An Open Label, Multicenter Study to Evaluate the Pharmacokinetics, Efficacy and Safety of ASCENIV™ (IGIV) in Pediatric Subjects With Primary Immunodeficiency Diseases (PIDD)

NCT05070455 | Unknown Phase 4 FDA Drug

• 1 other identifier: ADMA-004
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

Brief Summary

This is a Phase IV, multicenter, open-label study of Asceniv™ administered as an intravenous infusion of Asceniv™ (IGIV) 300-800 mg/kg every 21 or 28 days in approximately 12 pediatric subjects with Primary Immunodeficiency Diseases (PIDD). The study will be conducted at 5-7 centers in the United States, with subjects receiving six (28 day cycle) or seven (21 day cycle) doses of Asceniv™ during the study.

Conditions

Primary Immune Deficiency

Outcome Measures

Primary Outcomes (6)

• Cmax

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

• Tmax

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

• AUC(0-ʈ)

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

• AUC(0-∞)

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

• Terminal phase elimination half-life (ʈ½)

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

• Terminal phase elimination rate (λZ)

  Pharmacokinetic measure at 6th or 7th infusion

  At prior to, end of infusion, and 60 minutes, 2 hours, 24 hours, 48 hours, 4 days, 7 days, 14 days, and either 21 or 28 days dependent upon infusion schedule

Secondary Outcomes (7)

• Total IgG Trough

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

• IgG Subclasses

  Prior to first and last infusions

• Antibodies

  Prior to first and last infusions

• Infections

  Up to approximately 7 months

• Serious Bacterial Infections

  Up to approximately 7 months

Study Arms (1)

Asceniv

EXPERIMENTAL

Asceniv™ will be given as an intravenous infusion at the same dose, or higher dose where medically appropriate, as the subject's previous IV Immunoglobulin G treatment (300-800 mg/kg) every 21 or 28 days.

Biological: Asceniv™

Interventions

Asceniv™ BIOLOGICAL

Each subject will receive an intravenous infusion of Asceniv™ on Study Day 1 (required to be within 28 days of screening) and every 21 or 28 days thereafter according to their current interval of IGIV treatment. Subjects will receive Asceniv™ at the same dose or higher dose if medically appropriate (300-800 mg/kg), every 21 or 28 days for five months (seven or six doses respectively).

Asceniv

Eligibility Criteria

• Age: 2 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subject and/or legal guardian must be able to understand the study procedures, have agreed to participate in the study and have voluntarily signed an IEC/IRB approved written informed consent. The consent form or a specific assent form, where required, will be signed and dated by minors.
• Have confirmed and documented clinical diagnosis of primary immunodeficiency disease including but not limited to: common variable immunodeficiency, X-linked and autosomal forms of agammaglobulinemia, hyper-IgM syndrome, or antibody deficiencies.
• Be male or female, and ≥ 2 years and \< 12 years at the time of informed consent by subject or legal guardian.
• Have been receiving IGIV at a dose that has not been changed by \> 25% of the mean dose on a mg/kg basis for at least 3 months prior to study entry.
• Have two trough levels of IgG in the last year (screening level may be used), and maintained a trough serum IgG level \> 500 mg/dL on the previous 2 assessments prior to receiving Asceniv™. (The trough level must be at least 300 mg/dL above pre-treatment serum IgG levels; with exception for cases of X-linked agammaglobulinemia where no pre-treatment value is available. Documentation will need to include dose, treatment interval and trade name of the IGIV products used for the three doses prior to the first Asceniv™ infusion in this study.
• 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. Females of non-childbearing potential are defined as prepubertal girls.

You may not qualify if:

• Have a known hypersensitivity to immunoglobulin or any excipient in Asceniv™.
• Have a history of any severe anaphylactic or anaphylactoid reaction to blood or any blood-derived product.
• Have a specific Immunoglobulin A (IgA) deficiency (IgA ≤ 5 mg/dL and normal IgG and IgM), history of allergic reaction to products containing IgA or has demonstrable antibodies to IgA.
• Have uncompensated, hemodynamically significant, congenital or other heart disease. Including but not limited to acute coronary syndromes and chronic stable angina.
• Have a medical condition that is known to cause secondary immune deficiency, such as chronic lymphocytic leukemia, lymphoma, multiple myeloma, or HIV infection.
• Have a significant T-cell or granulocyte deficiency in number or function (chronic or recurrent absolute neutrophil count \<1000 x 109/L).
• Have significant renal impairment (defined as an estimated Glomerular Filtration Rate ≤ 50 mL/min/1.73m2); or have a history of acute renal failure.
• Have abnormal liver function, defined as ALT or AST ≥ 2.5 x ULN.
• Have any chronic lung disease (uncontrolled or chronic, severe asthma, etc.)
• Have an infusion port, catheter, or other foreign body present (excluding PE tubes). Long-standing, infection-free ports may be permitted at the discretion of the Medical Monitor.
• Be planned or scheduled to undergo surgery during the course of study participation.
• Have ongoing failure to thrive per PI assessment.
• Be receiving chronic anti-coagulation therapy.
• Have a history of DVT, thrombotic or thrombo-embolic event, or are at increased risk for thrombotic event due to presence of, but not limited to, atrial fibrillation, disease or injury requiring prolonged immobilization, or other risk factor(s) including significant proteinuria or protein losing enteropathy.
• Current daily use of the following medications:

Sponsors & Collaborators

• ADMA Biologics, Inc.: lead

Study Sites (3)

Immunoe Research Centers

Centennial, Colorado, 80112, United States

NOT YET RECRUITING

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84112, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Primary Immunodeficiency Diseases

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Rebecca Avila

CONTACT

561-989-5853; reavila@admabio.com

Amy Doman

CONTACT

adoman@admabio.com

Study Design

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

Study Record Dates

• First Submitted: September 23, 2021
• First Posted: October 7, 2021
• Study Start: September 1, 2022
• Primary Completion: March 31, 2023
• Study Completion: June 30, 2023
• Last Updated: September 16, 2022

Record last verified: 2022-09

Locations

US (3)