Study to Evaluate Safety and Pharmacokinetics of BIVIGAM® in Primary Immune Deficiency Subjects Aged 2 to 16

NCT03164967 | Completed Phase 4 DMC FDA Drug

• 1 other identifier: 994
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 8

Brief Summary

This study is part of the BIVIGAM® post marketing requirement (PMR). It is being conducted in subjects aged 2-16 with primary immune deficiency disorders associated with defects in humoral immunity to generate additional data on these populations, and more specifically safety and pharmacokinetic (PK) assessments.

Conditions

Humoral Immune Response

Outcome Measures

Primary Outcomes (13)

• Temporally Associated Adverse Events

  Incidence of adverse events (During or within 1 hour, 24 hours and 72 hours of completion of an infusion)

  During each infusion (During or within 1 hour, 24 hours and 72 hours of completion of an infusion)

• Number of Temporally Associated Adverse Events

  Mean number of temporally associated per infusion

  Up to 72 hours of completion of an infusion

• Serious Adverse Events

  Incidence of serious adverse events

  Up to approximately 7 months

• Related Serious Adverse Events

  Incidence of related serious adverse events

  Up to approximately 7 months

• Treatment Emergent Adverse Events

  Incidence of treatment emergent adverse events

  Up to approximately 7 months

• Related Treatment Emergent Averse Events

  Incidence of adverse events that first appear, or that worsen relative to the pre-treatment state, which occur during and within 72 hours of treatment administration

  Within 72 hours of infusion

• Non-treatment Emergent Adverse Events

  Incidence of adverse events which do not have a causal relationship with study treatment

  Up to approximately 7 months

• Temporally Associated Infusion Adverse Events

  Incidence of adverse events which have a causal relationship with infusion treatment

  Up to approximately 7 months

• Adverse Reactions

  Number and incidence of adverse reactions plus suspected adverse reactions combined

  Up to approximately 7 months

• Related Adverse Reactions

  Incidence of adverse infusion related reactions

  Up to approximately 7 months

• Infusion Site Reactions

  Incidence reactions occuring at the infusion site

  Up to approximately 7 months

• Vital Signs

  Change in vital signs

  Before and after each administration of study drug through study completion, up to approximately 7 months

• Temporally Associated Adverse Events Following Infusions

  Incidence of adverse events

  Up to 72 hours after each infusion through study completion, up tp approximately 7 months

Secondary Outcomes (18)

• Total IgG Trough

  At each visit through study completion, up tp approximately 7 months

• IgG subclasses

  Prior to first and last infusion, up tp approximately 7 months

• Total IgG Post

  At each infusion through study completion, up tp approximately 7 months

• Cmax

  At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months

• Tmax

  At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months

Study Arms (1)

Active Drug

OTHER

All subjects will receive Bivigam based on their prior dosing to be adjusted as clinically necessary.

Biological: Bivigam

Interventions

Bivigam BIOLOGICAL

Active Drug

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Written informed consent/Assent
• Male or female between 2 and 16 years, inclusive, at time of Signing Informed Consent/Assent
• Have a confirmed and documented clinical diagnosis of Primary Immune Deficiency Disorder, including hypogammaglobulinemia or agammaglobulinemia.
• Have received IGIV therapy which was maintained at a steady dose (± 25% of the mean dose) for at least 3 months prior to study entry, and have maintained a trough IgG level at least 500mg/dL prior to receiving BIVIGAM®.
• Subjects and/or parents/legal guardians must be able to understand and adhere to the study visit schedule and all other protocol requirements.

You may not qualify if:

• Known intolerance to immunoglobulins or comparable substances (e.g. vaccination reaction).
• Known intolerance to proteins of human origin or known allergic reactions to components of the study product(s).
• Any previous randomization/participation in this clinical study must be discussed with and approved by the medical director (or designee).
• Inability or lacking motivation to participate in the study.
• Medical condition, laboratory finding, or physical exam finding (specify, e.g., vital signs outside of specific range that precludes participation. Per lab results at the Screening visit through Baseline.
• Confirmed Screening visit laboratory results ˃2.5 X ULN as defined for pediatric populations for any of the following: ALT (alanine aminotransferase/SGPT), AST (aspartate aminotransferase/SGOT), LDH (lactate dehydrogenase), BUN (blood urea nitrogen), Serum creatinine
• Has selective IgA deficiency or demonstrated antibodies to IgA.
• History of thrombotic complications of IGIV therapy or history of (deep vein thrombosis)DVT.
• Current use of daily corticosteroids (\>10 mg of prednisone equivalent/day),immunosuppressants or immunomodulators are not allowed unless approved in advance by the medical monitor. Intermittent use of corticosteroids during the study is allowed if medically necessary.
• Positive diagnosis of hepatitis B or hepatitis C.
• Positive human immunodeficiency virus (HIV) test.
• Subject has had a serious bacterial infection (SBI) within the last 3 months.
• Subject has an active infection and is receiving antibiotic therapy for the treatment of this infection at the time of Screening. Note: if the subject is deemed a Screen Failure due to a nonserious active infection requiring antibiotic therapy, the subject may be rescreened 3 or 4 weeks (depending on drug administration schedule) after the initial screening.
• Subject has a history of thrombotic events (including deep vein thrombosis, myocardial infarction, cerebrovascular accident and pulmonary embolism) within 6 months before 1st IGIV dose or has preexisting risk factors for thrombotic events.
• Acquired medical condition known to cause secondary immune deficiency such as chronic lymphacitic leukemia, lymphoma or multiple lymphoma.

Sponsors & Collaborators

• ADMA Biologics, Inc.: lead

Study Sites (8)

IMMUNOe Research Centers

Centennial, Colorado, 80112, United States

Location

Duke University Medical Center

Durham, Florida, 27710, United States

Location

Allergy Associates of the Palm Beaches

North Palm Beach, Florida, 33408, United States

Location

USF Health, Pediatric Allergy, Immunology & Rheumatology

St. Petersburg, Florida, 33701, United States

Location

Ohio Clinical Research Associates

Mayfield Heights, Ohio, 44124, United States

Location

Oklahoma Institute of Allergy and Asthma Clinical Research

Oklahoma City, Oklahoma, 73131, United States

Location

Discovery Clinical Trials

Dallas, Texas, 75225, United States

Location

Lysosomal Rare Disorders Research & Treatment Center

Fairfax, Virginia, 22030, United States

Location

MeSH Terms

Interventions

Immunoglobulin G

Intervention Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 30, 2017
• First Posted: May 24, 2017
• Study Start: December 29, 2016
• Primary Completion: August 30, 2022
• Study Completion: December 31, 2022
• Last Updated: February 1, 2023

Record last verified: 2023-01

Locations

US (8)