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

NCT ID: NCT03277313

Last Updated: 2023-10-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-09-25
• Study Completion Date: 2022-07-20

Brief Summary

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).

Conditions

Primary Immunodeficiency Diseases (PID)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Epoch 1

Pediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.

Group Type: EXPERIMENTAL

HYQVIA

Intervention Type: BIOLOGICAL

Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (IGI, 10% with rHuPH20)

Epoch 2

Epoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.

Group Type: EXPERIMENTAL

HYQVIA

Intervention Type: BIOLOGICAL

Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (IGI, 10% with rHuPH20)

Interventions

HYQVIA

Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (IGI, 10% with rHuPH20)

Intervention Type: BIOLOGICAL

Other Intervention Names

IGI 10% with rHuPH20

Eligibility 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 (Picard et al., 2015) 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):

1. Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 2.5 times the upper limit of normal (ULN) for the testing laboratory

2. 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.

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 15 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxalta Innovations GmbH, now part of Shire

INDUSTRY

Sponsor Role: collaborator

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Shire

Locations

University of Alabama Medical Center

Birmingham, Alabama, United States

Connecticut Children's Medical Center

Hartford, Connecticut, United States

University of Miami Pediatric Allergy and Immunology

Miami, Florida, United States

University of South Florida Physician Group

St. Petersburg, Florida, United States

Georgia Pollens Clinical Research Centers, Inc.

Albany, Georgia, United States

Emory Healthcare

Atlanta, Georgia, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Washington University

St Louis, Missouri, United States

Women & Children's Hospital of Buffalo

Buffalo, New York, United States

Northwell Health, Inc. PRIME

Great Neck, New York, United States

Stony Brook Children's Hospital

Stony Brook, New York, United States

Carolinas Healthcare System

Charlotte, North Carolina, United States

Allergy Asthma & Immunology Relief of Charlotte

Charlotte, North Carolina, United States

OK Institute of Allergy & Asthma Clinical Research, LLC

Oklahoma City, Oklahoma, United States

Vital Prospects Clinical Research Institute, P.C.

Tulsa, Oklahoma, United States

Allergy Partners of North Texas Research

Dallas, Texas, United States

University of Utah

Salt Lake City, Utah, United States

Section on Immunopathogenesis and Clinical Immunology

Fairfax, Virginia, United States

Marshall University Joan C. Edwards School of Medicine

Charleston, West Virginia, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://clinicaltrials.takeda.com/study-detail/5f6b5fc54db2bf003ab45c6c

To obtain more information on the study, click here/on this link

Other Identifiers

161503

Identifier Type: -

Identifier Source: org_study_id