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

NCT01814800 | Completed Phase 3 Results

• 1 other identifier: ADMA-003
• Study Type: interventional
• Target: 59
• Locations: 1 country
• Sites: 9

Brief Summary

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).

Conditions

Primary Immune Deficiency Disorder

Keywords

PIDD, PID, humoral immunity, antibody deficiency

Outcome Measures

Primary Outcomes (1)

• Number of Serious Bacterial Infections (SBIs) Per Subject Per Year (FDA Guidance for Industry (2008))

  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 Outcomes (30)

• Incidence of All Infections (Serious and Non-serious)

  Up to 1 Year

• Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Combined Days Lost

  Up to 1 year

• Number of Days Lost From Work/School/Daycare and Usual Activities Due to Infections and Their Treatment - Per Subject-Year

  Up to 1 year

• Number of Unscheduled Visits to Physician/ER Due to Infections - Total Number of Visits

  Up to 1 year

• Number of Unscheduled Visits to Physician/ER Due to Infections - Per Subject-Year

  Up to 1 year

Study Arms (1)

RI-002 Treatment

EXPERIMENTAL

Drug: RI-002 Dose: 300-800 mg/kg infusion Frequency: Once every 3 to 4 Weeks

Biological: RI-002

Interventions

RI-002 BIOLOGICAL

Immune Globulin Intravenous (IGIV)

Also known as: Immune Globulin (Human)

RI-002 Treatment

Eligibility Criteria

• Age: 2 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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))

Sponsors & Collaborators

• ADMA Biologics, Inc.: lead

Study Sites (9)

IMMUNOe Health Centers

Cenntennial, Colorado, 80112, United States

Location

Allergy Associates of the Palm Beaches, P.A.

North Palm Beach, Florida, 33408, United States

Location

Family Allergy Center, PC

Atlanta, Georgia, 30342, United States

Location

The South Bend Clinic, LLP

South Bend, Indiana, 46617, United States

Location

Asthma & Immunology Associates

Omaha, Nebraska, 68124, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

Dallas Immunology Research

Dallas, Texas, 75230, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

Baylor Texas Children's Hospital

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Interventions

gamma-Globulins

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Immunologic Deficiency Syndromes; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Results Point of Contact

• Title: Jimmy Mond, M.D., Ph.D
• Organization: ADMA Biologics

jmond@admabio.com

201-478-5552

Study Officials

• James Mond, M.D., Ph.D.

  ADMA Biologics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2013
• First Posted: March 20, 2013
• Study Start: February 1, 2014
• Primary Completion: December 1, 2014
• Study Completion: January 1, 2015
• Last Updated: October 5, 2016
• Results First Posted: October 5, 2016

Record last verified: 2016-08

Locations

US (9)