

Additional relevant MeSH terms:
Immunologic Deficiency Syndromes
Immune System Diseases
Immunoglobulins
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 27, 2014