Now Recruiting

Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects

WHAT IS THE PURPOSE OF THIS STUDY?

The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary immunodeficiency disease (PIDD).

TRIAL AT A GLANCE

Condition

Primary Immunodeficiency Diseases (PID)

Study Intervention

HYQVIA; GAMMAGARD LIQUID

Possibility of Active Treatment

Study Type

Interventional

Phase

Phase 3

IS THIS STUDY RIGHT FOR ME?

AGE

2 to 15

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

ENTRY CRITERIA

Inclusion Criteria: 1. Participant must have a documented diagnosis of a form of primary immunodeficiency (PI) involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies (IUIS) Scientific Committee 2015 1 prior to enrollment. The diagnosis must be confirmed by the sponsor´s Medical Director prior to first treatment with IP in the study. 2. Participant is at least two and below 16 years of age at the time of screening. 3. Participant has been receiving a consistent dose of Immunoglobulin G (IgG), administered in compliance with the respective product information for a period of at least three months prior to screening. The average minimum pre-study dose over that interval was equivalent to 300 mg/kg BW / 4 weeks and a maximum dose equivalent to 1000 mg/kg body weight (BW) / 4 weeks. 4. Participant has a serum trough level of IgG > 5 g/L at screening. 5. If female of childbearing potential, participant presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study. 6. Participant /legally authorized representative is willing and able to comply with the requirements of the protocol.

Exclusion Criteria: 1. Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2. 2. Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent): a. Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 2.5 times the upper limit of normal (ULN) for the testing laboratory b. Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] ≤ 500/mm^3) 3. Participant has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site. 4. Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following intravenous (IV) immunoglobulin, subcutaneous (SC) immunoglobulin, and/or Immune Serum Globulin (ISG) infusions. 5. Participant has severe Immunoglobulin A (IgA) deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity. 6. Participant has a known allergy to hyaluronidase. 7. Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening. 8. Participant has a bleeding disorder or a platelet count less than 20,000/μL, or who, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of SC therapy. 9. Participant has severe dermatitis that would preclude adequate sites for safe product administration in the opinion of the investigator. 10. Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. 11. Participant is a family member or employee of the investigator. 12. If female, participant is pregnant or lactating at the time of enrollment.

Study Locations

We are planning* on conducting this study in 1 countries around the world.

    • United States of America

Enter your city or postal code to find a location near you.

RESULTS

  • 1.

    OK Institute of Allergy & Asthma Clinical Research, LLC

    Oklahoma City, Oklahoma
    United States of America

    (405) 607-4333

    lwalker@okallergy.com

    miles

  • 2.

    Allergy Asthma & Immunology Relief of Charlotte

    Charlotte, North Carolina
    United States of America

    (704) 910-1402

    atash@aairofcharlotte.com

    miles

  • 3.

    Northwell Health, Inc. PRIME

    Great Neck, New York
    United States of America

    (516) 622-5070

    mjalal@northwell.edu

    miles

  • 4.

    Connecticut Children’s Medical Center

    Hartford, Connecticut
    United States of America

    (860) 837-5881

    hnielsen@connecticutchildrens.org

    miles

  • 5.

    University of Miami Pediatric Allergy and Immunology

    Miami, Florida
    United States of America

    (305) 243-4304

    toa11@med.miami.edu

    miles

  • 6.

    University of South Florida Physician Group

    St. Petersburg, Florida
    United States of America

    (727) 553-1259

    mellison@health.usf.edu

    miles

  • 7.

    Stony Brook Children’s Hospital

    Stony Brook, New York
    United States of America

    (631) 444-8832

    silvia.muniz@stonybrookmedicine.edu

    miles

  • 8.

    Marshall University Joan C. Edwards School of Medicine

    Huntington, West Virginia
    United States of America

    (304) 691-1830

    dellera@marshall.edu

    miles

  • 9.

    Section on Immunopathogenesis and Clinical Immunology

    Fairfax, Virginia
    United States of America

    (571) 308-1900

    ogokeralpan@oandoalpan.com

    miles

  • 10.

    Georgia Pollens Clinical Research Centers, Inc.

    Albany, Georgia
    United States of America

    (229) 883-8909

    jowens@aacoga.com

    miles

*Planning is an intent to conduct this study in the specified countries and is subject to change.