Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study.

• Register: EUCTR
• Last refreshed on: 19 November 2018
• Main ID: EUCTR2016-003438-26-GB
• Date of registration: 01/11/2016
• Prospective Registration: Yes
• Primary sponsor: Baxalta US Inc.
• Public title: Study to further assess the positive effect of the immunoglobulin product for subcutaneous use, HyQvia, in children (age <18 years) with Primary Immunodeficiency Diseases who have received prior immunoglobulin therapy before enrollment into the study.
• Scientific title: Post-Authorization Safety, Tolerability and Immunogenicity Evaluation of HyQvia in Pediatric Subjects with Primary Immunodeficiency Diseases
• Date of first enrolment: 14/02/2017
• Target sample size: 40
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003438-26
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Czech Republic, Denmark, France, Greece, Slovakia, Sweden, United Kingdom

Contacts

• Name: Fang Liu, Ph.D.
• Affiliation: Baxalta US Inc.

• Name: Fang Liu, Ph.D.
• Affiliation: Baxalta US Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Subject 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 IUIS (International Union of Immunological Societies) Scientific Committee 2015 1 prior to enrollment. The diagnosis must be confirmed by the sponsor Medical Director prior to first treatment with IP in the study.
2. Subject is at least two and below 18 years of age at the time of screening.
3. Subject has been receiving a consistent dose of 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/four weeks and a maximum dose equivalent to 1000 mg/kg BW/4 weeks.
4. Subject has a serum trough level of IgG > 5 g/L at screening.
5. If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
6. Subject/legally authorized representative is willing and able to comply with the requirements of the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Subject 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 amino transferase (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/mm3)
3. Subject has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site.
4. Subject has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
5. Subject has severe immunoglobulin A (IgA) deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity. .
6. Subject has a known allergy to hyaluronidase.
7. Subject has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
8. Subject 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. Subject has severe dermatitis that would preclude adequate sites for safe product administration in the opinion of the investigator.
10. Subject 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. Subject is a family member or employee of the investigator.
12. If female, subject is pregnant or lactating at the time of enrollment.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Primary Immunodeficiency Disease (PIDD)
MedDRA version: 20.0 Level: PT Classification code 10064859 Term: Primary immunodeficiency syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Trade Name: HyQvia 100 mg/ml solution for infusion for subcutaneous use
Product Name: HyQvia
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Trade Name: KIOVIG 100 mg/ml solution for infusion
Product Name: KIOVIG
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Human normal immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-

Trade Name: Cuvitru 200 mg/ml solution for subcutaneous injection
Product Name: Cuvitru
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Human Normal Immunoglobulin
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-

Primary Outcome(s)

Primary end point(s): Number and rate per infusion (excluding infections) of all severe related AEs
Number and rate per infusion (excluding infections) of related SAEs

Main Objective: Safety of HyQvia treatment in pediatric subjects with PIDD who have received prior immunoglobulin therapy before enrollment into the study.

Timepoint(s) of evaluation of this end point: Yearly

Secondary Objective: Further safety assessments (e.g. immunogenicity), tolerability, characteristic of product administration and efficacy (immunoglobulin G [IgG] trough levels).

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Efficacy: Up to three years.

Safety: Up to one year.

Secondary end point(s): Efficacy: Trough levels of IgG (for Study Epoch 1 and 2)

Safety/Tolerability:
Proportion of subjects who achieve a treatment interval of three or four weeks in Epoch 2
Proportion of subjects who maintain a treatment interval of three or four weeks in Epoch 2 for 12 months
Number and rate per infusion (excluding infections) of local AEs and ARs
Number and rate per infusion (excluding infections) of systemic AEs and ARs
Number and rate per infusion (excluding infections) of all AEs and all ARs
Number and rate per infusion (excluding infections) of all temporally associated AEs
Number and rate per infusion (excluding infections) of all causally related and/or temporally associated AEs
Number and rate per infusion (excluding infections) of all SAEs
Number/proportion of subjects who develop positive titer (=160) of binding or neutralizing antibodies to rHuPH20

Secondary ID(s)

161504

Source(s) of Monetary Support

Baxalta US Inc.