Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric PIDD Subjects

NCT ID: NCT03116347

Last Updated: 2023-01-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 42 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-05-30
• Study Completion Date: 2021-01-15

Brief Summary

The purpose of the study is to acquire additional data on safety, tolerability and immunogenicity of HyQvia in pediatric (age two to <18 years) patients with Primary Immunodeficiency Diseases (PIDD)

Conditions

Primary Immunodeficiency Diseases (PID)

Study Design

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

Study Groups

EPOCH 1

Ramp up period for participants who were not treated with HyQvia prior to this study

Group Type: EXPERIMENTAL

HYQVIA

Intervention Type: BIOLOGICAL

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

EPOCH 2

Participants who were treated with HyQvia prior to this study, and those who completed the ramp up period (Epoch 1). After one year in Epoch 2, participants with anti-rHuPH20 antibody titer \<160 at all time-points during the study will complete the study termination/completion visit at the next possible occasion. Participants with anti-rHuPH20 antibody titer \>=160 during the study and/or at the last measurement will continue for an additional two years of HyQvia treatment and observation.

Group Type: EXPERIMENTAL

HYQVIA

Intervention Type: BIOLOGICAL

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

Epoch 3

Safety follow-up for participants whose anti-rHuPH20 antibody titer was \>= 160 during Epoch 1 or Epoch 2 and who experience either a related serious adverse event (SAE) or a related severe adverse event (AE)

Group Type: EXPERIMENTAL

KIOVIG

Intervention Type: BIOLOGICAL

100 mg/ml solution for Immune Globulin Intravenous Infusion

Cuvitru

Intervention Type: BIOLOGICAL

200 mg/ml solution for Immune Globulin Subcutaneous Injection

Interventions

HYQVIA

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

Intervention Type: BIOLOGICAL

KIOVIG

100 mg/ml solution for Immune Globulin Intravenous Infusion

Intervention Type: BIOLOGICAL

Cuvitru

200 mg/ml solution for Immune Globulin Subcutaneous Injection

Intervention Type: BIOLOGICAL

Other Intervention Names

IGI 10% with rHuPH20; IGIV 10%; IGI 10%; IGSC 20%; IGI 20%

Eligibility Criteria

Inclusion Criteria

1. Participant must have a documented diagnosis of a form of primary humoral immunodeficiency involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies (IUIS) Scientific Committee 2015 prior to enrollment. The diagnosis must be confirmed by the sponsor´s Medical Director prior to first treatment with investigational product (IP) in the study.

2. Participant is at least two and below 18 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 body weight (BW)/four weeks and a maximum dose equivalent to 1000 mg/kg 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 amino transferase (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 (< 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 < 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: 17 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

Fakultni nemocnice Brno Odd. Detska klinika

Brno, , Czechia

Fakultní nemocnice Hradec Králové

Nový Hradec Králové, , Czechia

FN v Motole Interni klinika

Prague, , Czechia

Rigshospitalet

Copenhagen, , Denmark

Groupe Hospitalier Pellegrin - Hôpital des Enfants

Bordeaux, Gironde, France

CHU Angers - Hôpital Hôtel Dieu

Angers, , France

Hospices Civils de Lyon - Hôpitaux Est - IHOP

Lyon, , France

Agia Sophia Children's Hospital

Athens, , Greece

General Hospital of Thessaloniki Ippokrateio

Thessaloniki, , Greece

General Hospital of Thessaloniki Papageorgiou

Thessaloniki, , Greece

United St. Istvan and St. Laszlo Hospital

Budapest, , Hungary

1.Detská klinika

Bratislava, , Slovakia

Univerzitna Nemocnica Klinika detí a dorastu

Martin, , Slovakia

Queen Silvia Children's Hospital

Gothenburg, , Sweden

Dept. of Pediatric Oncology/Hematology/Immunology-Skånes Universitetssjukhus

Lund, , Sweden

Bristol Royal Hospital for Children

Bristol, Avon, United Kingdom

Leeds Children's Hospital

Leeds, West Yorkshire, United Kingdom

Royal Victoria Hospital

Belfast, , United Kingdom

University Hospital of Wales Heath Park Clinical Research Facility

Cardiff, , United Kingdom

The Great North Children's Hospital Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Countries

Czechia; Denmark; France; Greece; Hungary; Slovakia; Sweden; United Kingdom

References

Ciznar P, Roderick M, Schneiderova H, Jesenak M, Krivan G, Brodszki N, Jolles S, Atisso C, Fielhauer K, Saeed-Khawaja S, McCoy B, Yel L. fSCIG 10% in pediatric primary immunodeficiency diseases: a European post-authorization safety study. Allergy Asthma Clin Immunol. 2024 Sep 17;20(1):47. doi: 10.1186/s13223-024-00904-9.

Reference Type: DERIVED

PMID: 39289739 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2016-003438-26

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

161504

Identifier Type: -

Identifier Source: org_study_id